Actively Recruiting
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
Led by Hoffmann-La Roche · Updated on 2026-05-08
28
Participants Needed
19
Research Sites
241 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 in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.
CONDITIONS
Official Title
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Under 2 years of age at the time of informed consent
- Confirmed diagnosis of 5q-autosomal recessive SMA with genetic confirmation of homozygous deletion or compound heterozygosity causing loss of function of the SMN1 gene
- Confirmed presence of two SMN2 gene copies by laboratory testing
- Received onasemnogene abeparvovec gene therapy pre- or post-symptomatically
- Onasemnogene abeparvovec treatment at least 13 weeks before enrollment
- If treated with risdiplam before gene therapy, treatment lasted no more than 3 weeks and stopped 1 day before gene therapy
- Demonstrated a plateau or decline in function after gene therapy within 26 weeks, documented by two time points in swallowing and one other function such as respiratory or motor
You will not qualify if you...
- Previous or current participation in another investigational study before this treatment
- Unresolved laboratory abnormalities per onasemnogene abeparvovec prescribing information
- Previous or current use of SMN2-targeting antisense oligonucleotides
- Previous or current use of anti-myostatin agents
- Need for invasive ventilation or tracheostomy
- Having a feeding tube with an OrSAT score of 0
- Hospitalization for a lung event within 2 months or planned hospitalization at screening
- Major illness requiring hospitalization within 1 month before screening or any fever illness within 1 week before screening and the first dose
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 19 locations
1
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72103
Actively Recruiting
2
Valley Children's Hospital
Madera, California, United States, 93636
Actively Recruiting
3
Stanford Univ Medical Center
Palo Alto, California, United States, 94304
Actively Recruiting
4
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
5
University of Florida Pediatrics
Gainesville, Florida, United States, 32610
Actively Recruiting
6
Children's Healthcare of Atlanta Center for Advanced Pediatrics
Atlanta, Georgia, United States, 30329-2309
Actively Recruiting
7
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, United States, 49503
Actively Recruiting
8
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
9
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Actively Recruiting
10
Children's Hospital of the King's Daughter
Norfolk, Virginia, United States, 23510
Actively Recruiting
11
Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie
Berlin, Germany, 13353
Actively Recruiting
12
UKGM Standort Gießen
Giessen, Germany, 35392
Actively Recruiting
13
Soroka Medical Center
Beersheba, Israel, 8410101
Actively Recruiting
14
Schneider Children's Medical Center of Israel
Petah Tikva, Israel, 4920235
Actively Recruiting
15
Sourasky MC, Dana-Dwek Children's Hospital
Tel Aviv, Israel, 6423906
Actively Recruiting
16
Uniwersyteckie Centrum Kliniczne
Gdansk, Poland, 80-952
Actively Recruiting
17
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, Poland, 04-730
Actively Recruiting
18
Sidra Medicine
Al Rayyan, Qatar
Actively Recruiting
19
Great Ormond Street Hospital For Children
London, United Kingdom, WC1N 3JH
Actively Recruiting
Research Team
R
Reference Study ID Number: BN44621 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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