[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Ionis Pharmaceuticals, Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":289},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,12,0,[8,43,69,93,116,140,161,187,209,229,250,270],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"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},"100586379","phase-3-reveal-a-phase-3-study-of-ion582-in-angelman-syndrome-100586379",false,"NCT06914609","REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome","Phase 3 Study of the Efficacy and Safety of ION582 in Children and Adults With Angelman Syndrome","Key Inclusion Criteria:\n\n1. The participants caregiver(s)\u002F legally authorized representative must have given written informed consent and any authorizations required by local law and be able to comply with all study requirements.\n2. Medically stable and can undergo sedation and\u002For general anesthesia without intubation.\n3. Male or female between 2 and lesser than or equal to (≤)50 years of age, depending on specific cohort, at the time of the in-clinic Screening visit.\n4. Participant has a clinical diagnosis of Angelman syndrome (AS) with molecular confirmation of either Ubiquitin-protein ligase E3A (UBE3A) deletion or UBE3A mutation.\n5. Currently receiving stable doses of concomitant medications typically prescribed for AS, such as anti-epileptic medication, behavioral management medications, sleep medications, gabapentin, cannabidiol, and special diets, supplements, or nutritional support for at least 8 weeks prior to the Baseline visit.\n6. Legally authorized representative\u002Fcaregiver(s) agree(s) not to post any of the participant's personal medical data or information related to the study on any website or social media site (e.g., Facebook, Instagram, X (formerly Twitter), YouTube, TikTok, etc.) from the time of enrollment until they are notified that the study is completed.\n\nKey Exclusion Criteria:\n\n1. Must not have any clinically significant abnormalities in medical history (e.g., major surgery within 3 months of screening), or on physical examination for which treatment with an antisense oligonucleotide (ASO) would be contraindicated or which, in the opinion of the Principal Investigator (PI), could confound the results of this study.\n2. Known brain or spinal disease that would interfere with the lumbar puncture (LP) procedure, cerebrospinal fluid (CSF) circulation, or presence of other factors would affect the safety of the LP procedure.\n3. Must not have any other conditions, which, in the opinion of the Investigator, would make the participant unsuitable for inclusion or could interfere with the participant participating in or completing the study.\n4. Must not have any laboratory abnormalities or any other clinically significant abnormalities that would, as assessed by the Investigator, at screening or Baseline, render a participant unsuitable for inclusion.\n5. Previous treatment with an oligonucleotide (including small interfering ribonucleic acid (RNA) \\[siRNA\\], ASOs) gene therapy or gene editing. This exclusion criterion does not apply to approved nucleic acid-based vaccines, including mRNA vaccines, which are allowed.\n6. Has molecular confirmation of AS due to paternal uniparental disomy, imprinting center defect, or mosaic findings.\n\nOther inclusion\u002Fexclusion criteria may apply.","ALL","2 Years","50 Years",{"count":20,"type":21},158,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","The purpose of this study is to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene.",[27],"Angelman Syndrome",[29,27],"ION582","RECRUITING","2026-06-22",{"date":33,"type":34},"2026-06-23","ACTUAL",{"date":36,"type":34},"2025-06-10",{"date":38,"type":21},"2030-04",{"name":40,"class":41},"Ionis Pharmaceuticals, Inc.","INDUSTRY",40,{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":50,"enrollmentInfo":51,"targetDuration":4,"studyType":22,"phases":53,"briefSummary":56,"conditions":57,"keywords":59,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":61,"lastUpdatePostDateStruct":62,"startDateStruct":63,"completionDateStruct":65,"leadSponsor":67,"locationsCount":68},"100633827","phase-1-ascend-safety-and-tolerability-of-ion337-for-the-treatment-of-dravet-syndrome-100633827","NCT07531745","ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome","Phase 1-2, Open-Label, Single and Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION337 in Patients With Dravet Syndrome","Key Inclusion Criteria:\n\n1. Participant is aged ≥ 2 to ≤ 12 years old at the time of informed consent.