A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy (NCT05861986)
A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of Arkansas for Medical Sciences — Little Rock, Arkansas
Age range: 3 Months to 24 Months
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (16 Sites)
- University of Arkansas for Medical Sciences — Little Rock, Arkansas
- Children's Hospital of Colorado — Aurora, Colorado
- University of Florida Pediatrics — Gainesville, Florida
- Children's Healthcare of Atlanta Center for Advanced Pediatrics — Atlanta, Georgia
- Ann and Robert H. Lurie Children Hospital of Chicago — Chicago, Illinois
- Helen DeVos Children's Hospital at Spectrum Health — Grand Rapids, Michigan
- Columbia University Medical Center — New York, New York
- Children'S Hospital of Philadelphia — Philadelphia, Pennsylvania
- The University of Texas Southwestern Medical Center at Dallas — Dallas, Texas
- Cook Children's Jane and John Justin Neurosciences Center — Fort Worth, Texas
- Children's Hospital of the King's Daughter — Norfolk, Virginia
- Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie — Berlin,
- UKGM Standort Gießen — Giessen,
- Uniwersyteckie Centrum Kliniczne — Uniwersyteckie Centrum Kliniczne,
- Instytut Pomnik Centrum Zdrowia Dziecka — Warsaw,
- Great Ormond Street Hospital For Children — London,
📞 Contact the Study Coordinator
Phone: (888) 662-6728
Email: global-roche-genentech-trials@gene.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Muscular Atrophy, Spinal
- A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy — Orlando, Florida
- 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 — Little Rock, Arkansas
- A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK) — Orange, California
- A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec — Flower Mound, Texas
- A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies — Palo Alto, California
Explore by condition: All Muscular Atrophy, Spinal clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.