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ClinicalTrials.gov public records Last synced Oct 17, 2026, 8:54 AM EDT

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Showing 1–24 of 32 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: nusinersen Clear all
Recruiting No phase listed Observational

A Registry of Cases of Spinal Muscular Atrophy

NCT07805278
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
30 participants
Timeline
2026 – 2033
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 17, 2026, 8:54 AM EDT
Completed Phase 1 Interventional

An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)

NCT01780246
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 15 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
4
States / cities
Boston, Massachusetts • New York, New York • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Oct 17, 2026, 8:54 AM EDT
Completed No phase listed Observational

Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy

NCT05518773
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Observational
Other
Lead sponsor
Columbia University
Other
Eligibility
8 Years to 55 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 3, 2025 · Synced Oct 17, 2026, 8:54 AM EDT
Completed Phase 3 Interventional Results available

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)

NCT02292537
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen, Sham procedure
Drug · Procedure
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 12 Years
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
11
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2021 · Synced Oct 17, 2026, 8:54 AM EDT
Completed Phase 3 Interventional Results available

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCT05386680
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Norfolk, Virginia • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 17, 2026, 8:54 AM EDT
Completed No phase listed Observational

Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States

NCT06178393
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Up to 5 Years
Enrollment
162 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
Bannockburn, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 21, 2023 · Synced Oct 17, 2026, 8:54 AM EDT
Completed Phase 4 Interventional

A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)

NCT04488133
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Months to 36 Months
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
9
States / cities
Little Rock, Arkansas • Palo Alto, California • Aurora, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Oct 17, 2026, 8:54 AM EDT
Recruiting Phase 2 Interventional

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

NCT07047144
View directory record Official record
Conditions
Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3, Neuromuscular Manifestations, Anti-myostatin
Interventions
Apitegromab, Nusinersen, Risdiplam
Drug
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
Up to 2 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
12
States / cities
Phoenix, Arizona • Orange, California • Palo Alto, California + 9 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 17, 2026, 8:54 AM EDT
Completed Phase 1Phase 2 Interventional Results available

An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy

NCT01703988
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 15 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
4
States / cities
Boston, Massachusetts • New York, New York • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2021 · Synced Oct 17, 2026, 8:54 AM EDT
Completed Phase 1 Interventional

An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)

NCT02052791
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
Not listed
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
4
States / cities
Boston, Massachusetts • New York, New York • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Oct 17, 2026, 8:54 AM EDT
Completed Phase 2 Interventional Results available

A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)

NCT01839656
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
21 Days to 210 Days
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
3
States / cities
Stanford, California • Orlando, Florida • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 17, 2021 · Synced Oct 17, 2026, 8:54 AM EDT
Completed No phase listed Observational

Adults With SMA Treated With Nusinersen

NCT04591678
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Ohio State University
Other
Eligibility
18 Years to 60 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 22, 2021 · Synced Oct 17, 2026, 8:54 AM EDT
Completed No phase listed Observational

Effect of Nusinersen on Adults With Spinal Muscular Atrophy

NCT03878030
View directory record Official record
Conditions
Adult Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Northwell Health
Other
Eligibility
18 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Great Neck, New York
Source: ClinicalTrials.gov public record
Updated Mar 20, 2023 · Synced Oct 17, 2026, 8:54 AM EDT
Completed No phase listed Observational

A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies

NCT07378943
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
4,114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 30, 2026 · Synced Oct 17, 2026, 8:54 AM EDT
Completed Phase 3 Interventional Results available

A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies

NCT02594124
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
Not listed
Enrollment
292 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
18
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 15 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2024 · Synced Oct 17, 2026, 8:54 AM EDT
Recruiting No phase listed Observational

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

NCT05789758
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
No Intervention
Other
Lead sponsor
Biogen
Industry
Eligibility
18 Years to 54 Years · Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2033
U.S. locations
4
States / cities
Palo Alto, California • Boston, Massachusetts • Cambridge, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Oct 17, 2026, 8:54 AM EDT
Terminated Phase 3 Interventional Results available

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy

NCT02193074
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen, Sham procedure
Drug · Procedure
Lead sponsor
Biogen
Industry
Eligibility
Up to 210 Days
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
12
States / cities
Los Angeles, California • Aurora, Colorado • Orlando, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2021 · Synced Oct 17, 2026, 8:54 AM EDT
Recruiting No phase listed Observational

Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies

NCT06532474
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
5 Years to 20 Years
Enrollment
24 participants
Timeline
2025 – 2028
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Oct 17, 2026, 8:54 AM EDT
Not listed Phase 4 Interventional

Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

NCT05522361
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Risdiplam
Drug
Lead sponsor
Clinic for Special Children
Other
Eligibility
2 Years to 35 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Strasburg, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Oct 17, 2026, 8:54 AM EDT
Terminated Phase 2 Interventional Results available

A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).

NCT02462759
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen, Sham Procedure
Drug · Procedure
Lead sponsor
Biogen
Industry
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
6
States / cities
Los Angeles, California • Hartford, Connecticut • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2021 · Synced Oct 17, 2026, 8:54 AM EDT
Completed Phase 1 Interventional

An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)

NCT01494701
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 14 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
4
States / cities
Boston, Massachusetts • New York, New York • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 18, 2021 · Synced Oct 17, 2026, 8:54 AM EDT
Recruiting No phase listed Observational

Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA

NCT06955897
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Observational
Other
Lead sponsor
Columbia University
Other
Eligibility
12 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 17, 2026, 8:54 AM EDT
Recruiting Phase 1 Interventional

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)

NCT06555419
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Nusinersen, ThecaFlex DRx System
Drug · Device
Lead sponsor
Biogen
Industry
Eligibility
3 Years and older
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
12
States / cities
Phoenix, Arizona • Orange, California • Palo Alto, California + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 17, 2026, 8:54 AM EDT
Completed Phase 3 Interventional

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

NCT05156320
View directory record Official record
Conditions
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
Interventions
Apitegromab, Placebo
Drug
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
2 Years to 21 Years
Enrollment
188 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
26
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Oct 17, 2026, 8:54 AM EDT