Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

• Status: Not yet recruiting
• Trial end date: 2026-06-15
• Target enrollment: 0
• Participant gender: All

Summary

Risdiplam Exchange (RISE) is a study of spinal muscular atrophy (SMA) patients who crossover to 36 months of open-label risdiplam monotherapy following a comparable period of nusinersen treatment. The schedule of assessments (SOAs) carry over seamlessly for the cohort from studies done while treated with nusinersen and continue to track the most informative outcomes from that trial (e.g. nine hole peg test and grip strength), while adding the Box and Block Test (BBT) as an additional measure of upper limb endurance and function.

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

Details

• Lead Sponsor: Clinic for Special Children
• Collaborator: Genentech, Inc.
• Treatments: Risdiplam

Criteria

Inclusion Criteria:

• Biallelic SMN1 deletions

• 3 or 4 copies of SMN2

• Prior treatment with nusinersen for a minimum of 22 months

Exclusion Criteria:

• Prior treatment with SMN gene replacement therapy

• Prior exposure to another investigational agent.

• Confounding neuromuscular disorder other than SMA