[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Biogen\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":464},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,20,0,[8,43,70,95,118,143,166,187,208,230,253,274,298,321,341,361,381,402,427,446],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100587974","phase-3-a-study-to-learn-about-the-effects-of-felzartamab-infusions-on-adults-with-immunoglobulin-a-nephropathy-igan-100587974",false,"NCT06935357","A Study to Learn About the Effects of Felzartamab Infusions on Adults With Immunoglobulin A Nephropathy (IgAN)","A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Felzartamab in Adults With IgA Nephropathy (PREVAIL)","PREVAIL","Key Inclusion Criteria:\n\n* Biopsy-confirmed diagnosis of IgAN within the past 10 years prior to signature of the informed consent form (ICF). For participants with diabetes mellitus type 2, an IgAN diagnostic biopsy within the past 24 months prior to signing the ICF.\n* An eGFR ≥ 30 mL\u002Fmin\u002F1.73 m\\^2 at Screening as calculated using the 2021 chronic kidney disease epidemiology (CKD-EPI) creatinine formula. An eGFR of ≥ 20 and \\\u003C 30 mL\u002Fmin\u002F1.73 m\\^2 is acceptable for the cohorts 3 and 4.\n* Proteinuria of ≥ 1.0 gram per day (g\u002Fday) or UPCR ≥0.8 gram per gram (g\u002Fg) as assessed by an adequate 24-hour urine collection.\n* Clinically stable on a maximally tolerated dose or maximally approved dose of angiotensin-converting enzyme inhibitors (ACEI) or angiotensin receptor blockers (ARB) for at least 12 weeks prior to Screening. Participants may also be using sodium-glucose cotransporter-2 inhibitors (SGLT2is); endothelin receptor antagonists (ERAs) or dual endothelin angiotensin receptor antagonist (DEARAs) approved for the treatment of IgAN; and\u002For mineralocorticoid receptor antagonists (MRAs) as long as the dose is stable for at least 12 weeks prior to Screening. Participants should remain on stable doses of these background medications for the duration of the study. Once the ICF is signed and thereafter, the doses cannot be changed during the study nor the drugs discontinued except if deemed related to an AE. Participants using a DEARA (e.g., sparsentan) will not be permitted to use simultaneous ACEI or ARB medication.\n\nKey Exclusion Criteria:\n\n* Any history of secondary forms of IgAN, indicated by the presence of any other systemic disease potentially leading to IgA deposits as determined by the Investigator.\n* History of rapidly progressive variant of IgAN, defined as eGFR loss by \\> 50% per 3 months and not explained by changes in renin-angiotensin system (RAS) blockade or other factors.\n* IgAN-(MCD) variant.\n* Concomitant other progressive glomerulonephritis or non-immunologic glomerular disease such as diabetic nephropathy.\n* Type 2 diabetes mellitus with Hemoglobin A1c (HbA1c) \\> 8% at Screening, or evidence of diabetic nephropathy on biopsy, history of diabetic microvascular or macrovascular disease (eg, diabetic retinopathy, peripheral neuropathy).\n* Any diagnosed or suspected immunosuppressed or immunodeficient state such as asplenia, human immunodeficiency virus (HIV), primary immunodeficiencies, organ or bone marrow transplantation, with the exception of corneal transplants.\n* Previously treated with immunosuppressive or other immunomodulatory agents such as but not limited to cyclophosphamide, rituximab, infliximab, eculizumab, canakinumab, mycophenolate mofetil (MMF) or mycophenolate sodium (MPS), cyclosporine, tacrolimus, sirolimus, everolimus, or systemic glucocorticoid exposure (\\> 7.5 milligrams per day \\[mg\u002Fd\\] prednisone-equivalent for indications other than IgAN or any dose if given for the treatment of IgAN) within 4 months prior to Screening. Use of hydroxychloroquine in Mainland China is allowed if the candidate has been on this for at least 6 months prior to Screening with a stable dose for at least 12 weeks prior to Screening. If a potential participant requires systemic glucocorticoids at any dose for IgAN during Screening, this will result in a screen fail. If a potential participant requires systemic glucocorticoids \\> 7.5 mg\u002Fday prednisone-equivalent for indications other than IgAN during Screening, this will result in a screen fail.\n* Participants currently treated with oral budesonide. Participants who have stopped this therapy ≥ 4 months prior to Screening may be eligible.\n* Active clinically significant infections, known history of recurrent clinically significant infection, or Screening laboratory evidence consistent with an active infection, or non-prophylactic treatment with IV anti-infectives (antibacterials, antiviral or antifungals). Participants with a history of opportunistic infections are excluded.\n* Hypogammaglobulinemia: serum Immunoglobin G (IgG) \\\u003C 6.0 gram per litre (g\u002FL), at Screening.\n\nNote: Other protocol-defined Inclusion\u002FExclusion criteria may apply.","ALL","18 Years",{"count":20,"type":21},454,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","In this study, researchers will learn more about the use of felzartamab in participants with immunoglobulin A nephropathy (IgAN). IgAN is a kidney disease caused by the buildup of an antibody called IgA in the kidneys over time. In people with IgAN, abnormal IgA and other antibodies form clusters that build up in the small filters of the kidneys, which leads to inflammation and damage. Felzartamab is designed to target certain immune cells that produce these abnormal antibodies. This study will focus on participants who have protein in their urine (proteinuria) as a result of damaged kidneys.\n\nThe main goal of the study is to learn about the effect felzartamab has on proteinuria. The main question that researchers want to answer is:\n\n• How much does the amount of protein in the urine change from the start of the study to Week 36?\n\nResearchers will learn about the effect felzartamab has on the kidneys' ability to filter blood. They will also learn more about the safety of felzartamab and how it is processed by the body.\n\nThe study will be done as follows:\n\n* Participants will be screened to check if they can join the study.\n* Participants will be randomized to receive either felzartamab or a placebo. A placebo looks like the study drug but contains no real medicine.\n* Neither the researchers nor the participants will know what the participants will receive.\n* Participants will receive felzartamab or placebo as intravenous (IV) infusions. The treatment period will last 24 weeks.\n* Afterwards, participants will enter a follow-up period which will last 80 weeks.\n* In total, participants will have 17 study visits. Participants will stay in the study for about 2 years.",[27],"Immunoglobulin A Nephropathy (IgAN)",[29],"Felzartamab","RECRUITING","2026-06-30",{"date":33,"type":34},"2026-07-01","ACTUAL",{"date":36,"type":34},"2025-05-08",{"date":38,"type":21},"2029-06-05",{"name":40,"class":41},"Biogen","INDUSTRY",246,{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":48,"acronym":49,"eligibilityCriteria":50,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":51,"enrollmentInfo":52,"targetDuration":4,"studyType":22,"phases":54,"briefSummary":56,"conditions":57,"keywords":59,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":62,"lastUpdatePostDateStruct":63,"startDateStruct":64,"completionDateStruct":66,"leadSponsor":68,"locationsCount":69},"100609781","phase-2-a-study-to-learn-about-the-effects-of-felzartamab-infusions-in-adults-with-kidney-transplants-who-have-late-isolated-microvascular-inflammation-100609781","NCT07219043","A Study to Learn About the Effects of Felzartamab Infusions in Adults With Kidney Transplants Who Have Late Isolated Microvascular Inflammation","A Double-Blind, Placebo-Controlled, Multicenter, Randomized Phase 2 Trial Evaluating the Efficacy and Safety of Felzartamab in Recipients of Kidney Transplants With Late Isolated Microvascular Inflammation (MVI)","TRANSPIRE","Key Inclusion Criteria:\n\n* MVI (MVI ≥2), donor specific antibody (DSA)-negative that is either complement activation (C4d) negative or C4d positive (biopsy-confirmed) without T cell-mediated rejection (TCMR) per central reading, as defined by the Banff 2022 criteria.\n* Biopsy must be within 3 months (preferably within 1 month) prior to randomization and meet adequate criteria (option a preferred over option b):\n\n  1. Adequate: 10 or more non-sclerotic\u002Fevaluable glomeruli and two muscular arteries\n  2. Minimally Adequate: at least 7 non-sclerotic\u002Fevaluable glomeruli and one muscular artery\n* For participants who received any prior treatment for antibody-mediated rejection (AMR), MVI, or TCMR as outlined in Exclusion Criterion 5, the biopsy must be performed at least 6 weeks after completing (or stopping) prior treatment.\n* Kidney transplant at least 6 months prior to Screening visit (recipients of either living or deceased donors).\n* DSA: Human leukocyte antigen (HLA) Class I and II antigen-specific DSA-negative (preformed and de novo DSA) as determined by the local laboratory's definition of positivity using single-antigen bead-based assays within 3 months prior to randomization.\n\nKey Exclusion Criteria:\n\n* Transplant: Blood type (ABO)-incompatible transplant.\n* History of multiple organ transplants including en bloc and dual kidney transplants.\n* Presence of HLA donor-specific antibodies.\n* Acute, rapid decline in renal function, defined as a participant likely to require renal replacement therapy within the next 30 days as determined by the Investigator.\n* Prior AMR or TCMR treatment (with the exception of corticosteroids) within 3 months prior to randomization is excluded as listed below. Participants who received any of these treatments between 3 and 6 months prior to randomization must have both a renal biopsy (IC3) and DSA testing at least 6 weeks after completing (or stopping) treatment in order to confirm continuing MVI≥2 and DSA negative status and to determine eligibility:\n\n  1. Intravenous or subcutaneous immunoglobulin (IVIg or subcutaneous immunoglobulin \\[SCIg\\]) or plasma exchange (PLEX).\n  2. Complement system inhibitors (e.g., eculizumab).\n  3. Proteasome inhibitors (e.g., bortezomib).\n  4. The anti-interleukin-6 receptor (anti-IL-6R) tocilizumab.\n  5. Any B cell-depleting therapy (including anti-CD20 agents \\[e.g., rituximab\\]) within 3 months prior to randomization.\n  6. Any other investigational agent within 3 months or 5 half-lives (whichever is longer) of randomization.\n\nNote: Other protocol-defined Inclusion\u002FExclusion criteria may apply.","74 Years",{"count":53,"type":21},81,[55],"PHASE2","In this study, researchers will learn more about a drug called felzartamab in people who have received a kidney transplant and later developed a condition called microvascular inflammation (MVI). MVI is a type of injury to small blood vessels in the transplanted kidney and may be a sign of rejection by the body. It can lead to serious kidney problems over time.\n\nIn many cases, MVI is caused by antibodies that attack the transplanted kidney. But in some people, MVI happens without these antibodies. This type of MVI is called isolated MVI. There are currently no approved treatments for isolated MVI.\n\nThe main goal of the study is to learn about the effect felzartamab has on inflammation in the transplanted kidney. The main question researchers want to answer is:\n\n• How many participants have no signs of active inflammation in the transplanted kidney after 24 weeks of treatment with felzartamab?\n\nResearchers will also study how felzartamab affects kidney function, immune activity, and overall health. They will monitor safety through kidney biopsies, lab tests, and by recording adverse events throughout the study.\n\nAdverse events are health problems that may or may not be caused by the study drug.\n\nThe study will be done in 2 parts as follows:\n\n* Participants will be randomly assigned to receive either felzartamab or a placebo. A placebo looks like the study drug but contains no real medicine.\n* In Part A, participants will receive their assigned drug for 24 weeks. Neither the researchers nor the participants will know who is receiving felzartamab or placebo.\n* Part B will last another 28 weeks. All participants will receive felzartamab and both participants and researchers will know this.\n* All treatments will be given by intravenous (IV) infusion at the study site.\n* Participants will have kidney biopsies at the start of the study, at Week 24, and at Week 52 to help measure changes in inflammation.