A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
Hoffmann-La Roche
Study ID
NCT05861986
Phase
PHASE4
Status
Recruiting
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Conditions

  • Muscular Atrophy, Spinal

Eligibility Criteria

Sex
ALL
Age
3 Months - 24 Months
Healthy Volunteers
Not accepted

Interventions

  • Risdiplam — DRUG
    Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.

Study Details

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.

Key Dates

Start date
May 30, 2024
Status verified
May 2026
Primary completion
Mar 31, 2028
Completion
Mar 31, 2029

Study Design

Enrollment
28 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: 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.

Primary Outcome Measure

Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment [ Time Frame: Baseline, Week 72 ]

Central Contacts

  • Reference Study ID Number: BN44620 https://forpatients.roche.com/
    888-662-6728 (U.S. Only)
    [email protected]

Locations (11)

FacilityCityStateZIPSite coordinators
University of Arkansas for Medical SciencesLittle RockArkansas72103-
Children's Hospital of ColoradoAuroraColorado80045-
University of Florida PediatricsGainesvilleFlorida32610-
Children's Healthcare of Atlanta Center for Advanced PediatricsAtlantaGeorgia30329-2309-
Ann and Robert H. Lurie Children Hospital of ChicagoChicagoIllinois60611-
Helen DeVos Children's Hospital at Spectrum HealthGrand RapidsMichigan49503-
Columbia University Medical CenterNew YorkNew York10032-
Children'S Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
The University of Texas Southwestern Medical Center at DallasDallasTexas75390-
Cook Children's Jane and John Justin Neurosciences CenterFort WorthTexas76104-
Children's Hospital of the King's DaughterNorfolkVirginia23510-

Find similar trials in Little Rock, AR

By research site
University of Arkansas for Medical Sciences· Little Rock, ARChildren's Hospital of Colorado· Aurora, COUniversity of Florida Pediatrics· Gainesville, FLChildren's Healthcare of Atlanta Center for Advanced Pediatrics· Atlanta, GAAnn and Robert H. Lurie Children Hospital of Chicago· Chicago, ILHelen DeVos Children's Hospital at Spectrum Health· Grand Rapids, MI

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