NCT05522361: RISE

Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

Active, enrolled Phase 4 Last updated 7 February 2024

What this trial tests

Phase 4 trial testing Risdiplam in Spinal Muscular Atrophy in 10 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

6 November 2022

Primary endpoint
31 December 2025

15 June 2026

Quick facts

Lead sponsor	Clinic for Special Children
Phase	Phase 4
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	10
Start date	6 November 2022
Primary completion	31 December 2025
Estimated completion	15 June 2026
Sites	1 location across United States

Drugs / interventions tested

• Risdiplam (RISDIPLAM) — full drug profile →

Conditions studied

• Spinal Muscular Atrophy — all drugs for Spinal Muscular Atrophy →

Sponsor

Clinic for Special Children

Who can join

Adults 2 to 35, any sex, with Spinal Muscular Atrophy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines.
   Liu M, Wang Y, Zhang Y, Hu D, et al · · 2025 · cited 62× · PMID 40059188 · DOI 10.1038/s41392-024-02112-8
2. Current clinical applications of AAV-mediated gene therapy.
   Byrne BJ, Flanigan KM, Matesanz SE, Finkel RS, et al · · 2025 · cited 43× · PMID 40329530 · DOI 10.1016/j.ymthe.2025.04.045
3. Pharmacological Therapies of Spinal Muscular Atrophy: A Narrative Review of Preclinical, Clinical-Experimental, and Real-World Evidence.
   Crisafulli S, Boccanegra B, Vitturi G, Trifirò G, et al · · 2023 · cited 11× · PMID 37891814 · DOI 10.3390/brainsci13101446
4. An updated review of the SMA clinical trial landscape in the United States: Findings from analysis of recruitment targets on ClinicalTrials.gov and a survey of SMA clinical trial sites on factors affecting site capacity and readiness.
   Sarr F, Peterson I, Glascock J, Curry M. · · 2026 · cited 1× · PMID 41658435 · DOI 10.1016/j.conctc.2026.101601