\n* Participants will stay in the study for about 1 year.",[58],"Microvascular Inflammation",[60,61],"Kidney Transplant","Kidney Transplant Rejection","2026-06-29",{"date":31,"type":34},{"date":65,"type":34},"2026-01-05",{"date":67,"type":21},"2028-02-10",{"name":40,"class":41},38,{"id":71,"slug":72,"hasResults":11,"nctId":73,"briefTitle":74,"officialTitle":75,"acronym":4,"eligibilityCriteria":76,"healthyVolunteers":77,"sex":17,"minAge":18,"maxAge":78,"enrollmentInfo":79,"targetDuration":4,"studyType":22,"phases":81,"briefSummary":83,"conditions":84,"keywords":4,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":87,"lastUpdatePostDateStruct":88,"startDateStruct":90,"completionDateStruct":92,"leadSponsor":94,"locationsCount":4},"100644383","phase-1-a-study-to-learn-more-about-the-safety-of-a-single-dose-of-biib144-and-how-it-is-processed-in-the-body-of-healthy-adult-participants-ages-18-to-55-years-old-as-compared-to-a-placebo-100644383","NCT07662148","A Study to Learn More About the Safety of a Single Dose of BIIB144 and How it is Processed in the Body of Healthy Adult Participants Ages 18 to 55 Years Old as Compared to a Placebo","A Phase 1, Randomized, Blinded, Placebo-Controlled, Single Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB144 in Healthy Adult Participants","Key Inclusion Criteria:\n\n* Have a body mass index (BMI) between 18 and 32 kilograms per square metre (kg\u002Fm\\^2), inclusive, at Screening.\n* Must be in good health as determined by the Investigator, based on medical history and Screening evaluations.\n\nKey Exclusion Criteria:\n\n* History of any clinically significant cardiac, endocrine, gastrointestinal, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, or renal disease, or other major disease, as determined by the Investigator.\n* History of severe allergic or anaphylactic reactions, or of any allergic reactions that in the opinion of the Investigator are likely to be exacerbated by any component of the study treatment.\n* History of, or ongoing, malignant disease, including solid tumors and hematologic malignancies (with the exception of basal cell carcinomas and squamous cell carcinomas that have been completely excised and considered cured at least 1 year prior to Day -1).\n* History of, or positive test result at Screening for, human immunodeficiency virus (HIV).\n* Any live or attenuated immunization or vaccination given within 14 days prior to Screening, between Screening and Day -1, or planned to be given during the study period.\n\nNote: Other protocol-defined Inclusion\u002FExclusion criteria may apply.",true,"55 Years",{"count":80,"type":21},48,[82],"PHASE1","In this study, researchers will learn how the body processes a study drug called BIIB144. This is a \"first-in-human\" study. This means that this study drug will be given to people for the first very time in this study. These studies are important because they help researchers learn about the safety of the study drug, how the body processes it, and what dose might be appropriate before testing it in larger groups.\n\nThe main goal of this study is to learn more about the safety of a single dose of BIIB144 compared to a placebo. A placebo is something that looks like the study drug but does not contain any medicine. A placebo is also given in the same way as the study drug.\n\nThe main question researchers want to answer in this study is:\n\nHow many participants have adverse events (AEs) and serious adverse events (SAEs)? An AE is a health problem that may or may not be caused by the study drug.\n\nResearchers will also learn more about:\n\n* How the body processes BIIB144 compared to placebo.\n\nThis study will be done as follows:\n\n* Participants will be screened to check if they can join the study. The screening period will be up to 28 days, after which eligible participants will check into their study research center.\n* Participants will stay at their study research center for about 8 days. Afterwards, they will have up to 13 visits to their study research center.\n* This will be a \"dose escalation\" study. In this kind of study, increasing amounts of study drug are given to different groups of participants. Usually, this is done until researchers find the highest dose that does not cause harmful effects. In this study, the number of dose groups and maximum dose are planned in advance. The dose escalation will stop once these planned groups are done or if safety concerns arise.\n* There will be a total of 6 groups. In each group, participants will receive a single dose of either BIIB144 or the placebo through an intravenous (IV) needle.\n* Throughout the study, participants will give blood and urine samples. Participants will also have physical exams and answer questions about how they are feeling.\n* Each participant will be in the study for up to 44 weeks.",[85],"Healthy Volunteer","NOT_YET_RECRUITING","2026-06-18",{"date":89,"type":34},"2026-06-23",{"date":91,"type":21},"2026-07-07",{"date":93,"type":21},"2028-06-19",{"name":40,"class":41},{"id":96,"slug":97,"hasResults":11,"nctId":98,"briefTitle":99,"officialTitle":100,"acronym":101,"eligibilityCriteria":102,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":103,"enrollmentInfo":104,"targetDuration":4,"studyType":22,"phases":106,"briefSummary":107,"conditions":108,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":110,"lastUpdatePostDateStruct":111,"startDateStruct":112,"completionDateStruct":114,"leadSponsor":116,"locationsCount":117},"100590083","phase-3-a-study-to-learn-more-about-the-effects-and-safety-of-felzartamab-infusions-in-adults-with-primary-membranous-nephropathy-pmn-100590083","NCT06962800","A Study to Learn More About the Effects and Safety of Felzartamab Infusions in Adults With Primary Membranous Nephropathy (PMN)","An Open-Label, Multicenter, Randomized Phase 3 Study Evaluating the Efficacy and Safety of Felzartamab in Participants With Primary Membranous Nephropathy (PMN) [PROMINENT]","PROMINENT","Key Inclusion Criteria:\n\n* Diagnosed with PMN in need of IST according to the Investigator's clinical judgment. The diagnosis of PMN must be documented with the presence of nephrotic syndrome, and hypoalbuminemia, and confirmed with a kidney biopsy either during Screening or within 5 years of signing the informed consent form (ICF) \\[see kidney biopsy exception below for participants positive for anti-PLA2R antibodies\\]. For these participants, the biopsy report with redacted protected health information must be available to be reviewed by the Sponsor or an independent nephropathologist. If the participant requires a kidney biopsy during Screening, medical monitor approval must be obtained and all other eligibility criteria should be reviewed to ensure that the participant is otherwise eligible prior to performing the kidney biopsy.\n\n  a. Kidney biopsy exception for anti-PLA2R antibody positive participants: Participants who are positive for anti-PLA2R antibodies and have not had a kidney biopsy performed within 5 years of signing the ICF, may be eligible for the study without undergoing a kidney biopsy based on medical monitor review confirming normal estimated glomerular filtration rate (eGFR), presence of nephrotic syndrome, hypoalbuminemia, positive anti-PLA2R antibody test (defined as an anti-PLA2R antibody titer \\> 20 RU\u002FmL), and documentation provided by the Investigator that the work-up for secondary causes of membranous nephropathy (MN) was negative with no identifiable secondary causes.\n* Meets one of the following:\n\n  1. Newly diagnosed PMN, defined as having never received IST for PMN in the past.\n  2. Relapsed PMN, defined as documented achievement of CR or partial remission (PR) after treatment with an IST for PMN followed by reappearance of nephrotic range proteinuria (urine protein to creatinine ratio \\[UPCR\\] ≥ 3.0 gram per gram \\[g\u002Fg\\] from a 24-hour urine collection or proteinuria ≥ 3.5 gram per 24 hour \\[g\u002F24 h\\]).\n* Participants must be on the maximally approved dose or maximally tolerated dose of angiotensin-converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB) for at least 3 months prior to Screening. Participants not on the maximally approved dose of renin-angiotensin-aldosterone system (RAAS) inhibition may be enrolled provided there is documented intolerance to maximal RAAS inhibition (e.g., angioedema, development of postural hypotension, lightheadedness, hyperkalemia, etc).\n* A UPCR of ≥ 3.0 g\u002Fg (as determined by a 24-hour urine collection) or total proteinuria ≥ 3.5 g\u002F24 h (as determined by a 24-hour urine collection) at Screening after best supportive care for at least 3 months prior to signing the ICF.\n\nKey Exclusion Criteria:\n\n* Secondary cause of MN (e.g., malignancies, medications, systemic lupus erythematosus \\[SLE\\], hepatitis B, hepatitis C, etc).\n* Severe renal impairment defined as an eGFR ≤ 30 mL\u002Fmin\u002F1.73m\\^2 at Screening or including the need for dialysis or renal replacement therapy.\n\nNote: Other protocol-defined Inclusion\u002FExclusion criteria may apply.","80 Years",{"count":105,"type":21},180,[24],"In this study, researchers will learn more about the use of felzartamab in participants with primary membranous nephropathy, also known as PMN. In people with PMN, autoantibodies build up in the glomeruli of the kidney. Antibodies are proteins that help the body fight off infection. An autoantibody is a type of antibody that mistakenly targets and attacks the body's own tissues. Glomeruli are the filters of the kidney that remove waste and extra fluid from the body. In PMN, the build-up of autoantibodies in the glomeruli causes damage to the kidneys.\n\nKidney damage can lead to too much protein and blood leaking into the urine. High levels of protein in the urine, called proteinuria, are common in people with PMN. Symptoms of PMN can include swelling in the legs and body, tiredness, and high blood pressure. If left untreated, PMN can eventually lead to kidney failure.\n\nIn this study, researchers will learn more about how a study drug called felzartamab affects people with PMN. Felzartamab is a monoclonal antibody, which means it is an antibody made in a laboratory. Felzartamab can target immune cells that produce autoantibodies, helping to lower their buildup in the kidneys. The main goal of this study is to compare how felzartamab works compared to a drug called tacrolimus. Tacrolimus is another drug given to people with PMN and kidney disease.\n\nThe main question that researchers want to answer is:\n\n* How many participants achieve a complete response after 104 weeks of treatment?\n* A complete response means that their urine protein levels decrease to a low level and their kidney function remains stable.\n\nResearchers will also learn about:\n\n* How long it takes before the participants' disease gets worse\n* How long the participants' urine protein levels stay low\n* How many participants develop antibodies against felzartamab in the blood?\n* How many participants achieve a complete response after 76 weeks of treatment\n* How many participants have medical problems during the study\n* How felzartamab is processed by the body\n* How felzartamab affects participants' tiredness and overall physical health\n\nThe study will be done as follows:\n\n* Participants will be screened to check if they can join the study. This may take up to 42 days.\n* Participants will be randomized to receive either felzartamab as intravenous (IV) infusions or tacrolimus, taken orally as tablets.\n* If participants have worsening kidney function or worsening proteinuria, or if their PMN relapses, or if they show no signs of improvement in their PMN, they will have a chance to receive rescue treatment.\n* If a participant stops treatment early, there will be follow-up visits every 12 weeks until they reach Week 104.\n* In total, participants will have up to 23 study visits. Participants who do not need rescue treatment will stay in the study for up to 104 weeks. Participants who need rescue treatment will stay in the study for up to 156 weeks.",[109],"Primary Membranous Nephropathy","2026-06-17",{"date":87,"type":34},{"date":113,"type":34},"2025-05-22",{"date":115,"type":21},"2029-03-29",{"name":40,"class":41},103,{"id":119,"slug":120,"hasResults":11,"nctId":121,"briefTitle":122,"officialTitle":123,"acronym":124,"eligibilityCriteria":125,"healthyVolunteers":11,"sex":17,"minAge":126,"maxAge":127,"enrollmentInfo":128,"targetDuration":4,"studyType":22,"phases":130,"briefSummary":131,"conditions":132,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":134,"lastUpdatePostDateStruct":135,"startDateStruct":137,"completionDateStruct":139,"leadSponsor":141,"locationsCount":142},"100589374","phase-3-a-study-to-learn-more-about-the-effects-and-long-term-safety-of-omaveloxolone-biib141-in-children-and-teens-with-friedreichs-ataxia-100589374","NCT06953583","A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia","A Phase 3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to \u003C 16 Years","BRAVE","Part 1: Key inclusion criteria:\n\n* Diagnosed with genetically confirmed Friedreich's Ataxia (FA), i.e., homozygous for guanine-adenine-adenine (GAA) repeat expansion in intron-1 of the frataxin gene, or GAA repeat expansion in 1 allele and with point mutations or deletions, or other non-GAA expansion mutations in the other allele.