Risdiplam for Spinal Muscular Atrophy
(Jewelfish Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.
Who Is on the Research Team?
Clinical Trials
Principal Investigator
Hoffmann-La Roche
Are You a Good Fit for This Trial?
This trial is for adults, children, and infants with Spinal Muscular Atrophy who have been part of a previous study or treated with specific drugs. Women must not be pregnant and agree to contraception; men also need to use birth control. Participants should be well enough in the investigator's opinion, and young children may need feeding tubes or ventilation as recommended.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive multiple doses of risdiplam orally once daily
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants may opt into continuation of treatment long-term
What Are the Treatments Tested in This Trial?
Interventions
- Risdiplam
Risdiplam is already approved in United States, European Union, Brazil, China for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Hoffmann-La Roche
Lead Sponsor
Dr. Levi Garraway
Hoffmann-La Roche
Chief Medical Officer since 2019
MD from the University of Basel
Dr. Thomas Schinecker
Hoffmann-La Roche
Chief Executive Officer since 2023
PhD in Molecular Biology from New York University