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

Recruiting now · Phase 4

Conditions studied: Muscular Atrophy, Spinal

In brief

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 facts

Study ID

NCT05861986

Run by

Hoffmann-La Roche

People needed

28

Starts

2024-05-30

Expected to finish

2029-03-31

Last updated by the study team

2026-08-04

Who can join

Age: 0 and older, up to 2. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• <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

You may not qualify if…

• 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.

Where it is running

• Great Ormond Street Hospital For Children — London, United Kingdom (enrolling)
• Children's Hospital of Colorado — Aurora, Colorado, United States (enrolling)
• University of Florida Pediatrics — Gainesville, Florida, United States (enrolling)
• Children's Healthcare of Atlanta Center for Advanced Pediatrics — Atlanta, Georgia, United States (enrolling)
• Instytut Pomnik Centrum Zdrowia Dziecka — Warsaw, Poland (enrolling)
• University of Arkansas for Medical Sciences — Little Rock, Arkansas, United States (enrolling)
• Helen DeVos Children's Hospital at Spectrum Health — Grand Rapids, Michigan, United States (enrolling)
• Columbia University Medical Center — New York, New York, United States (enrolling)
• Children'S Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• The University of Texas Southwestern Medical Center at Dallas — Dallas, Texas, United States (enrolling)
• Cook Children's Jane and John Justin Neurosciences Center — Fort Worth, Texas, United States (enrolling)
• Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie — Berlin, Germany (enrolling)
• UKGM Standort Gießen — Giessen, Germany (enrolling)
• Uniwersyteckie Centrum Kliniczne — Uniwersyteckie Centrum Kliniczne, Poland (enrolling)
• Ann and Robert H. Lurie Children Hospital of Chicago — Chicago, Illinois, United States
• Children's Hospital of the King's Daughter — Norfolk, Virginia, United States

Full record on ClinicalTrials.gov

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