\n* Symptomatic for FA as confirmed by clinician assessment. a. Children 7 to \\\u003C 16 years must also have an upright stability score (USS) score of 10 to ≤ 34 at baseline\n\nPart 1: Key exclusion criteria:\n\n* Glycosylated hemoglobin A1C (HbA1c) \\> 11%\n* B-type natriuretic peptide (BNP) \\> 200 picograms per milliliter (pg\u002FmL) at screening\n* Ejection fraction (EF) \\\u003C 40% \\[based on echocardiogram (ECHO) performed at screening visit\\]\n* Clinically significant cardiac disease except mild to moderate cardiomyopathy\n\nPart 2A: Eligibility criteria:\n\n* They have completed Part 1 of the study and no discontinuation criteria have been met.\n* Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the investigator.\n\nPart 2B: Eligibility criteria:\n\n* Participants have completed Part 1 of the study and no discontinuation criteria have been met.\n* Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the Investigator.\n\nNote: Other protocol-defined Inclusion\u002FExclusion criteria may apply.","2 Years","15 Years",{"count":129,"type":21},255,[24],"In this study, researchers will learn more about omaveloxolone, also known as BIIB141 or SKYCLARYS®. Omaveloxolone is already approved for people with Friedreich's Ataxia (FA) who are 16 years of age or older. However, it is not yet available for younger teens and children. The main goal of this study is to learn how omaveloxolone affects symptoms of FA and its safety in younger participants between the ages of 2 and 15 years old.\n\nThe main questions researchers want to answer in this study are:\n\n* How does omaveloxolone affect the participants' FA symptoms?\n* How many participants have adverse events during the study?\n* Are there any changes in the participants' overall health or heart health? Adverse events are health problems that may or may not be caused by the study drug.\n\nResearchers will use the modified Friedreich's Ataxia Rating Scale (mFARS) to test nerve function. The mFARS tests movement ability, balance, coordination, speech, and arm and leg functions.\n\nThey will also use a number of questionnaires to learn more about participants' quality of life, muscle strength, and ability to perform daily tasks. Researchers will also note any changes as participants go through puberty.\n\nFinally, researchers will learn more about how the body processes omaveloxolone in children and teenagers.\n\nThis study will be done in 2 parts as follows:\n\n* Participants will be screened for up to 4 weeks to check if they can join the study.\n* In Part 1, participants will be randomly assigned to take either omaveloxolone or a placebo by mouth once a day for about 1 year. A placebo looks like the study drug but contains no real medicine.\n* Part 1 will be double blind. This means that the participants, study doctor, and site staff will not know if the participants are receiving omaveloxolone or a placebo.\n* Including screening, participants will have up to 9 clinic visits and 1 phone call during Part 1. If a participant does not join Part 2, they will have another safety follow-up phone call a month after their last dose of omaveloxolone.\n* Participants who complete Part 1 will move onto Part 2 where everyone will receive omaveloxolone for about 2 years.\n* During Part 2, participants will have up to 8 clinic visits and 1 phone call. Participants will also have a follow-up phone call about a month after they stop taking omaveloxolone.\n* In total, participants will have up to 17 clinic visits and 3 phone calls. Each participant will be in the study for up to 3 years.",[133],"Friedreich Ataxia","2026-06-15",{"date":136,"type":34},"2026-06-16",{"date":138,"type":34},"2025-06-09",{"date":140,"type":21},"2029-11-22",{"name":40,"class":41},34,{"id":144,"slug":145,"hasResults":11,"nctId":146,"briefTitle":147,"officialTitle":148,"acronym":149,"eligibilityCriteria":150,"healthyVolunteers":11,"sex":17,"minAge":151,"maxAge":152,"enrollmentInfo":153,"targetDuration":4,"studyType":22,"phases":155,"briefSummary":156,"conditions":157,"keywords":4,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":159,"lastUpdatePostDateStruct":160,"startDateStruct":161,"completionDateStruct":163,"leadSponsor":165,"locationsCount":4},"100627116","phase-3-a-study-to-learn-about-the-safety-and-effects-of-salanersen-biib115-when-given-to-babies-with-spinal-muscular-atrophy-sma-who-were-previously-treated-with-onasemnogene-abeparvovec-100627116","NCT07444450","A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec","A Phase 3, Randomized, Double-Blinded Study to Evaluate the Safety and Efficacy of Salanersen (BIIB115) After Onasemnogene Abeparvovec Treatment in Infants With Genetically Diagnosed Spinal Muscular Atrophy","STELLAR-2","Key Inclusion Criteria:\n\n* Genetic documentation of 5q spinal muscular atrophy (SMA) homozygous gene deletion or mutation or compound heterozygous mutation.\n* 2 copies of the survival motor neuron 2 (SMN2) gene.\n* Onasemnogene Abeparvovec (OA) dose given at ≤ 42 days of age and screening initiated less than 6 months from OA dosing.\n* OA dose given while participant was presymptomatic, per Investigator attestation. For this study, presymptomatic is defined as follows:\n\n  * No clinical signs or symptoms at the time of OA dosing that are, in the opinion of the Investigator, strongly suggestive of SMA.\n  * No absence of tendon reflexes (i.e., absence of all of biceps, knee and ankle tendons) at the time of OA dosing (e.g., Hammersmith Infant Neurological Examination (HINE) Section 1 or equivalent).\n  * If Compound Muscle Action Potential (CMAP) data is available at the time of dosing, ulnar CMAP amplitude ≥ 2 millivolt (mV).\n\nKey Exclusion Criteria:\n\n* Any unresolved post-OA laboratory abnormalities defined as follows:\n\n  * Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) must be less than 2 × Upper Limit of Normal (ULN) while not receiving corticosteroids within 30 days prior to dosing with salanersen or sham procedure (repeat testing may be performed if necessary).\n  * Evidence of thrombocytopenia, indicated by the platelet count being lower than the normal range for the laboratory.\n  * Evidence of elevated troponin-I levels, identified as elevated post-OA, and has not returned to the normal range.\n* Confirmed demonstration of corrected QT interval, using Fridericia's correction method, of \\> 450 milliseconds (ms).\n* Other than OA, any prior treatment with an approved SMA disease modifying therapy (e.g. nusinersen and\u002For risdiplam), a myostatin inhibitor therapy, or an investigational drug given for the treatment of SMA.\n* Steroid treatment administered for the purpose of treating complications following OA within 14 days prior to dosing with salanersen or sham procedure on Day 1.\n\nNote: Other protocol-defined inclusion\u002Fexclusion criteria will apply.","0 Days","7 Months",{"count":154,"type":21},42,[24],"In this study, researchers will learn more about the safety and effects of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have already been treated with onasemnogene abeparvovec (OA) after being diagnosed with SMA.\n\nMost people with SMA have changes in a gene called survival motor neuron 1, also known as SMN1. These changes lower the amount of SMN protein in their bodies. Without enough of this protein, motor neurons and muscles cannot work properly. A similar gene called SMN2 can help replace some of the lost SMN protein in the body. Salanersen works by helping the SMN2 gene to make more SMN protein.\n\nOA works by replacing the missing or abnormal SMN1 gene. Sometimes, OA treatment may not work as well as expected. As a result, researchers are exploring whether giving another drug after OA could lead to better outcomes for people with SMA.\n\nIn this study, participants will have 2 SMN2 copies. The higher the copy number, the less severe the participant's SMA is. They will also have received treatment with OA by the time they were 42 days old and before showing any symptoms of SMA.\n\nThe main goal of the study is to learn more about the safety of giving salanersen to babies after OA treatment. Researchers will also learn more about whether salanersen can help make SMA symptoms less serious.\n\nThe main question researchers want to answer in this study is:\n\n• How many participants have adverse events and serious adverse events after treatment?\n\nResearchers will also learn more about:\n\n* The effects on participants' motor symptoms and how many new movement milestones participants achieve.\n* How many participants stay free of SMA symptoms.\n* How much neurofilament protein is found in the blood after treatment.\n* How much salanersen gets into the fluid surrounding the brain and spinal cord.\n* How much salanersen gets into the blood. Researchers will use different tests to learn if motor symptoms are changing, including the World Health Organization (WHO) motor milestones and Hammersmith Infant Neurological Examination (HINE) Section 2 motor milestones.\n\nThe study will be done in 2 parts. Part A will last 1 year while Part B will last up to 4 years.\n\nThe study will be done as follows:\n\n* First, participants will be screened to check if they can join the study. The screening period will be up to 6 months. Participants must have received OA treatment before the age of 42 days and started screening within 6 months of the OA dose.\n* Participants will be assigned to 1 of 2 treatment groups by chance. This is a \"double blind\" study which means neither the participants, study doctor, nor site staff will know which treatment group the participants are assigned to.\n* In this study, salanersen will be given as an intrathecal injection, which is an injection into the fluid surrounding the spine. This is done by a procedure called a lumbar puncture (LP) which involves inserting a needle into the lower back into the space around the spinal cord.\n* During Part A, one group will receive 80 milligrams (mg) of salanersen while another group receives a sham (fake) procedure. This means that a small needle prick will be done, but no injection will be given.\n* For each participant, the first visit of Part A will be 6 months after they receive OA treatment.\n* Part A will have up to 6 clinic visits and 2 phone calls and last up to 1 year.\n* During Part B, both groups of participants will receive 80 mg of salanersen once a year.\n* Part B will have up to 12 clinic visits and 14 phone calls and last up to 4 years.\n* In total, participants will be in the study for up to 5 and a half years.",[158],"Muscular Atrophy, Spinal","2026-06-11",{"date":134,"type":34},{"date":162,"type":21},"2026-09-04",{"date":164,"type":21},"2033-07-26",{"name":40,"class":41},{"id":167,"slug":168,"hasResults":11,"nctId":169,"briefTitle":170,"officialTitle":171,"acronym":172,"eligibilityCriteria":173,"healthyVolunteers":11,"sex":17,"minAge":151,"maxAge":174,"enrollmentInfo":175,"targetDuration":4,"studyType":22,"phases":177,"briefSummary":178,"conditions":179,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":159,"lastUpdatePostDateStruct":180,"startDateStruct":181,"completionDateStruct":183,"leadSponsor":185,"locationsCount":186},"100609983","phase-3-a-study-to-learn-about-salanersens-biib115-effects-on-movement-and-its-safety-when-given-before-symptoms-appear-in-babies-with-genetically-diagnosed-spinal-muscular-atrophy-sma-100609983","NCT07221669","A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)","An Open-Label Study to Assess the Efficacy and Safety of Multiple Doses of Salanersen (BIIB115) Delivered Intrathecally to Treatment-Naïve, Presymptomatic Infants With Genetically Diagnosed Spinal Muscular Atrophy","STELLAR-1","Key Inclusion Criteria:\n\n-≤42 days of age at first dose of salanersen.\n\n* Genetic documentation of 5q SMA homozygous gene deletion or mutation or compound heterozygous mutation.\n* Two or three copies of the survival motor neuron 2 (SMN2) gene.\n* Ulnar compound muscle action potential (CMAP) amplitude ≥2 millivolt (mV) at Screening and Day 1 predose.\n* Body weight ≥3rd percentile for age based on World Health Organization (WHO) Child Growth Standards at the time of informed consent.\n\nKey Exclusion Criteria:\n\n* Any clinical signs or symptoms at Screening or Day 1 predose that are, in the opinion of the Investigator, strongly suggestive of SMA.\n* Areflexia on neurologic examination at biceps, knee, or ankle at Screening or Day 1 Predose.\n* Hypoxemia (oxygen saturation \\\u003C96% awake or asleep without any supplemental oxygen or respiratory support, or for altitudes \\>1000 meters (m), oxygen saturation of \\\u003C92% awake or asleep without any supplemental oxygen or respiratory support).\n* Diagnosis of neonatal respiratory distress syndrome necessitating surfactant replacement therapy or invasive ventilatory support.\n* Any reason, anatomical or otherwise (including hematology\u002Fcoagulation laboratory results), that presents increased risk of complication from the LP procedures or safety assessments.\n* Any prior treatment with an approved SMA disease-modifying therapy (e.g., nusinersen, onasemnogene abeparvovec-xioi \\[OA\\], and\u002For risdiplam), a myostatin inhibitor therapy, or an investigational drug given for the treatment of SMA.\n\nNote: Other protocol-defined inclusion\u002Fexclusion criteria will apply.","42 Days",{"count":176,"type":21},30,[24],"In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen.