Study Summary
This trial will study the long-term safety and effectiveness of the drug risdiplam in patients with spinal muscular atrophy. The trial will last for up to 5 years, and patients will be followed even if they discontinue the drug.
- Atrophy
- Spinal Muscular Atrophy (SMA)
Treatment Effectiveness
Effectiveness Progress
Study Objectives
1 Primary · 27 Secondary · Reporting Duration: Up to 5 years
Trial Safety
Safety Progress
Trial Design
1 Treatment Group
Risdiplam
1 of 1
Experimental Treatment
500 Total Participants · 1 Treatment Group
Primary Treatment: Risdiplam · No Placebo Group · Phase 4
Trial Logistics
Trial Timeline
Who is running the clinical trial?
Eligibility Criteria
Age Any Age · All Participants · 2 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:About The Reviewer
Michael Gill - B. Sc.
First Published: October 26th, 2021
Last Reviewed: November 11th, 2022
Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.
Who else is applying?
How old are they?
| 18 - 65 | 100.0% |
What site did they apply to?
| Central Texas Neurology Consultants | 100.0% |
What portion of applicants met pre-screening criteria?
| Did not meet criteria | 100.0% |