\n2. Participant has at least 1 parent or caregiver ≥ 18 years old who is willing and able to provide informed consent (signed and dated) and attend all scheduled study visits.\n3. Has a documented diagnosis of DS according to the International League Against Epilepsy (ILAE) criteria and as agreed by the Epilepsy Study Consortium, Inc (ESCI).\n4. Has confirmation of a pathogenic or likely pathogenic SCN1A variant.\n5. Must be currently receiving ≥ 1 concomitant ASM at a stable dose\u002Fregimen for ≥ 4 weeks prior to informed consent.\n6. Must have all other interventions for epilepsy (including ketogenic diet or VNS) as well as any other concomitant medications including medications for behavioral management, sleep, and supplements or nutritional support stable for ≥ 4 weeks prior to informed consent. Vagus nerve stimulator implantation must have occurred ≥ 6 months prior to informed consent.\n7. Experiences the required number of major motor seizures during the Screening Period.\n\nKey Exclusion Criteria:\n\n1. Known brain or spinal disease that would interfere with the LP procedure or CSF circulation, or presence of other factors that would affect the safety of the LP procedure.\n2. Pathogenic or likely pathogenic variant in another gene that causes epilepsy.\n3. Has had prior treatment with or is currently enrolled in an interventional clinical trial for a gene therapy or for another antisense oligonucleotide (ASO) for the treatment of DS.\n4. Has had treatment with or is currently enrolled in an interventional clinical trial of any other investigational drug, biological agent, or device within 30 days prior to Screening, or 5 half-lives of investigational agent, whichever is longer.\n5. Current treatment with an anti-seizure medication (ASM) acting primarily as a sodium channel blocker, as maintenance treatment.\n6. Prior brain surgeries including: corpus callosotomy, implantation of device for deep brain stimulation or any other palliative brain surgery intended to reduce seizure burden.\n\nNote: Other protocol pre-specified inclusion\u002Fexclusion criteria may apply.","12 Years",{"count":52,"type":21},32,[54,55],"PHASE1","PHASE2","The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).",[58],"Dravet Syndrome",[60],"Dravet syndrome","2026-06-20",{"date":33,"type":34},{"date":64,"type":34},"2026-05-21",{"date":66,"type":21},"2030-12",{"name":40,"class":41},3,{"id":70,"slug":71,"hasResults":11,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":4,"eligibilityCriteria":75,"healthyVolunteers":11,"sex":16,"minAge":76,"maxAge":4,"enrollmentInfo":77,"targetDuration":4,"studyType":22,"phases":79,"briefSummary":80,"conditions":81,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":84,"lastUpdatePostDateStruct":85,"startDateStruct":87,"completionDateStruct":89,"leadSponsor":91,"locationsCount":92},"100641263","phase-2-a-study-to-assess-the-safety-tolerability-and-efficacy-of-ion775-100641263","NCT07604974","A Study to Assess the Safety, Tolerability and Efficacy of ION775","A Phase 2, Open-label Trial Evaluating the Safety, Tolerability and Efficacy of ION775 in Adults With Hypertriglyceridemia","Key Inclusion Criteria:\n\n1. Adult males and females with HTG (fasting TG more than or equal to (≥) 350 milligrams per deciliter (mg\u002FdL) \\[3.95 millimoles per liter (mmol\u002FL)\\] or with sHTG (fasting TG ≥ 500 mg\u002FdL \\[5.65 mmol\u002FL\\]).\n2. Participants should be on standard of care lipid-lowering medications per local guidelines.\n\nKey Exclusion Criteria:\n\n1. Hemoglobin A1c (HbA1c) ≥ 8.5% at Screening.\n2. Alanine aminotransferase or aspartate aminotransferase \\> 2.0 × upper limit of normal.