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ClinicalTrials.gov public records Last synced Sep 17, 2026, 11:38 AM EDT

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Showing 1–20 of 20 matching trials from the live ClinicalTrials.gov search.
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Filters: Intervention: Nusinersen Treatments Clear all
Active, not recruiting No phase listed Observational

Spinraza in Adult Spinal Muscular Atrophy

NCT03709784
View directory record Official record
Conditions
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type 3
Interventions
Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care, One time survey
Drug · Other
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 70 Years
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
10
States / cities
Phoenix, Arizona • Washington D.C., District of Columbia • Baltimore, Maryland + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 19, 2024 · Synced Sep 17, 2026, 11:38 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 20, 2023 · Synced Sep 17, 2026, 11:38 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
13
States / cities
Phoenix, Arizona • Orange, California • Palo Alto, California + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2026 · Synced Sep 17, 2026, 11:38 AM EDT
Completed Phase 3 Interventional

A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)

NCT04729907
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years and older
Enrollment
115 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
7
States / cities
Sacramento, California • Aurora, Colorado • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Sep 17, 2026, 11:38 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 2, 2026 · Synced Sep 17, 2026, 11:38 AM EDT
Completed Phase 3 Interventional Results available

Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

NCT04089566
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
7 Days and older
Enrollment
145 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
7
States / cities
Palo Alto, California • Aurora, Colorado • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2025 · Synced Sep 17, 2026, 11:38 AM EDT
Recruiting Not applicable Interventional

Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy

NCT05866419
View directory record Official record
Conditions
Spinal Muscular Atrophy, Spine Deformity, Scoliosis
Interventions
ThecaFlex DRx System
Device
Lead sponsor
Alcyone Therapeutics, Inc
Industry
Eligibility
3 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
15
States / cities
Phoenix, Arizona • Orange, California • Palo Alto, California + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Sep 17, 2026, 11:38 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 8, 2026 · Synced Sep 17, 2026, 11:38 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 13, 2025 · Synced Sep 17, 2026, 11:38 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 21, 2021 · Synced Sep 17, 2026, 11:38 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 12, 2026 · Synced Sep 17, 2026, 11:38 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 16, 2021 · Synced Sep 17, 2026, 11:38 AM EDT
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 7, 2026 · Synced Sep 17, 2026, 11:38 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 8, 2026 · Synced Sep 17, 2026, 11:38 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 29, 2026 · Synced Sep 17, 2026, 11:38 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 6, 2024 · Synced Sep 17, 2026, 11:38 AM EDT
Withdrawn Early Phase 1 Interventional

Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments

NCT04050852
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen Treatments
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
5 Years to 21 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 28, 2021 · Synced Sep 17, 2026, 11:38 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 2, 2025 · Synced Sep 17, 2026, 11:38 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 21, 2026 · Synced Sep 17, 2026, 11:38 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)

NCT05067790
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
15 Years to 50 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
11
States / cities
Phoenix, Arizona • Palo Alto, California • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated May 21, 2026 · Synced Sep 17, 2026, 11:38 AM EDT