[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"muscular-atrophy-spinal\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:muscular-atrophy-spinal":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,7,0,[8,41,63,89,109,132,157],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"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",false,"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.","ALL","0 Days","7 Months",{"count":21,"type":22},42,"ESTIMATED","INTERVENTIONAL",[25],"PHASE3","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.",[28],"Muscular Atrophy, Spinal","NOT_YET_RECRUITING","2026-06-11",{"date":32,"type":33},"2026-06-15","ACTUAL",{"date":35,"type":22},"2026-09-04",{"date":37,"type":22},"2033-07-26",{"name":39,"class":40},"Biogen","INDUSTRY",{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":47,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":49,"enrollmentInfo":50,"targetDuration":4,"studyType":23,"phases":52,"briefSummary":53,"conditions":54,"keywords":4,"overallStatus":55,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":56,"startDateStruct":57,"completionDateStruct":59,"leadSponsor":61,"locationsCount":62},"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":51,"type":22},30,[25],"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.",[28],"RECRUITING",{"date":32,"type":33},{"date":58,"type":33},"2026-04-28",{"date":60,"type":22},"2032-05-29",{"name":39,"class":40},4,{"id":64,"slug":65,"hasResults":11,"nctId":66,"briefTitle":67,"officialTitle":68,"acronym":69,"eligibilityCriteria":70,"healthyVolunteers":11,"sex":17,"minAge":71,"maxAge":72,"enrollmentInfo":73,"targetDuration":4,"studyType":23,"phases":75,"briefSummary":77,"conditions":78,"keywords":4,"overallStatus":55,"whyStopped":4,"lastUpdateSubmitDate":79,"lastUpdatePostDateStruct":80,"startDateStruct":82,"completionDateStruct":84,"leadSponsor":86,"locationsCount":88},"100505485","phase-4-a-study-evaluating-the-effectiveness-and-safety-of-risdiplam-administered-in-pediatric-patients-with-spinal-muscular-atrophy-who-experienced-a-plateau-or-decline-in-function-after-gene-therapy-100505485","NCT05861999","A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy","A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy","HINALEA 2","Inclusion Criteria:\n\n* \\\u003C2 years of age at the time of informed consent\n* Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the Survival of Motor Neuron 1 (SMN1) gene\n* Confirmed presence of two SMN2 gene copies as documented through laboratory testing\n* Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically\n* Has received onasemnogene abeparvovec for SMA no less than 13 weeks prior to enrollment\n* If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration.\n* In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy (with a duration of 26 weeks or less) documented by 2 individual time points in the functions as follows: swallowing AND one additional function\u002Fability (respiratory, motor function, other) per appropriate expectation.\n\nExclusion Criteria:\n\n* Previous or current enrolment in investigational study prior to initiation of study treatment\n* Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information\n* Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide\n* Concomitant or previous use of an anti-myostatin agent\n* Participants requiring invasive ventilation or tracheostomy\n* Presence of feeding tube and an OrSAT score of 0\n* Hospitalization for pulmonary event within the last 2 months, or any planned hospitalization at the time of screening\n* Any major illness requiring hospitalization within 1 month before the screening examination or any febrile illness within 1 week prior to screening and up to first dose administration.","3 Months","24 Months",{"count":74,"type":22},28,[76],"PHASE4","This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \\\u003C2 years of age genetically diagnosed with SMA.",[28],"2026-06-05",{"date":81,"type":33},"2026-06-09",{"date":83,"type":33},"2024-08-14",{"date":85,"type":22},"2029-03-31",{"name":87,"class":40},"Hoffmann-La Roche",19,{"id":90,"slug":91,"hasResults":11,"nctId":92,"briefTitle":93,"officialTitle":94,"acronym":95,"eligibilityCriteria":96,"healthyVolunteers":11,"sex":17,"minAge":71,"maxAge":72,"enrollmentInfo":97,"targetDuration":4,"studyType":23,"phases":98,"briefSummary":99,"conditions":100,"keywords":4,"overallStatus":55,"whyStopped":4,"lastUpdateSubmitDate":101,"lastUpdatePostDateStruct":102,"startDateStruct":104,"completionDateStruct":106,"leadSponsor":107,"locationsCount":108},"100505484","phase-4-a-study-evaluating-the-effectiveness-and-safety-of-risdiplam-administered-as-an-early-intervention-in-pediatric-participants-with-spinal-muscular-atrophy-after-gene-therapy-100505484","NCT05861986","A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy","A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy","HINALEA 