Spinal muscular atrophy trials in France

In France 13 Also abroad 68

13 trials · From ClinicalTrials.gov and EU CTIS (euclinicaltrials.eu), checked 12 November 2026. Ask the study team whether a trial fits you.

Phase 1/2Not yet taking people in France
AAV9-ASAH1 intracisterna magna Gene Therapy (GNT0009) in Patients with Spinal Muscular Atrophy with Progressive Myoclonic Epilepsy (SMA - PME)
Where
Le Kremlin Bicetre Cedex, Paris
Age
Up to 17 years
People
3
Run by
Genethon
See details and how to join2026-527323-10-00 · EU 2026-527323-10-00
No phaseTaking people
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
Where
Paris
Age
18 years to 65 years
People
30
Run by
Institut de Myologie, France
See details and how to joinNCT07321977
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
Lille Cedex, Paris
Age
Up to 17 years
People
30 worldwide
Run by
Scholar Rock Inc.
See details and how to join2024-520130-29-00 · EU 2024-520130-29-00
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
Garches, Marseille, Toulouse
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
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Where
Paris
Age
18 years and over
People
550
Run by
Assistance Publique - Hôpitaux de Paris
See details and how to joinNCT06147414
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
Bordeaux, Brest, Montpellier Cedex 5, Paris, Strasbourg Cedex 2, Toulouse Cedex 9
Age
0 days to 7 months
People
42 worldwide
Run by
Biogen
See details and how to joinNCT07444450 · EU 2025-523857-32-00
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
Paris
Age
Up to 2 years
People
52 worldwide
Run by
Scholar Rock, Inc.
See details and how to joinNCT07047144
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
Garches
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
Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy
Where
Lyon, Paris, Saint-Etienne
Age
6 years and over
People
80
Run by
Centre Hospitalier Universitaire de Saint Etienne
See details and how to joinNCT06562283
No phaseTaking people
Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
Where
Brest, Bron, Garche, Lille, Marseille, Paris, Strasbourg, Toulouse
Age
0 years to 16 years
People
60
Run by
Hospices Civils de Lyon
See details and how to joinNCT06321965
No phaseTaking people
Long Read Analysis in Spinal Muscular Atrophy - LOREASI
Where
Rouen
Age
18 years and over
People
27
Run by
University Hospital, Rouen
See details and how to joinNCT07332702
No phaseTaking people
Test of Aquatic Mobility of SMA Infants
Where
Garches
Age
Up to 12 months
People
15
Run by
Assistance Publique - Hôpitaux de Paris
See details and how to joinNCT06152302
No phaseTaking people
French Register of Patients With Spinal Muscular Atrophy
Where
Garches
Age
0 days and over
People
1,000
Run by
Assistance Publique - Hôpitaux de Paris
See details and how to joinNCT04177134