Spinal muscular atrophy trials in United States

In United States 17 Also abroad 68

17 trials · From ClinicalTrials.gov, checked 11 November 2026. Ask the study team whether a trial fits you.

No phaseTaking people
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
Where
Austin, Baltimore, Boston, Charlottesville, Chicago, Cleveland, Grand Rapids, Hershey, Houston, New York, Norfolk, Orange, Orlando, Palo Alto, Philadelphia, Phoenix, San Diego
Age
3 years and over
People
90 worldwide
Run by
Alcyone Therapeutics, Inc
See details and how to joinNCT05866419
Phase 4Taking people
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
Where
Boston, Columbus, Little Rock, New York, Philadelphia, Stanford
Age
2 years to 100 years
People
36
Run by
Novartis Pharmaceuticals
See details and how to joinNCT07448610
Phase 3Taking people
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)
Where
Chicago, Flower Mound, Norfolk
Age
0 days to 42 days
People
30 worldwide
Run by
Biogen
See details and how to joinNCT07221669 · EU 2025-524054-34-00
No phaseTaking people
HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness
Where
New York
Age
5 years to 17 years
People
20
Run by
Teachers College, Columbia University
See details and how to joinNCT07488338
No phaseTaking people
A Registry of Cases of Spinal Muscular Atrophy
Where
Memphis
People
30
Run by
St. Jude Children's Research Hospital
See details and how to joinNCT07805278
Phase 3Taking people
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
Where
Flower Mound
Age
0 days to 7 months
People
42 worldwide
Run by
Biogen
See details and how to joinNCT07444450 · EU 2025-523857-32-00
No phaseTaking people
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
Where
New York
Age
12 years and over
People
45
Run by
Columbia University
See details and how to joinNCT06955897
Phase 2Taking people
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
Where
Baltimore, Boston, Chicago, Columbia, Flower Mound, Hartford, Hillsborough, Houston, Iowa City, Kissimmee, Little Rock, New York, Norfolk, Philadelphia, San Diego, Stanford
Age
5 years to 17 years
People
60 worldwide
Run by
argenx
See details and how to joinNCT07287982 · EU 2025-523496-32-00
Phase 2Taking people
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Where
Atlanta, Aurora, Dallas, Flower Mound, Fort Worth, Grand Rapids, Houston, Iowa City, Orange, Palo Alto, Philadelphia, Phoenix
Age
Up to 2 years
People
52 worldwide
Run by
Scholar Rock, Inc.
See details and how to joinNCT07047144
Phase 3Taking people
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam
Where
Norfolk, Palo Alto
Age
15 years to 60 years
People
90 worldwide
Run by
Biogen
See details and how to joinNCT07444476
Phase 1Taking people
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)
Where
Boston, Charlottesville, Chicago, Grand Rapids, Hershey, Houston, Norfolk, Orange, Orlando, Palo Alto, Philadelphia, Phoenix
Age
3 years and over
People
78 worldwide
Run by
Biogen
See details and how to joinNCT06555419 · EU 2024-514239-21-00
No phaseTaking people
Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
Where
Memphis
Age
5 years to 20 years
People
24
Run by
St. Jude Children's Research Hospital
See details and how to joinNCT06532474
No phaseTaking people
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
Where
Boston, New York, Palo Alto
Age
5 years and over
People
106
Run by
Columbia University
See details and how to joinNCT06839469
No phaseTaking people
Gait and Bone Health in SMA
Where
New York
Age
8 years to 50 years
People
22
Run by
Jacqueline Montes
See details and how to joinNCT07400198
No phaseTaking people
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
Where
Boston, Cambridge, New York, Palo Alto
Age
18 years to 54 years
People
20 worldwide
Run by
Biogen
See details and how to joinNCT05789758
No phaseTaking people
Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System
Where
Washinton
Age
18 years and over
People
200
Run by
Medstar Health Research Institute
See details and how to joinNCT07223489
No phaseTaking people
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Where
Akron, Aurora, Austin, Charlottesville, Cincinnati, Cleveland, Columbia, Columbus, Dallas, Durham, Farmington, Fort Myers, Fort Worth, Greenville, Hershey, Houston, Indianapolis, Iowa City, Kansas City, Little Rock, Loma Linda, Los Angeles, Louisville, Madera, Madison, Memphis, Miami, Milwaukee, Minneapolis, Morristown, New Haven, Norfolk, Oklahoma City, Orlando, Palmetto Bay, Phoenix, Pittsburgh, Portland, Richmond, Rochester, Sacramento, Salt Lake City, San Antonio, San Diego, Seattle, St Louis, Stony Brook, Tacoma
People
700 worldwide
Run by
Novartis Pharmaceuticals
See details and how to joinNCT04174157