\n\nSpecifically, researchers will learn more about how salanersen works in babies who have been diagnosed with SMA through genetic testing but have not yet started showing signs or symptoms. Most people with SMA have changes in a gene called survival motor neuron 1, also known as SMN1. These changes lower the amount of SMN protein in their bodies. Without enough of this protein, motor neurons and muscles cannot work properly. A similar gene called SMN2 can help replace some of the lost SMN protein in the body. Salanersen works by helping the SMN2 gene to make more SMN protein.\n\nIn this study, participants will have either 2 SMN2 copies or 3 SMN2 copies. The higher the copy number, the less severe the participant's SMA is.\n\nThe main goal of this study is to see if starting salanersen before signs or symptoms appear can prevent signs or symptoms of SMA or make them less severe. Researchers will use different tests to learn if motor symptoms are changing, including the World Health Organization (WHO) motor milestones.\n\nThe main questions researchers want to answer in this study are:\n\n* How many participants with 2 copies of the SMN2 gene can sit without support at 12 months?\n* How many participants with 3 copies of the SMN2 gene can walk alone at 18 months?\n\nResearchers will also learn more about:\n\n* The effects on participants' motor symptoms and how many new movement milestones participants achieve.\n* How many participants stay free of SMA symptoms\n* How much salanersen gets into the fluid surrounding the brain and spinal cord.\n* How much salanersen gets into the blood.\n* How many participants have adverse events or serious adverse events. Adverse events are health problems that may or may not be caused by the study drug.\n\nThis study will be done as follows:\n\n* First, participants will be screened to check if they can join the study. The screening period will be up to 28 days.\n* This is an \"open label\" study. This is a study in which the participants, study doctor, and site staff know which study drug participants are receiving. In this study, all participants will receive salanersen through an intrathecal injection, or one that is given into the fluid surrounding the brain and spinal cord.\n* There will be 2 parts in this study. During Part 1, participants will receive 2 doses of salanersen, about 12 months apart from each other. Part 1 will last up to 25 months.\n* During Part 2, participants will continue to receive salanersen. They will receive up to 3 doses, 12 months apart from each other. Part 2 will last up to 36 months.\n* During Part 1, participants will have up to 11 clinic visits and up to 3 phone calls. During Part 2, participants will have up to 7 clinic visits and up to 12 phone calls.",[158],{"date":134,"type":34},{"date":182,"type":34},"2026-04-28",{"date":184,"type":21},"2032-05-29",{"name":40,"class":41},4,{"id":188,"slug":189,"hasResults":11,"nctId":190,"briefTitle":191,"officialTitle":192,"acronym":4,"eligibilityCriteria":193,"healthyVolunteers":77,"sex":17,"minAge":18,"maxAge":78,"enrollmentInfo":194,"targetDuration":4,"studyType":22,"phases":196,"briefSummary":197,"conditions":198,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":199,"lastUpdatePostDateStruct":200,"startDateStruct":202,"completionDateStruct":204,"leadSponsor":206,"locationsCount":207},"100603231","phase-1-a-study-to-learn-about-the-safety-of-biib142-and-how-it-is-processed-in-the-body-of-healthy-adult-participants-aged-18-to-55-years-old-100603231","NCT07133828","A Study to Learn About the Safety of BIIB142 and How it is Processed in the Body of Healthy Adult Participants Aged 18 to 55 Years Old","A Phase 1, Randomized, Blinded, Placebo-Controlled, Single- and Multiple-Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB142 in Healthy Adults","Key Inclusion Criteria:\n\n* Have a body mass index between 18 and 32 kilograms per square meter (kg\u002Fm\\^2), inclusive, at screening.\n* Weight ≥ 50 kilograms (kg) at screening.\n* Negative polymerase chain reaction (PCR) test for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at Check-in prior to randomization.\n* Must be in good health as determined by the Investigator.\n\nKey Exclusion Criteria:\n\n* History of any clinically significant cardiac, endocrine, gastrointestinal, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, or renal disease, or other major disease, as determined by the Investigator.\n* History of severe allergic or anaphylactic reactions\n* History of or ongoing malignant disease (with limited exceptions)\n* Systolic blood pressure \\>150 millimeters of mercury (mmHg) or \\\u003C90 mmHg.\n* Clinically significant (as determined by the Investigator) electrocardiogram (ECG) abnormalities.\n* History of or positive test for human immunodeficiency virus (HIV).\n* Chronic, recurrent, or serious infection within 90 days prior to Screening.\n* Symptoms of bacterial, fungal, or viral infection within 14 days prior to Screening.\n* Any live or attenuated immunization within 14 days prior to Screening.\n* Use of prescription medications, over-the-counter medications that alter hepatic or renal clearance, or nutraceuticals within 28 days prior to Check-in.\n* MAD Cohorts only: Suicidal ideation with some intent to act within 6 months prior to the start of Screening or history of suicidal behavior within one year prior to Screening.\n\nNOTE: Other protocol defined Inclusion\u002FExclusion criteria may apply.",{"count":195,"type":21},78,[82],"In this study, researchers will learn more about the safety of BIIB142 and how it is processed in the body. This is the first time that researchers will learn about BIIB142 and how it affects people.\n\nThe main question researchers want to answer in this study is:\n\n• How many participants have adverse events (AEs) and serious adverse events (SAEs)?\n\nAn AE is a health problem that may or may not be caused by a drug during the study. An AE is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care.\n\nResearchers will also learn more about:\n\n• How the body processes BIIB142\n\nThis is a \"dose escalation study.\" This is a study in which increasing amounts of the study drug are given to different groups of participants. This is done until researchers find the highest dose that does not cause harmful effects.\n\nFirst, participants will be screened to check if they can join the study. The screening period will be up to 28 days. This study will be split into 2 parts - Part A and Part B.\n\nDuring Part A:\n\n* Participants will be randomly placed into 1 of 6 groups to receive a single dose of either BIIB142 or a placebo. A placebo looks like the study drug but contains no real medicine.\n* Participants in Groups 1 through 5 will take either BIIB142 or the placebo without food. Participants in Group 6 will take 2 doses of their assigned treatment - once with food and once without food.\n* Neither the researchers nor the participants will know if the participants will receive BIIB142 or the placebo.\n* Participants will stay at their study research center for 5 days. They will return for another 4 visits. Each participant in Part A will be in the study for up to 58 days.\n\nDuring Part B:\n\n* Participants will be randomly placed into 1 of 3 groups to receive BIIB142 or the placebo. In Part B, participants will take BIIB142 or the placebo once a day for 14 days.\n* Neither the researchers nor the participants will know if the participants will receive BIIB142 or the placebo.\n* Participants will stay at their study research center for 16 days. They will return for another 4 visits. Each participant in Part B will be in the study for up to 58 days.",[85],"2026-04-30",{"date":201,"type":34},"2026-05-06",{"date":203,"type":34},"2025-08-29",{"date":205,"type":21},"2026-08-06",{"name":40,"class":41},1,{"id":209,"slug":210,"hasResults":11,"nctId":211,"briefTitle":212,"officialTitle":213,"acronym":4,"eligibilityCriteria":214,"healthyVolunteers":11,"sex":17,"minAge":215,"maxAge":4,"enrollmentInfo":216,"targetDuration":218,"studyType":219,"phases":4,"briefSummary":220,"conditions":221,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":222,"lastUpdatePostDateStruct":223,"startDateStruct":224,"completionDateStruct":226,"leadSponsor":228,"locationsCount":229},"100564032","a-study-to-learn-more-about-the-long-term-safety-of-biib141-omaveloxolone-in-participants-with-friedreichs-ataxia-who-are-prescribed-it-by-their-own-doctors-100564032","NCT06623890","A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors","An Observational, Multinational, Post-Marketing Registry of Omaveloxolone-Treated Patients With Friedreich's Ataxia","Key Inclusion Criteria:\n\n* Documented diagnosis of FA, including confirmation via genetic testing.\n* Participants aged 16 years and older at initiation of omaveloxolone treatment.\n\nFor the omaveloxolone-naive cohort\n\n\\- Initiating omaveloxolone treatment as per an approved label concurrent with enrolling in this study.\n\nFor the omaveloxolone-non-naive cohort\n\n* Initiated omaveloxolone treatment as per an approved label less than 12 months prior to enrollment in this study\n* Prior to enrollment, maintained omaveloxolone treatment with no discontinuation of more than 60 days\n* Actively on treatment at the time of enrollment in this study\n* Treating physician is the study site principal investigator or sub-investigator\n* Study site confirms ability to provide required baseline data through medical record review, UNIFAI database, or other site-collected data\n* Enrolled in the UNIFAI study prior to initiation of omaveloxolone treatment\n\nKey Exclusion Criteria:\n\n* Received off-label prescription of omaveloxolone at any time.\n* Previously enrolled in a clinical trial of omaveloxolone.\n* Participating in a blinded interventional trial at the time of enrollment in the study; participants may participate in other clinical trials after baseline data are collected.\n\nNote: Other protocol-defined Inclusion\u002FExclusion criteria may apply.","16 Years",{"count":217,"type":21},300,"5 Years","OBSERVATIONAL","In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This is a drug available for doctors to prescribe for people with Friedreich's Ataxia, also known as FA. This is known as an \"observational\" study, which collects health information about study participants without changing their medical care. Participants for this study will be found using a group called the Friedreich's Ataxia Global Clinical Consortium (FA GCC) UNIFIED Natural History Study (UNIFAI). The FA-GCC is a group of study research centers that helps provide clinical care for FA patients and also helps researchers learn more about how FA affects patients over a long time.\n\nThe main objective of this study is to collect safety information in participants with FA from UNIFAI. Some of the participants in this study will be prescribed BIIB141 for the first time by their own doctors. Some of the participants will have started taking BIIB141 after joining UNIFAI, but less than 12 months before joining this study.\n\nThe main questions researchers want to answer in this study are:\n\n* How many participants had serious adverse events (SAEs)? An adverse event is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care.\n* How many participants had adverse events (AEs) related to heart failure or liver damage caused by the drug?\n\nResearchers will also learn more about :\n\n• Why and when participants stopped treatment, left the study, or took more of the drug than was prescribed\n\nThis study will be done as follows:\n\n* Participants will be screened to check if they can join the study.\n* After joining the study, the participants who had never started BIIB141 treatment before must start it within 6 months. Otherwise, all participants will take BIIB141 throughout this study as prescribed by their own doctor.\n* During the study, each participant's doctor will decide how often the participant visits the study research center to check on their health. This will be based on the doctor's own clinical judgment and what is recommended by the drug's label.\n* Data from the participants' regular visits to their doctor will be collected at 1 month, 2 months, 3 months, 6 months, 12 months, 24 months, 36 months, 48 months, and 60 months.\n* Each participant will be in the study for up to 5 years.",[133],"2026-04-23",{"date":182,"type":34},{"date":225,"type":34},"2024-12-12",{"date":227,"type":21},"2029-10-01",{"name":40,"class":41},14,{"id":231,"slug":232,"hasResults":11,"nctId":233,"briefTitle":234,"officialTitle":235,"acronym":236,"eligibilityCriteria":237,"healthyVolunteers":11,"sex":17,"minAge":127,"maxAge":238,"enrollmentInfo":239,"targetDuration":4,"studyType":22,"phases":241,"briefSummary":242,"conditions":243,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":245,"lastUpdatePostDateStruct":246,"startDateStruct":248,"completionDateStruct":250,"leadSponsor":252,"locationsCount":207},"100627118","phase-3-a-study-to-learn-about-salanersens-biib115-effects-on-movement-and-its-safety-in-participants-aged-15-to-60-years-with-spinal-muscular-atrophy-sma-who-are-either-new-to-sma-treatment-or-were-previously-treated-with-risdiplam-100627118","NCT07444476","A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam","An Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of Salanersen (BIIB115) in Participants Aged 15-60 Years With Spinal Muscular Atrophy Who Are Either Treatment-Naïve or Have Previously Been Treated With Risdiplam","SOLAR","Key Inclusion Criteria:\n\n* Participants aged 15 to 60 years, inclusive, at the time of informed consent\n* Participants with genetic documentation of 5q Spinal Muscular Atrophy (SMA) (homozygous gene deletion or mutation or compound heterozygous mutation).