A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
NCT ID: NCT05861986
Last Updated: 2026-02-09
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE4
28 participants
INTERVENTIONAL
2024-05-30
2029-03-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Risdiplam
Participants will receive risdiplam orally once daily for 72 weeks (Treatment Period). The Treatment Period will be followed by a 1-year Treatment Extension Period for a total study duration of 120 weeks (approximately 2.5 years) for each participant enrolled.
Risdiplam
Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.
Interventions
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Risdiplam
Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the Survival of Motor Neuron 1 (SMN1) gene
* Confirmed presence of two SMN2 gene copies as documented through laboratory testing
* Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically
* Has received onasemnogene abeparvovec for SMA no less than 13 weeks, but not more than months 30 weeks, prior to enrollment
* If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration
* Has, in the opinion of the investigator, not experienced clinically significant decline in function from the time of onasemnogene abeparvovec administration
Exclusion Criteria
* Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information
* Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide
* Concomitant or previous use of an anti-myostatin agent
* Participants requiring invasive ventilation or tracheostomy
* Participants requiring awake non-invasive ventilation or with awake hypoxemia (Arterial Oxygen Saturation \[SaO2\] \<95%) with or without ventilator support
* Presence of feeding tube and an OrSAT score of 0
* Hospitalization for pulmonary event within the last 2 months, or any planned hospitalization at the time of screening
* Any major illness requiring hospitalization within 1 month before the screening examination or any febrile illness within 1 week prior to screening and up to first dose administration.
3 Months
24 Months
ALL
No
Sponsors
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Hoffmann-La Roche
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trials
Role: STUDY_DIRECTOR
Hoffmann-La Roche
Locations
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University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
Children's Hospital of Colorado
Aurora, Colorado, United States
University of Florida Pediatrics
Gainesville, Florida, United States
Children's Healthcare of Atlanta Center for Advanced Pediatrics
Atlanta, Georgia, United States
Ann and Robert H. Lurie Children Hospital of Chicago
Chicago, Illinois, United States
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, United States
Columbia University Medical Center
New York, New York, United States
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
The University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, United States
Cook Children's Jane and John Justin Neurosciences Center
Fort Worth, Texas, United States
Children's Hospital of the King's Daughter
Norfolk, Virginia, United States
Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie
Berlin, , Germany
UKGM Standort Gießen
Giessen, , Germany
Uniwersyteckie Centrum Kliniczne
Gda?sk, , Poland
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, , Poland
Great Ormond Street Hospital For Children
London, , United Kingdom
Countries
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Central Contacts
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Reference Study ID Number: BN44620 https://forpatients.roche.com/
Role: CONTACT
Other Identifiers
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2023-504508-26-00
Identifier Type: CTIS
Identifier Source: secondary_id
BN44620
Identifier Type: -
Identifier Source: org_study_id
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