Recruiting

Risdiplam

Sponsor:

Hoffmann-La Roche

Code:

NCT05861999

Conditions

Muscular Atrophy, Spinal

Eligibility Criteria

Sex: All

Age: 0 - 2

Healthy Volunteers: Not accepted

Interventions

Risdiplam

Study Details

Brief summary:

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

Muscular Atrophy, Spinal

Study ID

NCT05861999

Start date

Aug 14, 2024

Status verified date

Aug, 2026

Completion date

Mar 31, 2029

Anticipated

Primary completion date

Mar 31, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 2

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • <2 years of age at the time of informed consent
  • 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:

  • Previous or current enrolment in investigational study prior to initiation of study treatment
  • 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.

Study Design

Enrollment

28 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

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.

Interventions

Risdiplam

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

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 and Locations

Central contacts

Reference Study ID Number: BN44621 https://forpatients.roche.com/ No attachments to email below.

888-662-6728 (U.S. Only)global-roche-genentech-trials@gene.com

Locations

University of Arkansas for Medical Sciences

Recruiting

Little Rock, Arkansas, United States, 72103

Valley Children's Hospital

Recruiting

Madera, California, United States, 93636

Stanford Univ Medical Center

Recruiting

Palo Alto, California, United States, 94304

Children's Hospital of Colorado

Recruiting

Aurora, Colorado, United States, 80045

University of Florida Pediatrics

Recruiting

Gainesville, Florida, United States, 32610

Children's Healthcare of Atlanta Center for Advanced Pediatrics

Recruiting

Atlanta, Georgia, United States, 30329-2309

Helen DeVos Children's Hospital at Spectrum Health

Recruiting

Grand Rapids, Michigan, United States, 49503

Children'S Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

University of Texas Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75390

More Information

Sponsor

Hoffmann-La Roche

Last update posted

Aug 11, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-05. This information was provided to ClinicalTrials.gov by Hoffmann-La Roche on 2026-08-11.