\n* Participants with clinical signs and symptoms consistent with SMA.\n* Survival motor neuron 2 (SMN2) copy number ≥ 1.\n* Participants with baseline Hammersmith Functional Motor Scale - Expanded (HFMSE) total score of ≥ 10 to ≤ 54.\n* Participants who are able to sit without using support for at least 10 seconds.\n* Participants with no prior treatment with myostatin inhibitors and a willingness to remain off concurrent myostatin inhibitor therapy for the duration of the study.\n* Ambulatory and nonambulatory participants:\n\n  * Ambulatory participants must be able to walk at least 10 meters independently without assistance and are willing and able to complete the 6 Minute Walk Test (6MWT) at Screening.\n* For participants in the treatment-naïve cohort:\n\n  * No prior treatment with an approved SMA Disease Modifying Therapy (DMT) or an investigational drug given for the treatment of SMA.\n* For participants in the risdiplam-treated cohort:\n\n  * Currently receiving risdiplam treatment and have been on once-daily 5 milligrams (mg) risdiplam treatment for at least 6 months prior to Screening.\n  * Willing to stop risdiplam therapy for the duration of the study. The last dose of risdiplam must be taken the day before the first dose of salanersen.\n  * No prior treatment with nusinersen, onasemnogene abeparvovec-xioi\u002Fonasemnogene abeparvovec-brve (OA), other approved DMTs for SMA or investigational drugs given for the treatment of SMA apart from risdiplam.\n\nKey Exclusion Criteria:\n\n* Respiratory insufficiency at Screening, defined by the medical necessity for invasive or noninvasive ventilation for \\> 6 hours during a 24-hour period (except for nocturnal bilevel positive airway pressure).\n* Medical necessity for a gastric feeding tube, where the majority of nutrition is provided by this route, as assessed by the site Investigator at Screening.\n* History of brain or spinal cord disease or other contraindications (e.g., severe scoliosis) that would interfere with the lumbar puncture (LP) procedures, Cerebrospinal fluid (CSF) circulation, efficacy assessments, or safety assessments (including a history of hydrocephalus or implanted shunt for CSF drainage), as assessed by the Investigator.\n* Hospitalization for surgery, a pulmonary event, or nutritional support within 2 months prior to Screening or plans to undergo elective procedures or surgeries at any time after signing the Informed Consent Form (ICF) through the end of the study. Note: If prior scoliosis surgery has been performed, it must be done at least 1 year prior to Screening.\n* Presence of an active medical issue (e.g., infection, recent fracture) that would make the participant unsuitable for inclusion, as assessed by the Investigator.\n* Current enrollment or a plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy for investigational use is administered within 90 days or 5 half-lives of the treatment (if known), whichever is longer, prior to Screening. This includes neuromodulation therapy such as spinal cord stimulation.\n\nNote: Other protocol-defined inclusion\u002Fexclusion criteria will apply.","60 Years",{"count":240,"type":21},90,[24],"In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen.\n\nSpecifically, researchers will learn more about how salanersen works in individuals with SMA who are between the ages of 15 and 60 years old. In most people living with SMA, changes to or a lack of a gene called survival motor neuron 1 (SMN1) - often referred to as gene mutations or variants - affect how this gene works. As a result, their bodies produce less SMN protein. Without enough of this protein, motor neurons and muscles cannot work properly. There is a similar gene called SMN2 that produces SMN protein, but it usually does not produce enough SMN protein on its own to make up for the changes in the SMN1 gene. Salanersen is a drug designed to help the SMN2 gene to make more working SMN protein.\n\nIn this study, there will be 2 groups of participants: a group who has never received treatment for SMA before joining this study, and a group who has been treated with risdiplam, an approved drug for SMA . Those participants must not have received any other SMA treatments before and will need to stop their risdiplam treatment for the duration of the study.\n\nThe main goal of this study is to learn more about how salanersen affects the participants' motor function. Researchers will use different tests and questionnaires to learn if motor function is changing over the study duration.\n\nThe main question researchers want to answer in this study is:\n\n• For the group who has never been treated for SMA, how much do scores on the HFMSE movement test change at 12 months compared to the beginning of the study? The Hammersmith Functional Motor Scale - Expanded (HFMSE) has 33 activities that are scored which include sitting, lying down, walking, jumping, and more.\n\nResearchers will also learn more about:\n\n* The effects on participants' motor function and how well their nerves and muscles function.\n* The effects on participants' overall sense of change and how they perform daily activities.\n* How many participants have adverse events or serious adverse events. Adverse events are health problems that may or may not be caused by the study drug.\n* How much salanersen gets into the fluid surrounding the brain and spinal cord.\n* How much salanersen gets into the blood.\n\nThis study will be done as follows:\n\n* First, participants will be screened to check if they can join the study. The screening period may be up to 4 weeks.\n* This is an \"open-label\" study. This is a study in which the participants, study doctor, and site staff will know that participants are receiving salanersen.\n* All participants will receive salanersen through an intrathecal injection, or one that is given into the fluid surrounding the brain and spinal cord.\n* Participants will receive salanersen once every year for a total of 5 times throughout the study.\n* Including screening, participants will have 17 study visits and 9 telephone calls during this study, which will last up to 61 months in total.",[244],"Spinal Muscular Atrophy","2026-04-16",{"date":247,"type":34},"2026-04-21",{"date":249,"type":21},"2026-04-03",{"date":251,"type":21},"2032-06-22",{"name":40,"class":41},{"id":254,"slug":255,"hasResults":11,"nctId":256,"briefTitle":257,"officialTitle":258,"acronym":4,"eligibilityCriteria":259,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":260,"targetDuration":262,"studyType":219,"phases":4,"briefSummary":263,"conditions":264,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":266,"lastUpdatePostDateStruct":267,"startDateStruct":269,"completionDateStruct":271,"leadSponsor":273,"locationsCount":207},"100612930","a-study-to-learn-more-about-the-long-term-safety-of-tofersen-qalsody-in-participants-with-superoxide-dismutase-1-sod-1-amyotrophic-lateral-sclerosis-als-100612930","NCT07259980","A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS)","An Observational Registry-Based Study to Evaluate the Long-Term Safety of Tofersen in People With SOD1-ALS","Key Inclusion Criteria:\n\n* Participants with an ALS diagnosis and a confirmed SOD1 mutation from contributing registry networks will be considered for inclusion in the study.\n\nKey Exclusion Criteria:\n\n* Data collected while a person with SOD1-ALS is participating in an interventional clinical trial (with tofersen or any other investigational medicinal product) will be excluded.\n\nNOTE: Other protocol- defined Inclusion\u002FExclusion criteria may apply.",{"count":261,"type":21},125,"7 Years","In this study, researchers will learn more about the safety of tofersen, also known as Qalsody®. This is a drug available for doctors to prescribe for participant with a certain type of amyotrophic lateral sclerosis, also known as ALS. This type is in participant who have a mutation in the superoxide dismutase 1 gene, also known as SOD-1.\n\nThis is known as an \"observational\" study, which collects health information about study participants without changing their medical care. Participants for this study will be found using 2 different groups of study research centers that help provide clinical care for participant with ALS. These groups are in Europe and the United States and are called:\n\n* the Precision-ALS programme\n* the ALS\u002FMotor Neuron Disease (MND) Natural History Consortium (NHC)\n\nThe main goal of this study is to collect safety information in participants with SOD-1 ALS who were in either of the groups.\n\nThe main question researchers want to answer in this study is:\n\n* What are the characteristics of the participants in this study?\n* How many participants had serious adverse events (SAEs), including ones that affect the brain, spinal cord, or nerves?\n\nAn adverse event is a health problem that may or may not be caused by a drug during the study. An adverse event is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care.\n\nResearchers will also learn more about:\n\n* How many participants develop other health conditions or become pregnant, including how the pregnancy turned out\n* Why and when participants stopped treatment\n\nThis study will be done as follows:\n\n* Participants will be screened to check if they can join the study.\n* Data from the participants' regular visits to their clinic will be collected based on which study research center they are in.\n* Each participant will be in the study until they decide to leave or until death. Currently, the study is planned to last at least 7 years.",[265],"Amyotrophic Lateral Sclerosis","2026-04-06",{"date":268,"type":34},"2026-04-09",{"date":270,"type":34},"2026-01-02",{"date":272,"type":21},"2033-12-30",{"name":40,"class":41},{"id":275,"slug":276,"hasResults":11,"nctId":277,"briefTitle":278,"officialTitle":279,"acronym":280,"eligibilityCriteria":281,"healthyVolunteers":11,"sex":17,"minAge":282,"maxAge":283,"enrollmentInfo":284,"targetDuration":4,"studyType":22,"phases":286,"briefSummary":287,"conditions":288,"keywords":4,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":290,"lastUpdatePostDateStruct":291,"startDateStruct":293,"completionDateStruct":295,"leadSponsor":297,"locationsCount":4},"100630127","phase-3-a-study-to-learn-about-the-safety-of-diroximel-fumarate-drf-and-dimethyl-fumarate-dmf-and-their-effects-on-relapses-in-pediatric-participants-with-relapsing-forms-of-multiple-sclerosis-rms-100630127","NCT07483632","A Study to Learn About the Safety of Diroximel Fumarate (DRF) and Dimethyl Fumarate (DMF) and Their Effects on Relapses in Pediatric Participants With Relapsing Forms of Multiple Sclerosis (RMS)","A Phase 3 Study With an Open-Label Extension in Pediatric Participants to Evaluate the Safety, Efficacy, and Pharmacokinetics of Diroximel Fumarate and Dimethyl Fumarate for the Treatment of Relapsing Forms of Multiple Sclerosis","VOYAGE","Key Inclusion Criteria:\n\nTreatment Period:\n\n* Must have a diagnosis of pediatric MS (as defined by the revised consensus definition of pediatric MS).\n* Must have an Expanded Disability Status Scale (EDSS) score between 0 and 5.0, inclusive.\n* Must have experienced at least 1 of the following conditions:\n\n  1. greater than or equal to 1 relapse in the 12 months prior to the Baseline Visit (Day 1),\n  2. greater than or equal to 2 relapses in the 24 months prior to the Baseline Visit (Day 1), or\n  3. evidence of gadolinium (Gd)-enhancing lesions of the brain on magnetic resonance imaging (MRI) within 6 months prior to the Baseline Visit (Day 1).\n* Participants must be neurologically stable with no evidence of relapse within 30 days prior to the Baseline Visit (Day 1).\n\nOpen-Label Extension Period:\n\n\\- Participants who completed the study treatment in Cohorts A or B through the Week 96 Visit.\n\nKey Exclusion Criteria\n\nTreatment Period:\n\n* History of progressive MS.\n* History of disorders mimicking MS, such as other demyelinating disorders (e.g., acute disseminated encephalomyelitis and neuromyelitis optical spectrum disorder), systemic autoimmune disorders (e.g., Sjögren's disease and lupus erythematosus), metabolic disorders (e.g., dystrophies), and infectious disorders.\n* History of severe allergic or anaphylactic reactions or any allergic reactions that in the opinion of the Investigator are likely to be exacerbated by any component of the study treatment.\n* History of, or ongoing, malignant disease, including solid tumors, skin malignancies, and hematologic malignancies.