Risdiplam for Atrophy

Phase-Based Progress Estimates
Atrophy+3 More
Risdiplam - Drug
Any Age
All Sexes
What conditions do you have?

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.

Eligible Conditions
  • Atrophy
  • Spinal Muscular Atrophy (SMA)

Treatment Effectiveness

Effectiveness Progress

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

Study Objectives

1 Primary · 27 Secondary · Reporting Duration: Up to 5 years

Up to 5 years
Average Length of Stay per Hospitalization
Dupuytren Contracture
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1)
Vital capacity
Change From Baseline in Maximal Expiratory Pressure (MEP)
Change From Baseline in Maximal Inspiratory Pressure (MIP)
Change from Baseline Percentile for Weight-for-Age, Length/Height-for-Age, Weight-for-Length/Height, and Head Circumference-for-Age
Change from Baseline in Best Sniff Nasal Inspiratory Pressure (SNIP)
Change from Baseline in Chest Circumference
Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score
Change from Baseline in Distance Walked During the Six-Minute Walk Test (6MWT)
Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE)
Change from Baseline in Motor Milestones Assessed in the Hammersmith Infant Neurological Examination Module 2 (HINE-2)
Change from Baseline in the Revised Upper Limb Module (RULM) Total Score
Change in the Caregiver-Reported Function, as Measured by the Pediatric Evaluation of Disability Inventory-Computer Adaptive Test (PEDI-CAT)
Number of Participants Requiring Hospitalizations (For Any Reason) per Patient-Year
Number of Participants With Adverse Events (AEs), Adverse Events of Special Interest (AESIs), and Serious Adverse Events (SAEs)
Participant Reported and Caregiver Reported Assessments Using SMA Independence Scale (SMAIS) - Upper Limb Module (ULM) and Ambulatory Module
Percentage Change in Distance Walked in the First Versus the Last Minute of the 6MWT
Percentage of Infants Who Achieve the Attainment Levels of Six World Health Organization (WHO) Gross Motor Milestones
Percentage of Participants Considered Improved on the Clinical Global Impression of Change (CGI-C) Scale
Percentage of Participants Who Achieved or Maintain Ability to Feed Orally
Percentage of Participants Who Achieved or Maintain Normal Crying Function and Speaking Function
Therapeutic procedure
Ratio Between Chest and Head Circumferences
Survival in Infants Aged < 2 Years With Type 1 SMA Over Time
Time to Loss of Swallowing
Time to Permanent Ventilation

Trial Safety

Safety Progress

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

Trial Design

1 Treatment Group

1 of 1

Experimental Treatment

500 Total Participants · 1 Treatment Group

Primary Treatment: Risdiplam · No Placebo Group · Phase 4

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

Screening: ~3 weeks
Treatment: Varies
Reporting: up to 5 years

Who is running the clinical trial?

Genentech, Inc.Lead Sponsor
1,488 Previous Clinical Trials
563,426 Total Patients Enrolled
12 Trials studying Atrophy
1,091 Patients Enrolled for Atrophy
Clinical TrialsStudy DirectorHoffmann-La Roche
2,134 Previous Clinical Trials
901,046 Total Patients Enrolled
26 Trials studying Atrophy
5,754 Patients Enrolled for Atrophy

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 preview

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 - 65100.0%
What site did they apply to?
Central Texas Neurology Consultants100.0%
What portion of applicants met pre-screening criteria?
Did not meet criteria100.0%