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Recruiting Phase 4

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

NCT05861999 · tracked via the Priya Life Science Poland tracker
Phase
Phase 4
Started
2024-08-14
Last updated
2026-09-10

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 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.

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 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.

Primary outcome measure(s)

  • 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 — Baseline, Week 72
    The BSID-III is a standardized assessment commonly used to evaluate developmental functioning of infants and young children between 1 month and 42 months of age. The gross motor scale measures the movement of the limbs and torso. Items assess static positioning (e.g., sitting, standing); dynamic movement, including locomotion and coordination; balance; and motor planning. The gross motor scale consists of 72 items scored at 0 (unable to perform) or 1 (criteria for item achieved). A higher raw score indicates improvement.

Trial sites (19)

FacilityCityRegionStatus
University of Arkansas for Medical Sciences Little Rock Arkansas Recruiting
Valley Children's Hospital Madera California Recruiting
Stanford Univ Medical Center Palo Alto California 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
Helen DeVos Children's Hospital at Spectrum Health Grand Rapids Michigan Recruiting
Children'S Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
University of Texas Southwestern Medical Center Dallas 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
Soroka Medical Center Beersheba Israel Recruiting
Schneider Children's Medical Center of Israel Petah Tikva Israel Recruiting
Sourasky MC, Dana-Dwek Children's Hospital Tel Aviv Israel Recruiting
Uniwersyteckie Centrum Kliniczne Gda?sk Poland Recruiting
Instytut Pomnik Centrum Zdrowia Dziecka Warsaw Poland Recruiting
Sidra Medicine Al Rayyan Qatar Recruiting
Great Ormond Street Hospital For Children London United Kingdom Recruiting
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 NCT05861999 on ClinicalTrials.gov ↗ ← All trials in Poland