\n* History of gastrointestinal (GI) surgery (except appendectomy or cholecystectomy that occurred more than 6 months prior to the Screening Visit), inflammatory bowel disease (Crohn's disease, ulcerative colitis), or another clinically significant and active GI condition at the Investigator's discretion.\n* Known hypersensitivity to fumaric acid derivatives or any excipients of DRF or DMF.\n* Diagnosis of macular edema prior to randomization.\n\nOpen-Label Extension Period:\n\n* Any significant changes in medical history occurring after enrollment in the Treatment Period, including laboratory test abnormalities or current clinically significant conditions that, in the opinion of the Investigator, would have excluded the participant's participation in the Treatment Period. The Investigator must reassess the participant's medical fitness for participation and consider any factors that would preclude treatment.\n* Participants who discontinued study treatment due to tolerability issues.\n* Other unspecified reasons that, in the opinion of the Investigator or the Sponsor, make the participant unsuitable for participation in the OLE Period.\n\nNOTE: Other protocol-defined Inclusion\u002FExclusion criteria may apply.","10 Years","17 Years",{"count":285,"type":21},185,[24],"In this study, researchers will learn more about the study drugs diroximel fumarate (DRF) and dimethyl fumarate (DMF) in children with MS who may be experiencing relapses.\n\nParticipants will be divided into 2 groups based on their weight:\n\n* Group A will include children who weigh 40 kilograms (kg) or less. They will receive DRF in both Part 1 and Part 2 of the study.\n* Group B will include children who weigh more than 40 kg. They will be randomly assigned to receive DRF, DMF, or fingolimod. Fingolimod is a drug already used to treat MS in adults. In Part 2, they will receive DRF.\n\nThis is a 2-part study. Part 1 treatment will last 96 weeks. Participants who complete Part 1 may move to Part 2. Part 2 is an extension period. Treatment will last another 96 weeks and will help researchers learn about the long-term safety and effects of treatment.\n\nThe main goal of the study is to learn about the safety of DRF and DMF and compare their effect on relapses and brain lesions with fingolimod.\n\nThe main questions researchers want to answer are:\n\n* How many participants have adverse events and serious adverse events?\n* How often do participants relapse after treatment with DRF and DMF compared to fingolimod?\n\nResearchers will take brain imaging scans to check for any new areas of brain inflammation and compare the brain lesions before and after treatment.\n\nResearchers will also measure the amount of drug in the blood to understand how the body processes it. To check safety, they will monitor participants' growth and development, and compare changes in heart tests, vital signs, and lab tests. They will also use rating scales to monitor depression symptoms.\n\nThe study will be done as follows:\n\nPart 1 (Treatment Period)\n\n* After screening, participants will join Part 1 and be divided into 2 groups based on their weight.\n* Participants in Group A will receive DRF.\n* Participants in Group B will be randomly assigned to receive either DRF, DMF, or fingolimod.\n* Neither the researchers nor the participants will know which drug or dose the participants will receive in Group B. Participants in Group B will also receive a placebo so they do not know which drug is being given. A placebo looks like a study drug but contains no real medicine.\n* All study drugs will be taken by mouth, once or twice a day. Participants who take DRF or DMF will start with a lower dose during the 1st week, then move to a standard dose.\n* Treatment in Part 1 will last for 96 weeks. Participants will have up to 11 study visits and 7 phone calls.\n* Participants who do not move onto Part 2 will also have a safety follow-up period of 4 to 8 weeks. This will include 1 study visit and 1 phone call.\n* The total length of Part 1, including the screening, treatment, and follow-up, will be up to 108 weeks.\n\nPart 2 (Extension Period)\n\n* Participants who complete Part 1 can move on to Part 2 of the study.\n* All participants in Part 2 will receive DRF by mouth for 96 weeks.\n* Participants will have up to 10 more study visits and 1 telephone call during treatment.\n* They will also have a 4-week safety follow-up, including 1 study visit and 1 phone call.\n* The total length of Part 2 will be up to 100 weeks.",[289],"Relapsing Forms of Multiple Sclerosis","2026-03-17",{"date":292,"type":34},"2026-03-19",{"date":294,"type":21},"2026-11-16",{"date":296,"type":21},"2035-06-19",{"name":40,"class":41},{"id":299,"slug":300,"hasResults":11,"nctId":301,"briefTitle":302,"officialTitle":303,"acronym":304,"eligibilityCriteria":305,"healthyVolunteers":11,"sex":17,"minAge":306,"maxAge":4,"enrollmentInfo":307,"targetDuration":4,"studyType":22,"phases":309,"briefSummary":310,"conditions":311,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":312,"lastUpdatePostDateStruct":313,"startDateStruct":315,"completionDateStruct":317,"leadSponsor":319,"locationsCount":320},"100558768","phase-1-a-study-to-find-out-how-nusinersen-is-processed-in-the-body-when-given-through-the-thecaflex-drx-system-in-adult-and-pediatric-participants-with-spinal-muscular-atrophy-pierre-pk-100558768","NCT06555419","A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)","An Open Label, Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)","PIERRE-PK","Key Inclusion Criteria:\n\n* Participant is on regular maintenance dosing of nusinersen (12 milligrams \\[mg\\] dose) every 4 months, with 4 months (± 2 weeks) between the LP-delivered study dose and the last nusinersen dose prior to study enrollment.\n* Participants must be enrolled in the PIERRE study to be eligible for enrolment in the PIERRE PK study.\n\nKey Exclusion Criteria:\n\n* Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrollment in other interventional clinical trials for the treatment of SMA (except for the PIERRE study or interventional clinical trials of myostatin inhibitors \\[e.g., apitegromab and taldefgrobep alfa\\]).\n* Participant is naïve to nusinersen treatment.\n* Participant is receiving nusinersen at a dose other than 12 mg.\n* Participant has already undergone implantation of the ThecaFlex DRx system.\n* Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study.\n\nNOTE: Other protocol-defined Inclusion\u002FExclusion criteria may apply","3 Years",{"count":308,"type":21},58,[82],"In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP). The ThecaFlex DRx system is an investigational implantable medical device developed by Alcyone Therapeutics, Inc. It consists of a catheter, which is a flexible tube, connected to a port which is placed under the skin. Alcyone Therapeutics, Inc. has an ongoing study called PIERRE to test the ThecaFlex DRx system. Participants with spinal muscular atrophy (SMA) in the PIERRE study may be enrolled in the PIERRE-PK study.\n\nThe main objective of the PIERRE-PK study is to learn how the body processes nusinersen when given by the ThecaFlex DRx system compared to a lumbar puncture. The main questions researchers want to answer are:\n\n* What is the highest amount of nusinersen found in the blood after dosing?\n* How much nusinersen is found in the blood over the first 24 hours after dosing?\n\nThe PIERRE-PK study will be done as follows:\n\n* Participants will be screened to check if they can join the study. The screening period will be up to 30 days for this study and may overlap with the PIERRE study.\n* Participants will receive a dose of nusinersen by lumbar puncture.\n* The ThecaFlex DRx system will be implanted after the lumbar puncture, as part of the PIERRE study.\n* Participants will receive a dose of nusinersen by the ThecaFlex DRx system, as part of the PIERRE study.\n* Researchers will take blood samples before and after each dose. The last blood sample will be taken 24 hours after the dose.\n* The total study duration for each participant in the PIERRE-PK study will be approximately 5 months. This period will overlap with the participant's first 5 months in the PIERRE study.",[158],"2026-03-16",{"date":314,"type":34},"2026-03-18",{"date":316,"type":34},"2025-01-16",{"date":318,"type":21},"2027-06-25",{"name":40,"class":41},19,{"id":322,"slug":323,"hasResults":11,"nctId":324,"briefTitle":325,"officialTitle":326,"acronym":4,"eligibilityCriteria":327,"healthyVolunteers":11,"sex":328,"minAge":18,"maxAge":329,"enrollmentInfo":330,"targetDuration":126,"studyType":219,"phases":4,"briefSummary":331,"conditions":332,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":333,"lastUpdatePostDateStruct":334,"startDateStruct":336,"completionDateStruct":338,"leadSponsor":340,"locationsCount":229},"100499938","a-study-to-learn-how-nusinersen-spinraza-affects-participants-with-spinal-muscular-atrophy-sma-who-took-it-before-or-during-pregnancy-and-about-the-health-of-their-babies-100499938","NCT05789758","A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies","Spinraza (Nusinersen) SMA Pregnancy Exposure Study Within Existing SMA Registries","Key Inclusion Criteria:\n\n* Participant who is currently pregnant (or was pregnant during the relevant exposure window) and enrolled in the United States (US) network of the ISMAR registry, the United Kingdom (UK) Adult SMA REACH, or the Germany, Austria, or Switzerland network of the SMArtCARE registry\n* Genetic documentation of spinal muscular atrophy linked to chromosome 5q (5q SMA)\n* Documentation that the participant was exposed to nusinersen up to 14 months prior to the first day of their LMP before conception, 14.5 months before conception, and\u002For at any time during their pregnancy\n\nKey Exclusion Criteria:\n\n* Treatment with risdiplam at any time from the first day of their LMP, 2 weeks prior to the date of conception (approximately 5 half-lives), and\u002For plans to receive treatment with risdiplam during pregnancy\n\nNOTE: Other protocol defined Inclusion\u002FExclusion criteria may apply","FEMALE","54 Years",{"count":5,"type":21},"In this study, researchers will know more about the effects of nusinersen, also known as Spinraza®, in pregnant participants with spinal muscular atrophy, also known as SMA. This is a drug available for doctors to prescribe for people with SMA. Due to the current treatment options that exist, people with SMA may now reach the age where they can become pregnant. But, there is not enough information known yet about what the effects of nusinersen may be on pregnant people with SMA or on their babies.\n\nThis is known as an \"observational\" study, which collects health information about study participants without changing their medical care. The pregnant participants for this study will be found using 3 different groups of SMA study research centers:\n\n* ISMAR-US (International SMA Registry in the United States)\n* UK Adult SMA-REACH (Adult SMA Research and Clinical Hub in the United Kingdom)\n* SMArtCARE (Austria, Germany, and Switzerland)\n\nThe main goal of this study is to collect birth and health information from 3 groups of participants and their babies. These groups are:\n\n* Those who received nusinersen 14 months before the first day of their last period before getting pregnant\n* Those who received nusinersen 14.5 months before the day they got pregnant\n* Those who received nusinersen during any time in their pregnancy\n\nThe main questions researchers want to learn about in this study are:\n\n* Loss of pregnancy overall\n* Loss of pregnancy before the baby was 20 weeks old\n* Loss of pregnancy after the baby becomes 20 weeks old\n* Live births\n* Loss of the baby after birth\n* Babies who have problems in their body that develop during pregnancy\n* Babies who are small for their age while in the participant's uterus\n* Pregnancy that happens outside of the uterus\n* How many participants die during pregnancy, while the baby is being born, and up to 12 weeks after delivering the baby\n* Babies who develop problems in their body after birth\n\nResearchers will also compare this information to people without SMA who have not received nusinersen.\n\nThis study will be done as follows:\n\n* Information will start being collected when the participant decides to join the study.\n* Participants will be contacted at each trimester (about every 3 months) to learn about their health and pregnancy.\n* Participants' doctors will be contacted at each trimester, when the participants are about 6 or 7 months pregnant, and about 4 weeks after the delivery of the baby.\n* The babies' doctors will be contacted when the baby is 1, 2, 6, 12, 18, and 24 months old.\n* Each participant will be in the study until the end of their pregnancy and for up to 12 weeks after delivery. Each baby will be in the study for up to 2 years after birth.