Spinal muscular atrophy: open trials in France

13 open trials have a site in France. Registry data; only the trial team decides who can take part.

2026-527323-10-00
AAV9-ASAH1 intracisterna magna Gene Therapy (GNT0009) in Patients with Spinal Muscular Atrophy with Progressive Myoclonic Epilepsy (SMA - PME)
Trial Treatment · Phase 1/2 · Genethon
Bicetre Hospital, Le Kremlin Bicetre Cedex · Trousseau Hospital, Paris
Registry updated 2026-11-09
NCT07321977
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
Trial Treatment · Institut de Myologie, France
Centre d'Exploration et d'Évaluation Neuromusculaire, Paris
Registry updated 2026-11-03
2024-520130-29-00
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Trial Treatment · Phase 2 · Scholar Rock Inc.
Centre Hospitalier Universitaire De Lille, Lille Cedex · Association Institut De Myologie, Paris
Registry updated 2026-10-29
NCT07221669
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)
Trial Treatment · Phase 3 · Biogen
Assistance Publique Hopitaux De Paris, Garches · Centre Hospitalier Regional De Marseille, Marseille · Centre Hospitalier Universitaire de Toulouse, Toulouse
Registry updated 2026-10-23
NCT06147414
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Research · Assistance Publique - Hôpitaux de Paris
This trial only enrols pregnant participants
Hôpital Cochin, Maternité Port-Royal, service de Gynécologie obstétrique, Paris
Registry updated 2026-10-21
NCT07444450
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
Trial Treatment · Phase 3 · Biogen
Pellegrin Hospital, Bordeaux · Centre Hospitalier Régional et Universitaire de Brest, Brest · Centre Hospitalier Universitaire De Montpellier, Montpellier Cedex 5 · Hopital Necker Enfants Malades, Paris · Les Hopitaux Universitaires De Strasbourg, Strasbourg Cedex 2 · Hopital Des Enfants, Toulouse Cedex 9
Registry updated 2026-10-15
NCT07047144
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Trial Treatment · Phase 2 · Scholar Rock, Inc.
Hopital Trousseau - I-Motion, Paris
Registry updated 2026-10-02
NCT06555419
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)
Trial Treatment · Phase 1 · Biogen
Assistance Publique Hopitaux De Paris, Garches
Registry updated 2026-09-03
NCT06562283
Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy
Trial Treatment · Centre Hospitalier Universitaire de Saint Etienne
HFME - HospiceS Civils De Lyon, Lyon · HCL - Hôpital Croix Rousse, Lyon · Aphp - Hopital Pitie Salpetriere, Paris · Unités de Myologie et de Médecine du Sport, Saint-Etienne
Registry updated 2026-05-06
NCT06321965
Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
Trial Treatment · Hospices Civils de Lyon
CHRU of Brest, Brest · Pediatric Rehabilitation Service - L'Escale Mother and Child Hospital, Bron · Pediatric Neurology and Resuscitation Raymond-Poincare Hospital, Garche · Pediatric Neurology Swynghedauw Hospital, Lille · Marseille University Hospital - Timone Hospital Department of Pediatric Neurology - Specialized Pediatrics and Child Medicine, Marseille · I-Motion Pediatric Clinical Trial Platform Armand Trousseau Hospital, Paris · Hautepierre Hospital - Mother and Child Hospital, Strasbourg · Department of Pediatrics - Neurology and Infectious Diseases Toulouse University Hospital - Children's Hospital, Toulouse
Registry updated 2026-01-30
NCT07332702
Long Read Analysis in Spinal Muscular Atrophy - LOREASI
Trial Treatment · University Hospital, Rouen
CHU Rouen, Rouen
Registry updated 2026-01-12
NCT06152302
Test of Aquatic Mobility of SMA Infants
Trial Treatment · Assistance Publique - Hôpitaux de Paris
Infantile neuromuscular unity - Department of paediatric neurological care and intentive care unity, Raymond Poincaré Hospital - APHP, Garches
Registry updated 2025-11-21
NCT04177134
French Register of Patients With Spinal Muscular Atrophy
Research · Assistance Publique - Hôpitaux de Paris
Unité neuromusculaire, Service de Neurologie et Réanimation Pédiatrique, (French neuromuscular reference network (FILNEMUS), Hôpital Raymond Poincaré,, Garches
Registry updated 2025-04-06

All Spinal muscular atrophy trials and the map

This page is not medical advice. Talk to your doctor before contacting a trial.

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