\n3. Total bilirubin \\> 1.5 upper limit of normal unless due to Gilbert's syndrome.\n4. Estimated GFR \\\u003C 30 mL\u002Fmin\u002F1.73 m\\^2.\n\nNote: Other protocol pre-specified inclusion\u002Fexclusion criteria may apply.","18 Years",{"count":78,"type":21},72,[55],"The main objective of this study is to evaluate the effect of ION775 on fasting triglyceride (TG) levels in participants with hypertriglyceridemia (HTG) and severe hypertriglyceridemia (sHTG).",[82,83],"Hypertriglyceridemia","Severe Hypertriglyceridemia","2026-06-17",{"date":86,"type":34},"2026-06-18",{"date":88,"type":34},"2026-05-12",{"date":90,"type":21},"2028-03",{"name":40,"class":41},16,{"id":94,"slug":95,"hasResults":11,"nctId":96,"briefTitle":97,"officialTitle":98,"acronym":4,"eligibilityCriteria":99,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":100,"enrollmentInfo":101,"targetDuration":4,"studyType":22,"phases":103,"briefSummary":104,"conditions":105,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":107,"lastUpdatePostDateStruct":108,"startDateStruct":110,"completionDateStruct":112,"leadSponsor":114,"locationsCount":115},"100615887","phase-3-donidalorsen-treatment-in-children-with-hereditary-angioedema-100615887","NCT07298447","Donidalorsen Treatment in Children With Hereditary Angioedema","An Open-Label Study of Donidalorsen in Pediatric Patients Age 2 to Less Than 12 Years Old With Hereditary Angioedema","Key Inclusion Criteria:\n\n1. Must be between the ages of 2 and less than 12 years, inclusive, at the time of informed consent and, as applicable, assent.\n2. Must weigh at least 9 kg at the time of informed consent and, as applicable, assent.\n3. Documented diagnosis of HAE-1\u002FHAE-2 based upon both of the following:\n\n   1. Documented clinical history consistent with HAE (SC or mucosal, non-pruritic swelling episodes without accompanying urticaria).\n   2. Diagnostic testing results that confirm HAE-1\u002FHAE-2: C1-inhibitor (C1-INH) functional level \\\u003C50% normal level AND complement factor C4 level below the lower limit of normal (LLN); OR a known pathogenic mutation in the SERPING1 gene.\n\nKey Exclusion Criteria:\n\n1. Must not have any screening laboratory abnormalities or any other clinically significant abnormalities during screening that would render a participant unsuitable for inclusion.\n2. Must not have been treated with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer.\n3. Concurrent diagnosis of any other type of recurrent angioedema, including idiopathic angioedema or HAE with normal C1-INH (HAE-nC1-INH or Type III).\n\nNote: Other protocol-specified inclusion\u002Fexclusion criteria may apply.","11 Years",{"count":102,"type":21},20,[24],"The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).",[106],"Hereditary Angioedema (HAE)","2026-05-27",{"date":109,"type":34},"2026-05-28",{"date":111,"type":34},"2026-04-21",{"date":113,"type":21},"2029-06",{"name":40,"class":41},10,{"id":117,"slug":118,"hasResults":11,"nctId":119,"briefTitle":120,"officialTitle":121,"acronym":122,"eligibilityCriteria":123,"healthyVolunteers":11,"sex":16,"minAge":124,"maxAge":125,"enrollmentInfo":126,"targetDuration":4,"studyType":22,"phases":127,"briefSummary":128,"conditions":129,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":131,"lastUpdatePostDateStruct":132,"startDateStruct":134,"completionDateStruct":136,"leadSponsor":138,"locationsCount":139},"100375208","phase-1-study-to-evaluate-the-safety-tolerability-and-pharmacokinetics-of-ion464-administered-to-adults-with-multiple-system-atrophy-horizon-100375208","NCT04165486","Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)","A Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of ION464 Administered Intrathecally to Adults With Multiple System Atrophy","HORIZON","Key Inclusion Criteria:\n\n* Screening single-photon emission computed tomography (SPECT) with DaTscan™ (ioflupane I123 injection) results demonstrating loss (whether symmetric or asymmetric) of dopamine nerve terminals in the striatum consistent with neurodegenerative parkinsonism, as assessed with qualitative, visual read.\n* Diagnosed with probable or possible MSA, either parkinsonian-type (MSA-P) or cerebellar-type (MSA-C).