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Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

NCT05522361 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2024-02-07

No results posted yet for this study

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.

Conditions

Interventions

DRUG

Risdiplam

administered to participants per product label insert

Sponsors & Collaborators

  • Genentech, Inc.

    collaborator INDUSTRY

  • Clinic for Special Children

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-11-06
Primary Completion
2025-12-31
Completion
2026-06-15
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05522361 on ClinicalTrials.gov