NCT05522361

Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

Risdiplam Exchange in Patients With Spinal Muscular Atrophy (SMA) Previously and Exclusively Treated With Nusinersen

• Status: Active Not Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Clinic for Special Children · Academic / Other
• Sex: All
• Age: 2 Years – 35 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Risdiplam is an orally bioavailable small molecule that distributes into the central nervous system (CNS) and peripheral tissues where it modifies SMN2 pre-mRNA splicing and increases tissue SMN protein levels. It was approved by the U.S. Food and Drug Administration for all SMA patients older than 2 months of age in August 2020. Clinical trial data prompted us to consider risdiplam a reasonable alternative to nusinersen administered intrathecally or by subcutaneous intrathecal catheter (SIC) for patients with more advanced SMA. Considered within this clinical context, risdiplam presents a significant advantage by eliminating the risks of mechanical failure, intrathecal bleeding, and CNS infection associated with the SIC device. The schedule of assessments (SOAs) used in this cohort during the previous three-year period as part of a nusinersen study provide us with an established framework for data collection. Thus, the overall clinical experience with risdiplam as compared to nusinersen can be assessed using a prospective, crossover design in a real-world setting.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
DRUG	Risdiplam	administered to participants per product label insert

Timeline

• Start date: 2022-11-06
• Primary completion: 2025-12-31
• Completion: 2026-06-15
• First posted: 2022-08-31
• Last updated: 2024-02-07

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study