NCT06321965Recruiting
Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
Source: ClinicalTrials.gov · Last updated 30 Jan 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Interventional
- Enrollment
- 60 (estimated)
- First submitted
- 6 Feb 2024
- Start date
- Jul 2024
- Primary completion date
- Jan 2029
- Lead sponsor
- Hospices Civils de Lyon
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT06321965
What you commit to
- Study type: Interventional
- Planned participants: 60
- From Jul 2024 to the primary completion date, Jan 2029
- 8 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Other condition likely to interfere significantly with ASI assessment and clearly unrelated to the disease
- Other associated neurological disease
Other
- Current pregnancy or breast-feeding (a pregnancy test will also be performed at inclusion).
- Please note that patients with a specific contraindication to MRI (i.e. metallic foreign body, claustrophobia and other reasons determined by the investigators) will be allowed to participate in the study, but MRI will not be performed.
Who the study is looking for
- Genetically confirmed infantile or juvenile spinal muscular atrophy
- Treated with a therapy that restores SMN protein expression (e.g. nusinersen, risdiplam, onasemnogene abeparvovec)
- Aged 0 to 15 years inclusive
- Informed consent signed by both parent(s)/legal guardian(s) and patient's assent
- Affiliated or beneficiary of a health insurance plan\*. \* for inclusion in France
Age in the registry: 0 years to 16 years · Sex: All · Does not accept healthy volunteers
Sites (8)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| CHRU of Brest Coordinator of this site? Confirm its status | Brest | France | Not yet recruiting |
| Pediatric Rehabilitation Service - L'Escale Mother and Child Hospital Coordinator of this site? Confirm its status | Bron | France | Recruiting |
| Pediatric Neurology and Resuscitation Raymond-Poincare Hospital Coordinator of this site? Confirm its status | Garche | France | Not yet recruiting |
| Pediatric Neurology Swynghedauw Hospital Coordinator of this site? Confirm its status | Lille | France | Not yet recruiting |
| Marseille University Hospital - Timone Hospital Department of Pediatric Neurology - Specialized Pediatrics and Child Medicine Coordinator of this site? Confirm its status | Marseille | France | Not yet recruiting |
| I-Motion Pediatric Clinical Trial Platform Armand Trousseau Hospital Coordinator of this site? Confirm its status | Paris | France | Not yet recruiting |
| Hautepierre Hospital - Mother and Child Hospital Coordinator of this site? Confirm its status | Strasbourg | France | Not yet recruiting |
| Department of Pediatrics - Neurology and Infectious Diseases Toulouse University Hospital - Children's Hospital Coordinator of this site? Confirm its status | Toulouse | France | Not yet recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.