\n* Must be able to walk unassisted for at least 10 meters (approximately 30 feet)\n\nKey Exclusion Criteria:\n\n* Presence of cognitive dysfunction (defined as Montreal Cognitive Assessment (MoCA) score \\\u003C25)\n* Family history of ataxia or parkinsonism and known genetic cause of ataxia or parkinsonism.\n\nNOTE: Other protocol defined Inclusion\u002F Exclusion criteria may apply.","40 Years","70 Years",{"count":42,"type":21},[54],"The primary objectives are to evaluate the safety and tolerability of multiple doses of ION464 administered via intrathecal (IT) injection (Part 1) and to evaluate the long-term safety and tolerability of ION464 (Part 2) in participants with multiple system atrophy (MSA).\n\nThe secondary objectives are to evaluate the pharmacodynamic (PD) effect of ION464 on the level of a potential biomarker of target engagement (Parts 1 and 2) and to evaluate the pharmacokinetic (PK) profile of ION464 in serum (Part 1).",[130],"Multiple System Atrophy","2026-04-24",{"date":133,"type":34},"2026-04-27",{"date":135,"type":34},"2022-07-21",{"date":137,"type":21},"2027-09",{"name":40,"class":41},15,{"id":141,"slug":142,"hasResults":11,"nctId":143,"briefTitle":144,"officialTitle":145,"acronym":4,"eligibilityCriteria":146,"healthyVolunteers":11,"sex":16,"minAge":76,"maxAge":4,"enrollmentInfo":147,"targetDuration":4,"studyType":22,"phases":149,"briefSummary":150,"conditions":151,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":153,"lastUpdatePostDateStruct":154,"startDateStruct":155,"completionDateStruct":157,"leadSponsor":159,"locationsCount":160},"100527915","phase-1-prprofile-a-study-to-assess-the-safety-tolerability-pharmacokinetics-and-pharmacodynamics-of-ion717-100527915","NCT06153966","PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717","A Phase 1\u002F2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease","Key Inclusion Criteria\n\n* A confirmed diagnosis of probable or definite prion disease.\n* Early-stage prion disease at the time of Screening.\n* Willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits.\n* Patients must have a caregiver who is ≥ 18 years old and who is able and willing to facilitate the patient's involvement, to the best of their ability, for the duration of the trial; caregivers must also be able and willing to provide information about themselves and the patient for the duration of the trial.\n* Aged ≥ 18 at the time of informed consent.\n\nKey Exclusion Criteria\n\n* Clinically significant abnormalities in medical history, laboratory tests or physical examination that would render a patient unsuitable for inclusion.\n* Any contraindication or unwillingness to undergo an MRI.\n* Obstructive hydrocephalus, presence of a functional ventriculoperitoneal shunt for the drainage of cerebrospinal fluid (CSF) or an implanted central nervous system (CNS) catheter.\n* Known brain or spinal disease that would interfere with the LP process, CSF circulation or safety assessment.\n* Have any other condition, which, in the opinion of the Investigator would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study.",{"count":148,"type":21},76,[54,55],"The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.",[152],"Prion Disease","2026-04-23",{"date":131,"type":34},{"date":156,"type":34},"2024-01-04",{"date":158,"type":21},"2030-06",{"name":40,"class":41},13,{"id":162,"slug":163,"hasResults":11,"nctId":164,"briefTitle":165,"officialTitle":166,"acronym":4,"eligibilityCriteria":167,"healthyVolunteers":11,"sex":168,"minAge":169,"maxAge":170,"enrollmentInfo":171,"targetDuration":4,"studyType":172,"phases":4,"briefSummary":173,"conditions":174,"keywords":176,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":178,"lastUpdatePostDateStruct":179,"startDateStruct":181,"completionDateStruct":183,"leadSponsor":185,"locationsCount":186},"100489959","rocket-study-a-study-to-characterize-biomarkers-and-disease-progression-in-participants-with-pelizaeus-merzbacher-disease-100489959","NCT05659901","Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease","Integrated Prospective and Retrospective