Risdiplam for Spinal Muscular Atrophy (SMA)

Phase-Based Progress Estimates
Clinic for Special Children, Strasburg, PA
Spinal Muscular Atrophy (SMA)+3 More
Risdiplam - Drug
< 65
All Sexes
What conditions do you have?

Study Summary

Risdiplam Exchange (RISE) is a study of spinal muscular atrophy (SMA) patients who crossover to 36 months of open-label risdiplam monotherapy following a comparable period of nusinersen treatment. The schedule of assessments (SOAs) carry over seamlessly for the cohort from studies done while treated with nusinersen and continue to track the most informative outcomes from that trial (e.g. nine hole peg test and grip strength), while adding the Box and Block Test (BBT) as an additional measure of upper limb endurance and function.

Eligible Conditions

  • Spinal Muscular Atrophy (SMA)

Treatment Effectiveness

Effectiveness Progress

3 of 3
This is further along than 93% of similar trials

Other trials for Spinal Muscular Atrophy (SMA)

Study Objectives

1 Primary · 5 Secondary · Reporting Duration: 36 months

36 months
Comparative intrasubject change in grip strength
Comparative intrasubject performance on nine hole peg test (NHPT)
Frequency and type of adverse events
Intrasubject change in pulmonary function
Intrasubject changes in lower limb and overall motor function
Intrasubject changes in upper limb motor function

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Other trials for Spinal Muscular Atrophy (SMA)

Side Effects for

Normal Hepatic Function
0%Upper gastrointestinal haemorrhage
0%Chest discomfort
0%Ear pain
This histogram enumerates side effects from a completed 2020 Phase 1 trial (NCT03920865) in the Normal Hepatic Function ARM group. Side effects include: Pruritus with 0%, Vomiting with 0%, Upper gastrointestinal haemorrhage with 0%, Diarrhoea with 0%, Chest discomfort with 0%.

Trial Design

1 Treatment Group

Open-label crossover
1 of 1
Experimental Treatment

10 Total Participants · 1 Treatment Group

Primary Treatment: Risdiplam · No Placebo Group · Phase 4

Open-label crossover
Experimental Group · 1 Intervention: Risdiplam · Intervention Types: Drug
First Studied
Drug Approval Stage
How many patients have taken this drug
Completed Phase 1

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 36 months
Closest Location: Clinic for Special Children · Strasburg, PA
Photo of pennsylvania 1Photo of pennsylvania 2Photo of pennsylvania 3
2019First Recorded Clinical Trial
2 TrialsResearching Spinal Muscular Atrophy (SMA)
3 CompletedClinical Trials

Who is running the clinical trial?

Genentech, Inc.Industry Sponsor
1,482 Previous Clinical Trials
563,335 Total Patients Enrolled
3 Trials studying Spinal Muscular Atrophy (SMA)
544 Patients Enrolled for Spinal Muscular Atrophy (SMA)
Clinic for Special ChildrenLead Sponsor

Eligibility Criteria

Age < 65 · All Participants · 3 Total Inclusion Criteria

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About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 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.