A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Condition(s) studied
Investigational drug(s) / intervention(s)
Risdiplam: Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.
Study summary
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.
Eligibility
Primary outcome measure(s)
- Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment — Baseline, Week 72
The BSID-III is a standardized assessment commonly used to evaluate developmental functioning of infants and young children between 1 month and 42 months of age. The gross motor scale measures the movement of the limbs and torso. Items assess static positioning (e.g., sitting, standing); dynamic movement, including locomotion and coordination; balance; and motor planning. The gross motor scale consists of 72 items scored at 0 (unable to perform) or 1 (criteria for item achieved). A higher raw score indicates improvement.
Trial sites (16)
| Facility | City | Region | Status |
|---|---|---|---|
| University of Arkansas for Medical Sciences | Little Rock | Arkansas | Recruiting |
| Children's Hospital of Colorado | Aurora | Colorado | Recruiting |
| University of Florida Pediatrics | Gainesville | Florida | Recruiting |
| Children's Healthcare of Atlanta Center for Advanced Pediatrics | Atlanta | Georgia | Recruiting |
| Ann and Robert H. Lurie Children Hospital of Chicago | Chicago | Illinois | Recruiting |
| Helen DeVos Children's Hospital at Spectrum Health | Grand Rapids | Michigan | Recruiting |
| Columbia University Medical Center | New York | New York | Recruiting |
| Children'S Hospital of Philadelphia | Philadelphia | Pennsylvania | Recruiting |
| The University of Texas Southwestern Medical Center at Dallas | Dallas | Texas | Recruiting |
| Cook Children's Jane and John Justin Neurosciences Center | Fort Worth | Texas | Recruiting |
| Children's Hospital of the King's Daughter | Norfolk | Virginia | Withdrawn |
| Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie | Berlin | Germany | Recruiting |
| UKGM Standort Gießen | Giessen | Germany | Recruiting |
| Uniwersyteckie Centrum Kliniczne | Uniwersyteckie Centrum Kliniczne | Poland | Recruiting |
| Instytut Pomnik Centrum Zdrowia Dziecka | Warsaw | Poland | Recruiting |
| Great Ormond Street Hospital For Children | London | United Kingdom | Recruiting |
On this site
📄 Evrysdi (risdiplam) drug profile →More Hoffmann-La Roche trials in Poland
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 NCT05861986 on ClinicalTrials.gov ↗ ← All trials in Poland