Actively Recruiting
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Led by Hoffmann-La Roche · Updated on 2026-05-04
28
Participants Needed
16
Research Sites
252 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Child is younger than 2 years old at the time of consent
- Confirmed genetic diagnosis of 5q-autosomal recessive SMA with loss of SMN1 gene function
- Presence of two copies of the SMN2 gene confirmed by lab testing
- Received onasemnogene abeparvovec gene therapy either before or after symptoms appear
- Onasemnogene abeparvovec administered at least 13 weeks and no more than 30 weeks before enrollment
- If treated with risdiplam before onasemnogene abeparvovec, risdiplam use was less than 3 weeks and stopped 1 day before gene therapy
- No significant decline in function since receiving onasemnogene abeparvovec, as judged by the investigator
You will not qualify if you...
- Previously or currently enrolled in any investigational study before starting this treatment
- Unresolved standard laboratory abnormalities per onasemnogene abeparvovec guidelines
- Use of SMN2-targeting antisense oligonucleotides before or during study
- Use of anti-myostatin agents before or during study
- Need for invasive ventilation or tracheostomy
- Need for awake non-invasive ventilation or awake low oxygen levels (SaO2 <95%) with or without ventilator
- Presence of feeding tube with an OrSAT score of 0
- Hospitalized for lung problems within the last 2 months or planned hospitalization at screening
- Major illness requiring hospital stay within 1 month before screening or fever within 1 week before screening and dosing
AI-Screening
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Trial Site Locations
Total: 16 locations
1
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72103
Actively Recruiting
2
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
3
University of Florida Pediatrics
Gainesville, Florida, United States, 32610
Actively Recruiting
4
Children's Healthcare of Atlanta Center for Advanced Pediatrics
Atlanta, Georgia, United States, 30329-2309
Actively Recruiting
5
Ann and Robert H. Lurie Children Hospital of Chicago
Chicago, Illinois, United States, 60611
Not Yet Recruiting
6
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, United States, 49503
Actively Recruiting
7
Columbia University Medical Center
New York, New York, United States, 10032
Actively Recruiting
8
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
9
The University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, United States, 75390
Actively Recruiting
10
Cook Children's Jane and John Justin Neurosciences Center
Fort Worth, Texas, United States, 76104
Actively Recruiting
11
Children's Hospital of the King's Daughter
Norfolk, Virginia, United States, 23510
Actively Recruiting
12
Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie
Berlin, Germany, 13353
Actively Recruiting
13
UKGM Standort Gießen
Giessen, Germany, 35392
Actively Recruiting
14
Uniwersyteckie Centrum Kliniczne
Uniwersyteckie Centrum Kliniczne, Poland, 80-952
Actively Recruiting
15
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, Poland, 04-730
Actively Recruiting
16
Great Ormond Street Hospital For Children
London, United Kingdom, WC1N 3JH
Actively Recruiting
Research Team
R
Reference Study ID Number: BN44620 https://forpatients.roche.com/
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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