Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

Study Identifier:
RISE
ClinicalTrials.gov Identifier:
NCT05522361
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Clinic for Special Children
Active, not recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: Risdiplam
Date
Nov 2022 - Dec 2025
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: 2 - 35 Years
Requirements Information
Inclusion and Exclusion 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

Protocol 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.

Trial Locations

Location
Status
Location
Clinic for Special Children
Strasburg, Pennsylvania, United States, 17579
Status
N/A

Contact Cure SMA

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