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

RECRUITINGPHASE4

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

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy

NCT05861986•Hoffmann-La Roche

View on ClinicalTrials.gov

Summary

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.

Eligibility

Age

0 - 2 years

Sex

ALL

Healthy Volunteers

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, 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

•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
•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.

Locations (16)

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

Key Dates

Start Date

May 30, 2024

Primary Completion Date

March 31, 2028

Completion Date

March 31, 2029

Last Updated

March 6, 2026

Enrollment

ESTIMATED participants

Conditions

Muscular Atrophy, Spinal

Interventions

Risdiplam

DRUG

Contacts

Reference Study ID Number: BN44620 https://forpatients.roche.com/

CONTACT

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

A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

NCT05808764

Muscular Atrophy, Spinal

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RECRUITING

Natural History of SMA

NCT05755451

Muscular Atrophy, Spinal

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ACTIVE_NOT_RECRUITING

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 6, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Natural History of SMA

Adult Spinal Muscular Atrophy (SMA) China Registry

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