Observational Study to Characterize Biomarkers and Disease Progression in Patients With Pelizaeus-Merzbacher Disease","Inclusion Criteria:\n\n1. Participant has a parent or caregiver capable of providing informed consent (signed and dated) and able to attend all scheduled study visits and provide feedback regarding the participant's symptoms and performance as described in the protocol and be able to comply with all study requirements\n2. Participant has a diagnosis of Pelizaeus-Merzbacher Disease with genetic confirmation of PLP1 duplication\n3. Male, 6 months-17 years old, inclusive, at the time of informed consent and phenotype consistent with classic PMD\n4. No contraindications for lumbar punctures (LPs), blood draws, neuroimaging, sedation (if necessary) or other study procedures\n\nExclusion Criteria:\n\n1. Clinically significant abnormalities in medical history or physical examination\n2. \\> 2 copies of the PLP1 gene\n3. Have any other conditions, which, in the opinion of the investigator would make the participant unsuitable for inclusion, or could interfere with the participant taking part in or completing the study","MALE","6 Months","17 Years",{"count":52,"type":21},"OBSERVATIONAL","The purpose of the study is to prospectively assess longitudinal changes in proteolipid protein 1 (PLP1) protein, disease-related biomarkers in cerebral spinal fluid (CSF) and blood, neuroimaging parameters relevant to Pelizaeus-Merzbacher disease (PMD) and longitudinal changes in performance on clinical, participant, and caregiver-reported outcome assessments to inform the development of therapies for PMD.",[175],"Pelizaeus-Merzbacher Disease",[177],"PMD","2026-04-16",{"date":180,"type":34},"2026-04-17",{"date":182,"type":34},"2022-10-03",{"date":184,"type":21},"2029-03",{"name":40,"class":41},9,{"id":188,"slug":189,"hasResults":11,"nctId":190,"briefTitle":191,"officialTitle":192,"acronym":4,"eligibilityCriteria":193,"healthyVolunteers":11,"sex":168,"minAge":17,"maxAge":194,"enrollmentInfo":195,"targetDuration":4,"studyType":22,"phases":197,"briefSummary":198,"conditions":199,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":201,"lastUpdatePostDateStruct":202,"startDateStruct":204,"completionDateStruct":206,"leadSponsor":207,"locationsCount":208},"100549159","phase-1-attune-a-study-to-evaluate-the-safety-tolerability-pharmacokinetics-and-pharmacodynamics-of-intrathecally-administered-ion440-in-participants-with-methyl-cpg-binding-protein-2-mecp2-duplication-syndrome-mds-100549159","NCT06430385","ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)","A Phase 1-2, Double-Blind, Sham-Controlled Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Patients With MECP2 Duplication Syndrome","Key Inclusion criteria for Part 1:\n\n1. Males aged ≥ 2 to ≤ 65 years, depending on specific cohort and group, at the time of informed consent.\n\n   1. Group A: ≥ 8 to ≤ 65 years old\n   2. Group B: 2 to 7 years old, inclusive\n2. Participant has at least one parent or caregiver ≥ 18 years old capable of providing informed consent and able to comply with all study requirements and activities.\n3. Participant has a documented diagnosis of MDS with genetic confirmation of MECP2 duplication.\n4. Is currently receiving stable doses of concomitant medications for at least 1 month prior to screening.\n5. Able to complete all study procedures, measurements and visits to support primary and secondary endpoints, in the opinion of the Investigator.\n\nKey Exclusion criteria for Part 1:\n\n1. Documented diagnosis of severe MECP2 duplications including terminal duplication and\u002For translocation or MECP2 triplication OR clinical features associated with severe variant structure including (a) onset of seizures prior to age 5 (for those aged 5 and above at signing of ICF), (b) oxygen dependence, (c) microcephaly, IF MECP2 genetic structure information is unavailable.\n2. Clinically significant vital sign or ECG abnormality at Screening\n3. Known brain or spinal disease that would interfere with the LP procedure, or CSF circulation or presence of other factors would affect the safety of the LP procedure.