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Study identifier: NCT06321965 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy

Condition: Spinal Muscular Atrophy  ·  Sponsor: Hospices Civils de Lyon

PhaseNA
Planned participants60
Who can joinAll sexes, 0 Years to 16 Years
Healthy volunteersNo

About this study

With the advent of new treatments for ASI, new phenotypes are emerging. The investigators propose to describe these new phenotypes by prospectively following children with ASI of all types treated with TRS and aged under 16 for 2 years. The investigators also propose to evaluate potential assessment tools to determine whether they are relevant for monitoring this population, either routinely or for future clinical trials. The investigators also aim to collect the total costs associated with ASI in order to propose a first prospective medico-economic study in France.

This description comes directly from the study's public registry record.

Talk to the study team

Laure LE GOFF, PI  ·  04.72.12.95.04  ·  laure.le-goff@chu-lyon.fr

Tiphanie GINHOUX  ·  04.27.85.77.23  ·  Tiphanie.ginhoux01@chu-lyon.fr

Always discuss trial participation with your own doctor first.

Locations (8)

Pediatric Rehabilitation Service - L'Escale Mother and Child HospitalBron, Rhone, FranceRecruiting
CHRU of BrestBrest, FranceNot Yet Recruiting
Pediatric Neurology and Resuscitation Raymond-Poincare HospitalGarche, FranceNot Yet Recruiting
Pediatric Neurology Swynghedauw HospitalLille, FranceNot Yet Recruiting
Marseille University Hospital - Timone Hospital Department of Pediatric Neurology - Specialized Pediatrics and Child MedicineMarseille, FranceNot Yet Recruiting
I-Motion Pediatric Clinical Trial Platform Armand Trousseau HospitalParis, FranceNot Yet Recruiting
Hautepierre Hospital - Mother and Child HospitalStrasbourg, FranceNot Yet Recruiting
Department of Pediatrics - Neurology and Infectious Diseases Toulouse University Hospital - Children's HospitalToulouse, FranceNot Yet Recruiting

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Source record: clinicaltrials.gov/study/NCT06321965