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
NCT ID: NCT05861999
Last Updated: 2025-12-22
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
2024-08-14
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 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.
* In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy (with a duration of 26 weeks or less) documented by 2 individual time points in the functions as follows: swallowing AND one additional function/ability (respiratory, motor function, other) per appropriate expectation.
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
* 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
Valley Children's Hospital
Madera, California, United States
Stanford Univ Medical Center
Palo Alto, California, 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
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, United States
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Texas Southwestern Medical Center
Dallas, 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
Soroka Medical Center
Beersheba, , Israel
Schneider Children's Medical Center of Israel
Petah Tikva, , Israel
Sourasky MC, Dana-Dwek Children's Hospital
Tel Aviv, , Israel
Uniwersyteckie Centrum Kliniczne
Gdansk, , Poland
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, , Poland
Sidra Medicine
Al Rayyan, , Qatar
Great Ormond Street Hospital For Children
London, , United Kingdom
Countries
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Central Contacts
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Reference Study ID Number: BN44621 https://forpatients.roche.com/
Role: CONTACT
Phone: 888-662-6728 (U.S. Only)
Email: [email protected]
Other Identifiers
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2023-505161-81-00
Identifier Type: CTIS
Identifier Source: secondary_id
BN44621
Identifier Type: -
Identifier Source: org_study_id