\n4. Has any concomitant disease or condition or circumstance, or any finding at Screening that, in the opinion of the Investigator, makes the participant unsuitable for enrollment or that could interfere with the conduct of the study or that would pose an unacceptable risk to the participant in this study.\n5. Treatment with an investigational drug, biological agent, or device within 30 days of Screening, or 5 half-lives of investigational agent, whichever is longer.\n6. Previous treatment with an oligonucleotide (including siRNA) within 4 months of Screening if single dose received, or within 12 months of Screening if multiple doses received (this exclusion does not apply to vaccines - both mRNA and viral vector vaccines are allowed including COVID-19). For centrally administered ASOs, a minimum of 12 months washout is required irrespective of the number of doses received.\n7. Currently enrolled in a clinical trial of an investigational agent or device or has used any investigational agent or device within 5 half-lives of investigational agent, whichever is longer.\n8. Has a history of gene therapy or cell transplantation or any other experimental brain surgery.\n9. Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to Baseline (Day 1).\n10. Has experienced Status Epilepticus in the past 6 months.\n\nKey Inclusion criteria for Part 2:\n\n1. Participants in ION440-CS1, Part 1\u002FMAD who received at least one dose of Study Drug \u002FSham in Part 1\u002FMAD, missed no more than 1 study visit, and attended the Follow Up visit (Visit 6).\n2. All inclusion criteria in Part 1\u002FMAD apply (participants will not be required to undergo new Screening bloodwork).\n\nKey Exclusion criteria for Part 2:\n\n1\\. Has developed any concomitant disease (e.g., gastrointestinal, renal, hepatic, endocrine, respiratory, or cardiovascular system disease) or condition or circumstance, or any finding during Part 1\u002FMAD that, in the opinion of the Investigator, makes the participant unsuitable for continued treatment (e.g., could interfere with the conduct of the study or that would pose an unacceptable risk to the participant in this study).","65 Years",{"count":196,"type":21},48,[54,55],"The primary purpose of this study is to evaluate the safety and tolerability of ION440.",[200],"Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome","2026-03-20",{"date":203,"type":34},"2026-03-24",{"date":205,"type":34},"2024-10-21",{"date":38,"type":21},{"name":40,"class":41},11,{"id":210,"slug":211,"hasResults":11,"nctId":212,"briefTitle":213,"officialTitle":213,"acronym":4,"eligibilityCriteria":214,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":215,"enrollmentInfo":4,"targetDuration":4,"studyType":216,"phases":4,"briefSummary":217,"conditions":218,"keywords":220,"overallStatus":224,"whyStopped":4,"lastUpdateSubmitDate":225,"lastUpdatePostDateStruct":226,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":228,"locationsCount":4},"100630416","zilganersen-expanded-access-program-for-individuals-with-alexander-disease-100630416","NCT07487389","Zilganersen Expanded Access Program for Individuals With Alexander Disease","Key Inclusion Criteria:\n\n1. Approved drug or drugs available for treatment did not work for the patient, or the patient cannot tolerate the side effects of the FDA-approved drug or drugs for treatment of AxD.\n2. Patients who are ≥ 2 years old.\n3. Patients who have a clinical phenotype and brain imaging consistent with a diagnosis of AxD.\n4. Patients who have a documented variant in the GFAP gene.\n5. Patient resides in and is a resident of the US.\n\nKey Exclusion Criteria:\n\n1. Patients who have any medical history, physical exam findings, or clinically significant laboratory abnormalities that contraindicate performing an LP for ITB administration of zilganersen.\n2. Patients who have current obstructive hydrocephalus.\n3. Patients who have the presence of a functional ventriculoperitoneal shunt for the drainage of CSF.\n4. Patients who have any other medical history or current conditions, which, in their Treating Physician's opinion, would make the patient unsuitable for inclusion (e.g., active Hepatitis B virus or hepatitis C virus infection, severe hepatic or renal disease, uncontrolled acute or chronic condition, etc.).