1","Inclusion Criteria:\n\n* \\\u003C2 years of age at the time of informed consent\n* Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the Survival of Motor Neuron 1 (SMN1) gene\n* Confirmed presence of two SMN2 gene copies as documented through laboratory testing\n* Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically\n* Has received onasemnogene abeparvovec for SMA no less than 13 weeks, but not more than months 30 weeks, prior to enrollment\n* If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration\n* Has, in the opinion of the investigator, not experienced clinically significant decline in function from the time of onasemnogene abeparvovec administration\n\nExclusion Criteria:\n\n* Previous or current enrolment in investigational study prior to initiation of study treatment\n* Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information\n* Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide\n* Concomitant or previous use of an anti-myostatin agent\n* Participants requiring invasive ventilation or tracheostomy\n* Participants requiring awake non-invasive ventilation or with awake hypoxemia (Arterial Oxygen Saturation \\[SaO2\\] \\\u003C95%) with or without ventilator support\n* Presence of feeding tube and an OrSAT score of 0\n* Hospitalization for pulmonary event within the last 2 months, or any planned hospitalization at the time of screening\n* Any major illness requiring hospitalization within 1 month before the screening examination or any febrile illness within 1 week prior to screening and up to first dose administration.",{"count":74,"type":22},[76],"This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \\\u003C 2 years of age genetically diagnosed with SMA.",[28],"2026-05-01",{"date":103,"type":33},"2026-05-04",{"date":105,"type":33},"2024-05-30",{"date":85,"type":22},{"name":87,"class":40},16,{"id":110,"slug":111,"hasResults":11,"nctId":112,"briefTitle":113,"officialTitle":114,"acronym":115,"eligibilityCriteria":116,"healthyVolunteers":11,"sex":17,"minAge":117,"maxAge":4,"enrollmentInfo":118,"targetDuration":4,"studyType":23,"phases":120,"briefSummary":122,"conditions":123,"keywords":4,"overallStatus":55,"whyStopped":4,"lastUpdateSubmitDate":124,"lastUpdatePostDateStruct":125,"startDateStruct":127,"completionDateStruct":129,"leadSponsor":131,"locationsCount":88},"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":119,"type":22},58,[121],"PHASE1","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.",[28],"2026-03-16",{"date":126,"type":33},"2026-03-18",{"date":128,"type":33},"2025-01-16",{"date":130,"type":22},"2027-06-25",{"name":39,"class":40},{"id":133,"slug":134,"hasResults":11,"nctId":135,"briefTitle":136,"officialTitle":137,"acronym":4,"eligibilityCriteria":138,"healthyVolunteers":11,"sex":139,"minAge":140,"maxAge":141,"enrollmentInfo":142,"targetDuration":144,"studyType":145,"phases":4,"briefSummary":146,"conditions":147,"keywords":4,"overallStatus":55,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":149,"startDateStruct":151,"completionDateStruct":153,"leadSponsor":155,"locationsCount":156},"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","18 Years","54 Years",{"count":143,"type":22},20,"2 Years","OBSERVATIONAL","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.",[28],"2026-03-06",{"date":150,"type":33},"2026-03-09",{"date":152,"type":33},"2023-12-15",{"date":154,"type":22},"2033-10-31",{"name":39,"class":40},14,{"id":158,"slug":159,"hasResults":11,"nctId":160,"briefTitle":161,"officialTitle":162,"acronym":163,"eligibilityCriteria":164,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":165,"targetDuration":167,"studyType":145,"phases":4,"briefSummary":168,"conditions":169,"keywords":170,"overallStatus":55,"whyStopped":4,"lastUpdateSubmitDate":173,"lastUpdatePostDateStruct":174,"startDateStruct":176,"completionDateStruct":178,"leadSponsor":180,"locationsCount":183},"100497300","natural-history-of-sma-100497300","NCT05755451","Natural History of SMA","Natural History of SMA: Long Term Longitudinal Study and Development of a Registry","iSMAR","Inclusion Criteria:\n\nall patients\n\nExclusion Criteria:\n\nif enrolled in clinical trials",{"count":166,"type":22},1200,"15 Years","This is an investigator initiated observational study with the aim to record several aspects of function, care and adverse events in a large cohort of SMA patients followed longitudinally by using a structured academic disease registry.",[28],[171,172],"natural history","spinal muscular atrophy","2023-03-02",{"date":175,"type":33},"2023-03-06",{"date":177,"type":33},"2018-06-21",{"date":179,"type":22},"2033-12-30",{"name":181,"class":182},"Fondazione Policlinico Universitario Agostino Gemelli IRCCS","OTHER",5]