NCT05522361

Brief 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 Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_4

Timeline
1mo left

Started Nov 2022

Longer than P75 for phase_4

Geographic Reach
1 country

1 active site

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress97%
Nov 2022Jun 2026

First Submitted

Initial submission to the registry

May 24, 2022

Completed
3 months until next milestone

First Posted

Study publicly available on registry

August 31, 2022

Completed
2 months until next milestone

Study Start

First participant enrolled

November 6, 2022

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2025

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 15, 2026

Expected
Last Updated

February 7, 2024

Status Verified

February 1, 2024

Enrollment Period

3.2 years

First QC Date

May 24, 2022

Last Update Submit

February 5, 2024

Conditions

Spinal Muscular Atrophy

Keywords

nusinersenprospectiverisdiplam

Outcome Measures

Primary Outcomes (1)

  • Comparative intrasubject performance on nine hole peg test (NHPT)

    Time in seconds to place and subsequently remove nine one inch pegs in holes assessed in dominant and non dominant hands

    36 months

Secondary Outcomes (5)

  • Intrasubject changes in lower limb and overall motor function

    36 months

  • Intrasubject changes in upper limb motor function

    36 months

  • Intrasubject change in pulmonary function

    36 months

  • Frequency and type of adverse events

    36 months

  • Comparative intrasubject change in grip strength

    36 months

Study Arms (1)

Open-label crossover

EXPERIMENTAL

Participants crossover to 36 months of open-label risdiplam mono therapy following a comparable period of nusinersen treatment.

Drug: Risdiplam

Interventions

RisdiplamDRUG

administered to participants per product label insert

Open-label crossover

Eligibility Criteria

Age2 Years - 35 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Biallelic SMN1 deletions
  • or 4 copies of SMN2
  • Prior treatment with nusinersen for a minimum of 22 months

You may not qualify if:

  • Prior treatment with SMN gene replacement therapy
  • Prior exposure to another investigational agent.
  • Confounding neuromuscular disorder other than SMA

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Clinic for Special Children

Strasburg, Pennsylvania, 17579, United States

Location

Related Publications (3)

  • Strauss KA, Carson VJ, Brigatti KW, Young M, Robinson DL, Hendrickson C, Fox MD, Reed RM, Puffenberger EG, Mackenzie W, Miller F. Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy. J Pediatr Orthop. 2018 Nov/Dec;38(10):e610-e617. doi: 10.1097/BPO.0000000000001247.

    PMID: 30134351BACKGROUND

  • Carson VJ, Young M, Brigatti KW, Robinson DL, Reed RM, Sohn J, Petrillo M, Farwell W, Miller F, Strauss KA. Nusinersen by subcutaneous intrathecal catheter for symptomatic spinal muscular atrophy patients with complex spine anatomy. Muscle Nerve. 2022 Jan;65(1):51-59. doi: 10.1002/mus.27425. Epub 2021 Oct 19.

    PMID: 34606118BACKGROUND

  • Ribero VA, Daigl M, Marti Y, Gorni K, Evans R, Scott DA, Mahajan A, Abrams KR, Hawkins N. How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison. J Comp Eff Res. 2022 Apr;11(5):347-370. doi: 10.2217/cer-2021-0216. Epub 2022 Jan 18.

    PMID: 35040693BACKGROUND

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Interventions

Risdiplam

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Medical Director

Study Record Dates

First Submitted

May 24, 2022

First Posted

August 31, 2022

Study Start

November 6, 2022

Primary Completion

December 31, 2025

Study Completion (Estimated)

June 15, 2026

Last Updated

February 7, 2024

Record last verified: 2024-02

Data Sharing

IPD Sharing
Will not share

Locations

US(1)