Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE4

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-11-06

Study Completion Date

2026-06-15

Brief Summary

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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.

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Detailed Description

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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

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Spinal Muscular Atrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Open-label crossover

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

Group Type EXPERIMENTAL

Risdiplam

Intervention Type DRUG

administered to participants per product label insert

Interventions

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Risdiplam

administered to participants per product label insert

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

2 Years

Maximum Eligible Age

35 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No