A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function 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 in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled 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 (19)
| Facility | City | Region | Status |
|---|---|---|---|
| University of Arkansas for Medical Sciences | Little Rock | Arkansas | Recruiting |
| Valley Children's Hospital | Madera | California | Recruiting |
| Stanford Univ Medical Center | Palo Alto | California | 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 |
| Helen DeVos Children's Hospital at Spectrum Health | Grand Rapids | Michigan | Recruiting |
| Children'S Hospital of Philadelphia | Philadelphia | Pennsylvania | Recruiting |
| University of Texas Southwestern Medical Center | Dallas | 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| Children's Hospital of the King's Daughter | Norfolk | Virginia | Withdrawn |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 |
| Soroka Medical Center | Beersheba | Israel | Recruiting |
| Schneider Children's Medical Center of Israel | Petah Tikva | Israel | Recruiting |
| Sourasky MC, Dana-Dwek Children's Hospital | Tel Aviv | Israel | Recruiting |
| Uniwersyteckie Centrum Kliniczne | Gda?sk | Poland | Recruiting |
| Instytut Pomnik Centrum Zdrowia Dziecka | Warsaw | Poland | Recruiting |
| Sidra Medicine | Al Rayyan | Qatar | 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 NCT05861999 on ClinicalTrials.gov ↗ ← All trials in Poland