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Clinical Trials in the UK / NCT05861986
Recruiting Phase 4

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

NCT05861986 · tracked via the Priya Life Science UK tracker
Sponsor
Hoffmann-La Roche
Phase
Phase 4
Started
2024-05-30
Last updated
2026-09-03

Condition(s) studied

Muscular Atrophy, Spinal

Investigational drug(s) / intervention(s)

Risdiplam

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

Study 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

Sex
ALL
Min age
3 Months
Max age
24 Months
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (16)

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Children's Hospital of the King's Daughter Norfolk Virginia Withdrawn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

On this site

📄 Evrysdi (risdiplam) drug profile →

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05861986 on ClinicalTrials.gov ↗ ← All trials in the UK