\n* The study overall will last at least 10 years from when the first participant joins the study.",[158],"2026-03-06",{"date":335,"type":34},"2026-03-09",{"date":337,"type":34},"2023-12-15",{"date":339,"type":21},"2033-10-31",{"name":40,"class":41},{"id":342,"slug":343,"hasResults":11,"nctId":344,"briefTitle":345,"officialTitle":346,"acronym":4,"eligibilityCriteria":347,"healthyVolunteers":77,"sex":17,"minAge":18,"maxAge":78,"enrollmentInfo":348,"targetDuration":4,"studyType":22,"phases":350,"briefSummary":351,"conditions":352,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":353,"lastUpdatePostDateStruct":354,"startDateStruct":356,"completionDateStruct":358,"leadSponsor":360,"locationsCount":207},"100610279","phase-1-a-study-to-learn-more-about-the-safety-of-biib145-and-how-it-is-processed-in-the-body-of-healthy-adult-participants-100610279","NCT07225517","A Study to Learn More About the Safety of BIIB145 and How it is Processed in the Body of Healthy Adult Participants","A Phase 1, Randomized, Blinded, Placebo-Controlled, Single- and Multiple-Ascending-Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB145 in Healthy Adult Participants","Key Inclusion Criteria:\n\n* Have a body mass index (BMI) between 18 and 32 kilograms per square metre (kg\u002Fm\\^2), inclusive, and a total body weight \\> 50 kilograms (kg) at Screening and Check-In.\n* Must be in good health as determined by the Investigator, based on medical history and Screening evaluations. Good health is defined as no clinically relevant abnormalities identified by a detailed medical history, full physical examination, including blood pressure and pulse rate measurement, 12-lead electrocardiogram (ECG), and clinical laboratory tests.\n\nKey Exclusion Criteria:\n\n* History of any clinically significant blood disorders or cardiac, endocrine, gastrointestinal, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, or renal disease, or other major disease, as determined by the Investigator.\n* History of, or ongoing, malignant disease, including solid tumours and hematologic malignancies (except for basal cell carcinomas and squamous cell carcinomas that have been completely excised and considered cured at least 1 year prior to Check-In).\n* History of uncontrolled bleeding, or any risk of bleeding that, in the opinion of the Investigator, is clinically significant.\n* History of torsades de pointes or additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT syndrome), in the opinion of the Investigator.\n* Any values of coagulation parameters including international normalized ratio, prothrombin time, and activated partial thromboplastin time above upper limit of normal at Screening or Check-In.\n* Systolic blood pressure \\> 150 millimetres of mercury (mmHg) or \\\u003C 90 mmHg after resting for 5 minutes in the supine position at Screening and prior to dosing. If out of range, testing may be repeated once at Screening and once prior to dosing.\n* Any live or attenuated immunization or vaccination given within 30 days prior to Check-In or planned to be given during the study period.\n* Prior exposure to BIIB145 or any lymphocyte-depleting therapy or exposure to any lymphocyte-targeting therapy within 3 months prior to Check-In, or at least 5 half-lives or for the anticipated duration of the product's pharmacodynamics (PD) effects, whichever is longer.\n* Known lumbar spine deformity, degenerative arthritis of the lumbar spine, history of lumbar spinal surgery, spinal infection, spinal mass or trauma, and\u002For known evidence on magnetic resonance imaging (MRI) contraindicating lumbar puncture (LP).\n\nNOTE: Other protocol-defined Inclusion\u002FExclusion criteria may apply.",{"count":349,"type":21},104,[82],"In this study, researchers will learn for the first time about the safety of a study drug called BIIB145 and how the body responds to it. This is a \"Phase 1\" study. This kind of study is an early step in clinical research where the goal is to focus on the safety of the study drug. Another goal may be to learn how the study drug is processed by the body. BIIB145 was designed to help people with multiple sclerosis (MS). But, before it can be tested in people with MS, it must first be tested in healthy volunteers to learn about its safety and other effects.\n\nThe main goal of this study is to learn about the safety of BIIB145 and how it is processed by the body, with or without food.\n\nThe main questions researchers want to answer are:\n\n* How many participants have adverse events and serious adverse events during the study? An adverse event is a health problem that may or may not be caused by the study drug.\n* How does BIIB145 affect the participants' overall health?\n\nResearchers will also learn more about:\n\n* How BIIB145 is processed by the body, with or without food.\n\nThis study will be done as follows:\n\n* Participants will be screened to check if they can join the study. The screening period will be up to 28 days, after which participants will check into the study research center.\n* There will be 3 parts to this study.\n* Part 1: Participants will take a single dose of BIIB145 or a placebo after not eating overnight. A placebo is something that looks like the study drug but does not contain any medicine. A placebo is also given in the same way as the study drug. Participants in Part 1 will be in the study for up to 42 days.\n* Part 2: This part of the study will have a \"crossover\" design. This means that all participants in Part 2 will all take BIIB145 twice, once with food and once without food. When taken with food, they will finish a meal about 30 minutes before their dose. Without food, they will not eat overnight before taking their dose. But, the order in which they take BIIB145 with or without food depends on the group to which they are randomly assigned. Participants in Part 2 will be in the study for up to 56 days.\n* Part 3: Participants will take a dose of BIIB145 or the placebo once a day for 14 days. For each dose, participants will not eat overnight before taking it. Participants in Part 3 will be in the study for up to 56 days.\n* For Part 1, participants will stay at the study research center for 4 days after screening. There will be 2 other visits to the center to check on participants' health on Day 7 and Day 14.\n* For Part 2, at 2 different times, participants will stay at the center for a period of 4 days at a time, after screening. There will be a break of about 7 days between stays. There will be 3 other visits to the center to check on their health on Day 7 (of each period) and on Day 14.\n* For Part 3, participants will stay at the center for 17 days after screening. There will be 2 other visits to the center to check on their health on Day 21 and Day 28.",[85],"2026-02-05",{"date":355,"type":34},"2026-02-06",{"date":357,"type":34},"2025-12-18",{"date":359,"type":21},"2027-02-02",{"name":40,"class":41},{"id":362,"slug":363,"hasResults":11,"nctId":364,"briefTitle":365,"officialTitle":366,"acronym":4,"eligibilityCriteria":367,"healthyVolunteers":11,"sex":328,"minAge":18,"maxAge":4,"enrollmentInfo":368,"targetDuration":4,"studyType":219,"phases":4,"briefSummary":370,"conditions":371,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":373,"lastUpdatePostDateStruct":374,"startDateStruct":376,"completionDateStruct":378,"leadSponsor":380,"locationsCount":207},"100623579","a-long-term-study-of-patient-reported-changes-in-postpartum-depression-symptoms-in-people-starting-zuranolone-100623579","NCT07398469","A Long-Term Study of Patient-Reported Changes in Postpartum Depression Symptoms in People Starting Zuranolone","A Longitudinal Online Survey of Patient-Reported Changes in Postpartum Depression Symptoms Following Zuranolone Use","Key Inclusion Criteria:\n\n* Participants with a prescription for zuranolone via Accredo Specialty Pharmacy.\n* Recent pregnancy in the last 12 months.\n* Confirmed diagnosis of PPD.\n* Able to complete the questionnaires by themselves in English.\n\nKey Exclusion Criteria:\n\n* Prior fill of zuranolone or brexanolone in last 12 months.\n* Participants who have taken 1 or more doses of zuranolone at the time of study screening.\n* Current or history of bipolar disorder.\n* Failure to complete baseline (Day 0) surveys.\n* Declined to consent.\n* Pregnancy that ended more than 12 months ago.\n\nNote: Other protocol-defined Inclusion\u002FExclusion criteria may apply.",{"count":369,"type":21},200,"In this study, researchers will learn how postpartum depression symptoms may change after people first start taking zuranolone. This is a drug available for doctors to prescribe for people with postpartum depression, also known as PPD. After giving birth, people with PPD can suffer from symptoms like tiredness, sadness, and a loss of interest in their daily activities.\n\nThis is known as an \"observational\" study, which collects health information about study participants without changing their medical care. Participants for this study will be found in the United States using a database from Accredo Specialty Pharmacy. This will include anyone who has a new prescription for zuranolone and were pregnant in the last 12 months before joining the study.\n\nThe main goal of this study is to learn more about how zuranolone affects the participants' PPD symptoms. This will be done using a questionnaire called the Edinburgh Postnatal Depression Scale, also known as the EPDS.\n\nThe main question that researchers want to answer is:\n\n• Do PPD symptoms change after treatment with zuranolone based on EPDS scores measured at Day 15?\n\nResearchers will also learn about :\n\n* Changes in participants' EPDS scores at Day 45 and Day 90 in the study\n* Changes in EPDS scores at Day 15, Day 45, and Day 90 in a group of participants who have moderately severe PPD before starting zuranolone\n* How many participants breastfeed their babies while taking zuranolone\n* How many participants report not starting new medicine after finishing their zuranolone treatment\n\nThe study will be done as follows:\n\n* People with PPD who get a new zuranolone prescription through Accredo Specialty Pharmacy will be contacted by email or phone to ask them about their interest in participating in the study.\n* Before taking their first dose of zuranolone, participants will be asked to answer written questions about their symptoms using the online EPDS survey. They will also answer other survey questions about their background, environment, and general health information. Participants must take their first dose of zuranolone within 7 days of joining the study.\n* Participants will then be asked to answer questions using the EPDS survey, 15 days, 45 days, and 90 days after taking the first dose of zuranolone. Each survey will take about 10-15 minutes to finish.",[372],"Postpartum Depression","2026-02-04",{"date":375,"type":34},"2026-02-10",{"date":377,"type":34},"2025-12-22",{"date":379,"type":21},"2027-03-29",{"name":40,"class":41},{"id":382,"slug":383,"hasResults":11,"nctId":384,"briefTitle":385,"officialTitle":386,"acronym":4,"eligibilityCriteria":387,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":388,"targetDuration":4,"studyType":22,"phases":390,"briefSummary":392,"conditions":393,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":394,"lastUpdatePostDateStruct":395,"startDateStruct":397,"completionDateStruct":399,"leadSponsor":401,"locationsCount":207},"100610141","phase-4-a-study-to-learn-more-about-the-long-term-safety-of-tofersen-qalsody-in-chinese-participants-with-sod-1-amyotrophic-lateral-sclerosis-als-100610141","NCT07223723","A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)","A Multicenter, Open-Label, Postmarketing Surveillance Study of Tofersen (BIIB067) in Adults With Amyotrophic Lateral Sclerosis (ALS) Associated With a Mutation in the Superoxide Dismutase 1 (SOD1) Gene in China","Key Inclusion Criteria:\n\n* Age ≥ 18 years at the time of informed consent.\n* Must have diagnosis of SOD1-ALS.\n* If taking riluzole, participant must be on a stable dose for ≥ 30 days prior to Day 1 and expected to remain at that dose until the final study visit.\n* If taking edaravone, participant must have initiated edaravone ≥ 60 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study.\n* All women of childbearing potential must practice effective contraception during the study.\n\nKey Exclusion Criteria:\n\n* Hypersensitivity to the active substance or any of the excipients of tofersen injection.\n* Current or past administration of tofersen injection, in either a commercial or a clinical study setting.\n* Prior or current treatment with small interfering ribonucleic acid (RNA), stem cell therapy, or gene therapy.\n* Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer, prior the Baseline Visit.\n* Participants who are pregnant or currently breastfeeding, and those intending to become pregnant during the study.\n\nNote: Other protocol-defined Inclusion\u002FExclusion criteria may apply.",{"count":389,"type":21},12,[391],"PHASE4","In this study, researchers will learn more about the safety of tofersen, also known as Qalsody®. This is a drug available for doctors to prescribe for people with a certain type of amyotrophic lateral sclerosis, also known as ALS. This type is in people who have a mutation in the superoxide dismutase 1 gene, also known as SOD-1.