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

• Status: Not yet recruiting
• Trial end date: 2028-03-31
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hoffmann-La Roche
• Treatments: Risdiplam

Criteria

Inclusion Criteria:

• <2 years of age at the time of informed consent

• Confirmed diagnosis of 5q-autosomal recessive SMA

• Confirmed presence of two SMN2 gene copies

• Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically

• Has received onasemnogene abeparvovec for SMA no less than 3 months prior to enrollment

• In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy (with a duration of 6 months 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:

• Treatment with investigational therapy prior to initiation of study treatment

• Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information

• Concomitant or previous administration of a SMN2-targeting antisense oligonucleotide or SMN2 splicing modifier either in a clinical study or as part of medical care

• Requiring invasive ventilation or tracheostomy

• Presence of feeding tube and/or 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.