Monitoring to the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA®
Condition(s) studied
Investigational drug(s) / intervention(s)
Spinraza intrathecal injection: The aim is to monitor the evolution of the Motor Function Measure-32 for SMA type II adult patients treated with SPINRAZA® (Nusinersen).
Study summary
SPINRAZA® (Nusinersen) is the first intrathecal administered drug which was approved by the FDA to treat SMA children and adults (2016). The aim is to monitor the evolution of the Motor Function Measure-32 for SMA type II adult patients treated with SPINRAZA® (Nusinersen).
Eligibility
Primary outcome measure(s)
- Change of the Motor Function Measure-32 (MFM-32) from Baseline (M0) to 1 month (M1), 3 months (M3), 7 months (M7), 15 months (M15) and 27 months (M27) — at baseline, 1, 3, 7, 15 and 27 months
Within MFM-32, 32 terms will be evaluated to describe patient's motor functions and grouped into 3 sub-scores at baseline (M0), M1, M3, M7, M15 and M27: * D1: standing position and transfer * D2: axial and proximal motor function * D3: distal motor function The MFM-32 ratings rely on the use of a 4-point Likert scale based on the subject's maximal abilities without assistance (0: cannot initiate the task or maintain the starting position; 1: performs the task partially; 2: performs the task incompletely or imperfectly; 3: performs the task fully and normally.)
Trial sites (7)
| Facility | City | Region | Status |
|---|---|---|---|
| CHU de Lyon | Lyon | Auvergne-Rhône-Alpes | |
| CHRU de Lille | Lille | Hauts-de-France | |
| CHU de Montpellier | Montpellier | Occitanie | |
| CHU de Toulouse | Toulouse | Occitanie | |
| Hopital de la Timone - APHM | Marseille | Provence-Alpes-Côte d'Azur Region | |
| CHU de Nice | Nice | Provence-Alpes-Côte d'Azur Region | |
| APHP | Paris | Île-de-France Region |
More Centre Hospitalier Universitaire de Nice trials in France
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04159987 on ClinicalTrials.gov ↗ ← All trials in France