\n\nThis is known as a \"postmarketing\" study. In this kind of the study, the goal is to learn more about how a drug works after it has been approved for use in the general public. Tofersen was approved in China in September 2024. The main goal of this study is to collect long-term safety information in Chinese participants with SOD-1 ALS.\n\nThe main question researchers want to answer in this study is:\n\n• How many participants have adverse events (AEs) and serious adverse events (SAEs)?\n\nAn AE is a health problem that may or may not be caused by a drug during the study. An AE is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care.\n\nResearchers will also learn more about :\n\n* How the body processes tofersen.\n* How much tofersen is found in the cerebrospinal fluid (CSF), or the fluid that surrounds the brain and the spinal cord.\n\nThis study will be done as follows:\n\n* Participants will be screened to check if they can join the study. The screening period will be up to 4 weeks.\n* After joining the study, participants will receive the first 3 doses of 100 milligrams (mg) of tofersen about 14 days apart. This will be given through an intrathecal (IT) injection. This means it will be given into the fluid surrounding the spine.\n* After that, participants will receive 10 more doses every 28 days through IT injections. Participants will have up to 13 total doses of tofersen in this study.\n* Participants will have up to 15 visits to their study research center. Each participant will be in the study for up to 52 weeks (1 year).",[265],"2026-01-16",{"date":396,"type":34},"2026-01-20",{"date":398,"type":34},"2025-12-11",{"date":400,"type":21},"2027-12-16",{"name":40,"class":41},{"id":403,"slug":404,"hasResults":11,"nctId":405,"briefTitle":406,"officialTitle":407,"acronym":4,"eligibilityCriteria":408,"healthyVolunteers":11,"sex":328,"minAge":4,"maxAge":4,"enrollmentInfo":409,"targetDuration":411,"studyType":219,"phases":4,"briefSummary":412,"conditions":413,"keywords":415,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":418,"lastUpdatePostDateStruct":419,"startDateStruct":421,"completionDateStruct":423,"leadSponsor":425,"locationsCount":426},"100489851","pregnancy-exposure-registry-for-vumerity-diroximel-fumarate-100489851","NCT05658497","Pregnancy Exposure Registry for Vumerity (Diroximel Fumarate)","Vumerity (Diroximel Fumarate) Prospective MS Pregnancy Exposure Registry","Key Inclusion Criteria:\n\n* Participant must have a diagnosis of MS\n* Documentation that the participant was one of the following:\n\n  1. exposed to DRF at any time from 2 weeks after the first day of their LMP (i.e., conception date) up through any time during pregnancy. (If exact exposure dates are unknown, the reporter must be able to specify or estimate trimester of exposure).\n  2. unexposed to any DMT during pregnancy, defined as having never received DMT therapy; discontinued treatment with DRF at least 1 day before 2 weeks after the first day of their LMP (i.e., conception date); or discontinued a non Registry-specified MS DMT more than 5 times its half-life prior to 2 weeks after the first day of their LMP (i.e., conception date)\n* Participants with knowledge of the outcome of the pregnancy (e.g., pregnancy loss or live birth)\n\nKey Exclusion Criteria:\n\n\\- None\n\nNOTE: Other protocol defined Inclusion criteria may apply",{"count":410,"type":21},908,"52 Weeks","The primary objectives of the study are to estimate the risk of major congenital malformations (MCMs) in infants born to women with multiple sclerosis (MS) who were exposed to diroximel fumarate (DRF) at any time from 2 weeks after the first day of their last menstrual period (LMP) up through the first trimester of pregnancy and to comparatively evaluate pregnancy outcomes with MCMs in women with MS who were exposed to DRF at any time from 2 weeks after the first day of their LMP through the first trimester of pregnancy with the following: i) women with MS who were unexposed to disease modifying therapies (DMTs) and, ii) women with MS who were exposed to other DMTs (e.g., Avonex and Tysabri Pregnancy Registries).\n\nThe secondary objective of the study is to evaluate pregnancy outcomes in women with DRF exposure at any time from 2 weeks after the first day of their LMP through the end of pregnancy compared with the following: i) women with MS who were unexposed to DMTs, ii) women with dimethyl fumarate (DMF) exposure, iii) women with MS who were exposed to other DMTs (e.g., Avonex and Tysabri Pregnancy Registries), and iv) women without MS (e.g., women from external, general population comparators).",[414],"Multiple Sclerosis",[416,417],"Pregnancy","Relapsing forms of MS","2025-07-31",{"date":420,"type":34},"2025-08-01",{"date":422,"type":34},"2023-10-27",{"date":424,"type":21},"2032-07-06",{"name":40,"class":41},7,{"id":428,"slug":429,"hasResults":11,"nctId":430,"briefTitle":431,"officialTitle":432,"acronym":4,"eligibilityCriteria":433,"healthyVolunteers":11,"sex":328,"minAge":18,"maxAge":4,"enrollmentInfo":434,"targetDuration":4,"studyType":219,"phases":4,"briefSummary":435,"conditions":436,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":438,"lastUpdatePostDateStruct":439,"startDateStruct":441,"completionDateStruct":443,"leadSponsor":445,"locationsCount":207},"100596619","a-study-to-learn-more-about-how-zuranolone-affects-postpartum-depression-symptoms-in-participants-who-took-it-within-1-year-after-the-end-of-their-pregnancy-100596619","NCT07047820","A Study to Learn More About How Zuranolone Affects Postpartum Depression Symptoms in Participants Who Took it Within 1 Year After The End of Their Pregnancy","Prospective, Observational Survey Study Assessing Effectiveness of Zuranolone in Improving PPD Symptoms in the Real-world Setting","Key Inclusion Criteria:\n\n* Adults who were prescribed and had one successful fill for zuranolone within 12 months following end of pregnancy for the treatment of PPD from June 2025 to May 2026 through CVS Specialty Pharmacy.\n* Currently reside in the United States.\n* Able to complete the questionnaires independently.\n\nKey Exclusion Criteria:\n\n* Prior fill of zuranolone or brexanolone in last 12 months.\n* Participants with 2 or more doses of zuranolone at the time of screening.\n* Current or history of bipolar disorder.\n* Failure to complete baseline surveys prior to 2nd dose of zuranolone.\n* Pregnancy that ended more than 12 months ago.\n\nOther protocol-defined Inclusion\u002FExclusion criteria may apply.",{"count":369,"type":21},"In this study, researchers will learn more about how zuranolone affects the symptoms of postpartum depression, also known as PPD. Zuranolone is a drug that healthcare professionals can prescribe for adults with PPD. After giving birth, adults with PPD can suffer from symptoms such as tiredness, sadness, and a loss of interest in their daily activities.\n\nThis study is known as an observational study, which means it collects health information about study participants after a healthcare professional has already prescribed treatment. Participants for this study will be found in the United States using a database from CVS Specialty Pharmacy. This will include anyone who was prescribed zuranolone between June 2025 and May 2026 and who filled the prescription within 1 year after the end of their pregnancy.\n\nThe main goal of this study is to learn more about how zuranolone affects the participants' PPD symptoms. This will be measured using a questionnaire completed by participants called the Edinburgh Postnatal Depression Scale, also known as the EPDS. A higher score on the EPDS may indicate more severe PPD symptoms.\n\nThe main question researchers want to answer in this study is:\n\n\\- Do PPD symptoms change after treatment with zuranolone based on EPDS scores measured at Day 15?\n\nResearchers will also learn more about:\n\n* Changes in participants' EPDS scores from before treatment to Day 45, which is 30 days after treatment ends.\n* How many participants breastfeed their babies while taking zuranolone\n* How many participants do not start new medicine after finishing their zuranolone treatment\n* How many participants take new medicines after finishing zuranolone\n* How many participants already tried other medicines for their depression symptoms before joining this study\n* How many participants take other medicines in general while taking zuranolone\n\nThis study will be done as follows:\n\nPeople who fill their zuranolone prescription through CVS Specialty Pharmacy will be contacted by email or phone to ask them about their interest in participating in the study.\n\nThose who agree to take part will answer written questions about their symptoms using the EPDS tool. They will also answer other survey questions about their background, environment, and general health information. Participants must take their first dose of zuranolone within 7 days of joining the study.\n\nParticipants will then be asked to answer questions using the EPDS tools, 15 days and 45 days after taking the first dose of zuranolone.",[437],"Depression, Postpartum","2025-07-30",{"date":440,"type":34},"2025-08-03",{"date":442,"type":34},"2025-06-30",{"date":444,"type":21},"2026-08-24",{"name":40,"class":41},{"id":447,"slug":448,"hasResults":11,"nctId":449,"briefTitle":450,"officialTitle":451,"acronym":4,"eligibilityCriteria":452,"healthyVolunteers":11,"sex":328,"minAge":215,"maxAge":4,"enrollmentInfo":453,"targetDuration":4,"studyType":219,"phases":4,"briefSummary":454,"conditions":455,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":456,"lastUpdatePostDateStruct":457,"startDateStruct":459,"completionDateStruct":461,"leadSponsor":463,"locationsCount":207},"100564400","a-study-to-learn-how-biib141-omaveloxolone-affects-the-health-of-participants-with-friedrichs-ataxia-who-took-it-during-pregnancy-andor-during-breastfeeding-and-about-the-health-of-their-babies-100564400","NCT06628687","A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and\u002For During Breastfeeding and About the Health of Their Babies","A Post-marketing, Observational, Descriptive Study to Assess the Risk Associated With Pregnancy, the Maternal Complications, and Adverse Effects on the Developing Fetus, Neonate, and Infant Among Individuals Exposed to Omaveloxolone During Pregnancy and\u002For Lactation","Key Inclusion Criteria:\n\n\\- Exposure to omaveloxolone for FA at any time during pregnancy (from 12 days prior to conception to pregnancy outcome) and\u002For at any time during lactation (up to 1 year of infant age or weaning, whichever comes first).\n\nKey Exclusion Criteria:\n\n\\- Not having exposure to omaveloxolone for FA.\n\nNote: Other protocol-defined Inclusion\u002FExclusion criteria may apply.",{"count":5,"type":21},"In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS. This is a drug available for doctors to prescribe for people with Friedrich's Ataxia, also known as FA. This is known as an \"observational\" study, which collects health information about study participants without changing their medical care. Participants for this study will have taken BIIB141 at any time during pregnancy and\u002For while breastfeeding or pumping up through the first year after delivery. Participants can join this study on their own or they may be enrolled by their regular doctors. This study is also known as the \"SKYCLARYS (Omaveloxolone) Pregnancy and Lactation Surveillance Program.\"\n\nThe main objective of this study is to learn more about how BIIB141 may affect pregnancy, as well as any effects on the health of the mother and of the baby during its first year of life.\n\nThe main question researchers want to answer in this study is:\n\n· Does taking BIIB141 during pregnancy or breastfeeding lead to any major birth defects?\n\nResearchers will also learn more about:\n\n* Does taking BIIB141 during pregnancy or breastfeeding lead to any minor birth defects?\n* Does taking BIIB141 during pregnancy or breastfeeding affect the following:\n* Gestational diabetes, a disease that can happen during pregnancy that affects how your body uses sugar\n* Pre-eclampsia, a pregnancy-related high blood pressure disease\n* Unborn baby being small for its expected age (usually in weeks)\n* Loss of an unborn baby\n* Live birth\n* Premature birth\n* Loss of a newborn\n* Growth or developmental delays in the baby\n* Serious illness in the baby resulting in hospitalization\n* Serious infections in the baby, or ones in babies with a weakened immune system\n\nThis study will be done as follows:\n\n* Participants will join the study after signing an informed consent form, also known as an ICF.\n* During the study, health information from the participants' regular visits to their doctor will be collected based on whether participant joined the study while pregnant or after the baby is born.\n* Each participant will be in the study for up to 1 year after the birth of their child, unless they decide to leave early. Overall, this study is expected to last at least 10 years.",[133],"2025-06-23",{"date":458,"type":34},"2025-06-26",{"date":460,"type":21},"2026-10-26",{"date":462,"type":21},"2035-04-30",{"name":40,"class":41},""]