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

Study Identifier:
BN44620
ClinicalTrials.gov Identifier:
NCT05861986
EudraCT Identifier:
N/A
EU CT Number:
2023-504508-26-00
Sponsor:
Hoffmann-La Roche
Recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: Risdiplam
Date
May 2024 - Mar 2028
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: 3 - 24 Months
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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.

Protocol 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.

Trial Locations

Location
Status
Location
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72103
Status
Recruiting
Location
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
Status
Recruiting
Location
University of Florida Pediatrics
Gainesville, Florida, United States, 32610
Status
Recruiting
Location
Children's Healthcare of Atlanta Center for Advanced Pediatrics
Atlanta, Georgia, United States, 30329-2309
Status
Recruiting
Location
Ann and Robert H. Lurie Children Hospital of Chicago
Chicago, Illinois, United States, 60611
Status
Not yet recruiting
Location
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, United States, 49503
Status
Recruiting
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