\n5. Patients who are pregnant or plan to become pregnant, or patients who are breastfeeding.\n\nOther inclusion\u002Fexclusion criteria may apply.","99 Years","EXPANDED_ACCESS","The purpose of the expanded access program (EAP) is to provide access to zilganersen for eligible individuals with Alexander disease (AxD).",[219],"Alexander Disease",[221,222,223],"Alexander disease","leukodystrophy","expanded access","AVAILABLE","2026-03-17",{"date":227,"type":34},"2026-03-23",{"name":40,"class":41},{"id":230,"slug":231,"hasResults":11,"nctId":232,"briefTitle":233,"officialTitle":234,"acronym":4,"eligibilityCriteria":235,"healthyVolunteers":11,"sex":168,"minAge":17,"maxAge":170,"enrollmentInfo":236,"targetDuration":4,"studyType":22,"phases":238,"briefSummary":239,"conditions":240,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":241,"lastUpdatePostDateStruct":242,"startDateStruct":244,"completionDateStruct":246,"leadSponsor":248,"locationsCount":249},"100527665","phase-1-orbit-study-a-study-to-evaluate-the-safety-pharmacokinetics-and-pharmacodynamics-of-intrathecally-administered-ion356-in-participants-with-pelizaeus-merzbacher-disease-pmd-100527665","NCT06150716","Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)","A Phase 1b Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION356 in Patients With Pelizaeus Merzbacher Disease","Key Inclusion Criteria\n\n1. Participant's parent or legally accepted representative can provide informed consent, attend all scheduled study visits, provide feedback regarding the participant's symptoms, and can comply with all study requirements.\n2. Diagnosis of PMD with genetic confirmation of PLP1 gene duplication.\n3. Clinical phenotype and brain imaging consistent with a diagnosis of PMD.\n4. Male between the ages of 2 and 17 years, inclusive, at the time of informed consent.\n5. Able and willing to meet all study requirements (in the opinion of the Investigator), including travel to Study Center, procedures, measurements, and visits.\n\nExclusion Criteria\n\n1. Clinically significant abnormalities in medical history, laboratory tests or physical examination.\n2. Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator.\n3. Any contraindication or unwillingness to undergo magnetic resonance imaging (MRI).\n4. Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of the investigational agent, whichever is longer.\n5. Previous treatment with an oligonucleotide (including small interfering ribonucleic acid) within 4 months of Screening if a single dose was received, or within 12 months of Screening if multiple doses were received. This exclusion does not apply to vaccines (both messenger ribonucleic acid \\[mRNA\\] and viral vector vaccines).\n6. History of gene therapy or cell transplantation, or any experimental brain surgery.\n7. Current obstructive hydrocephalus.\n8. Known brain or spinal disease or previous spinal surgery that would interfere with the lumbar puncture (LP) process, CSF circulation, or safety assessment.\n9. Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks prior to Screening or planned during the study.\n10. Have any other conditions, which, in the opinion of the Investigator, would make the participant unsuitable for inclusion, or could interfere with the participant participating in or completing the study.",{"count":237,"type":21},24,[54],"The primary purpose of this study is to evaluate the safety and tolerability of ION356.",[175],"2025-12-05",{"date":243,"type":34},"2025-12-12",{"date":245,"type":34},"2024-04-10",{"date":247,"type":21},"2028-06",{"name":40,"class":41},7,{"id":251,"slug":252,"hasResults":11,"nctId":253,"briefTitle":254,"officialTitle":255,"acronym":4,"eligibilityCriteria":256,"healthyVolunteers":11,"sex":16,"minAge":257,"maxAge":18,"enrollmentInfo":258,"targetDuration":4,"studyType":22,"phases":260,"briefSummary":261,"conditions":262,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":263,"lastUpdatePostDateStruct":264,"startDateStruct":266,"completionDateStruct":268,"leadSponsor":269,"locationsCount":208},"100449034","phase-1-halos-a-safety-tolerability-pharmacokinetics-and-pharmacodynamics-study-of-multiple-ascending-doses-of-ion582-in-participants-with-angelman-syndrome-100449034","NCT05127226","HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome","HALOS: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients With Angelman Syndrome","Key Inclusion Criteria:\n\n1. Participant has a documented and certified diagnosis of Angelman syndrome (AS) (ubiquitin-protein ligase E3A \\[UBE3A\\] deletion or UBE3A mutation)\n2. Male or female between the ages of 0-50 years of age, with signed informed consent from parent(s) or legal guardian(s)\n3. Currently receiving stable standard of care treatments such as, stable doses of anti-epileptic medication, behavioral management medications, sleep medications, gabapentin, cannabidiol, and including special diets, supplements or nutritional support for at least 3 months prior to first dose.\n4. Follow good study practice and not participate in the sharing of personal or study information on social media platforms, such as any website or social media site (e.g., Facebook, Instagram, Twitter, YouTube, etc.) until notified that the study is completed.\n\nKey Exclusion Criteria:\n\n1. Has documented molecular AS confirmation of paternal uniparental disomy (UPD) or imprinting defect (ID).\n2. Any clinically significant (CS) cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in the judgment of the Investigator, will pose a safety risk, will make the patient unsuitable for participation in, and\u002For unable to complete the study procedures. Has poorly controlled seizures as determined by the Investigator or has documented Status Epilepticus in the past 6 months that could pose a safety risk while on study.\n3. Known bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture. Previous treatment with an oligonucleotide (including small interfering ribonucleic acid, antisense oligonucleotide \\[ASOs\\]). COVID-19 vaccinations are allowed.\n4. Any prior use of gene therapy. Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion or could interfere with the participant taking part in or completing the study.","0 Years",{"count":259,"type":21},70,[54,55],"The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.",[27],"2025-11-12",{"date":265,"type":34},"2025-11-14",{"date":267,"type":34},"2021-12-22",{"date":184,"type":21},{"name":40,"class":41},{"id":271,"slug":272,"hasResults":11,"nctId":273,"briefTitle":274,"officialTitle":275,"acronym":4,"eligibilityCriteria":276,"healthyVolunteers":11,"sex":16,"minAge":76,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":216,"phases":4,"briefSummary":277,"conditions":278,"keywords":280,"overallStatus":224,"whyStopped":4,"lastUpdateSubmitDate":285,"lastUpdatePostDateStruct":286,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":288,"locationsCount":4},"100548012","donidalorsen-expanded-access-program-for-patients-with-hereditary-angioedema-100548012","NCT06415448","Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema","Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema (HAE)","Inclusion Criteria:\n\n* Patients in the ISIS 721744-CS3 (Open-Label Extension \\[OLE\\] Study) who have completed 205 weeks of treatment.\n* Female patients of childbearing potential, and male patients with partners of childbearing potential must be willing to use acceptable contraception, or refrain from sexual activity.\n\nExclusion Criteria:\n\n* Any patient who is pregnant or plans to become pregnant.\n* Any patient who was withdrawn from the ISIS 721744-CS3 OLE study due to a serious adverse event (SAE) related to donidalorsen therapy or who voluntarily withdrew prior to 205 weeks of treatment.","The purpose of the Expanded Access Program is to provide pre-approval access of donidalorsen to eligible patients with Hereditary Angioedema (HAE) who complete the ISIS 721744-CS3 clinical trial.",[279],"Hereditary Angioedema",[281,282,283,284],"HAE","IONIS PKK-LRx","Donidalorsen","ISIS 721744","2025-07-09",{"date":287,"type":34},"2025-07-11",{"name":40,"class":41},""]