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

Study Identifier:
BN44621
ClinicalTrials.gov Identifier:
NCT05861999
EudraCT Identifier:
N/A
EU CT Number:
2023-505161-81-00
Sponsor:
Hoffmann-La Roche
Recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: Risdiplam
Date
Aug 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
  • \<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.

Protocol 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.

Trial Locations

Location
Status
Location
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72103
Status
Recruiting
Location
Valley Children's Hospital
Madera, California, United States, 93636
Status
Recruiting
Location
Stanford Univ Medical Center
Palo Alto, California, United States, 94304
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
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