Home » Clinical Trials » Spinal muscular atrophy » NCT05861999
NCT05861999

A Phase IV Open-Label 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

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.

Want to learn more about this trial?

Request More Info

Interventions

RisdiplamDRUG
Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.

Study Locations

FacilityCityStateCountry
University of Arkansas for Medical SciencesLittle RockArkansasUnited States
Valley Children's HospitalMaderaCaliforniaUnited States
Stanford Univ Medical CenterPalo AltoCaliforniaUnited States
Children's Hospital of ColoradoAuroraColoradoUnited States
University of Florida PediatricsGainesvilleFloridaUnited States
Children's Healthcare of Atlanta Center for Advanced PediatricsAtlantaGeorgiaUnited States
Helen DeVos Children's Hospital at Spectrum HealthGrand RapidsMichiganUnited States
Children'S Hospital of PhiladelphiaPhiladelphiaPennsylvaniaUnited States
University of Texas Southwestern Medical CenterDallasTexasUnited States
Children's Hospital of the King's DaughterNorfolkVirginiaUnited States
Charité - Universitätsmedizin Berlin SPZ Abteilung NeuropaediatrieBerlinGermany
UKGM Standort GießenGiessenGermany
Soroka Medical CenterBeershebaIsrael
Schneider Children's Medical Center of IsraelPetah TikvaIsrael
Sourasky MC, Dana-Dwek Children's HospitalTel AvivIsrael
Uniwersyteckie Centrum KliniczneGdanskPoland
Instytut Pomnik Centrum Zdrowia DzieckaWarsawPoland
Sidra MedicineAl RayyanQatar
Great Ormond Street Hospital For ChildrenLondonUnited Kingdom

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026