Risdiplam for Spinal Muscular Atrophy

(RISE Trial)

Overseen By Karlla W Brigatti, MS

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 4

Waitlist Available

Sponsor: Clinic for Special Children

No Placebo Group

Prior Safety Data

Approved in 9 Jurisdictions

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

This trial is for people with Spinal Muscular Atrophy who have two missing SMN1 genes and either three or four copies of the SMN2 gene. They must have been treated with nusinersen for at least 22 months. Those who've had other experimental treatments, gene replacement therapy, or another neuromuscular disorder besides SMA cannot join.

Inclusion Criteria

I have a genetic condition with two missing SMN1 genes.

My genetic test shows I have 3 or 4 copies of the SMN2 gene.

I have been treated with nusinersen for at least 22 months.

Exclusion Criteria

You have a neuromuscular disorder other than SMA that could affect the study results.

Prior exposure to another investigational agent

I have previously received SMN gene therapy.

Treatment Details

Interventions

• Risdiplam (Small Molecule)

Trial Overview The study tests risdiplam in patients previously treated with nusinersen. Participants will switch to risdiplam for a continuous period of 36 months, and their motor functions will be assessed using established tests from previous studies along with an additional test called the Box and Block Test.

Participant Groups

Group I: Open-label crossover Experimental Treatment 1 Intervention

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

Risdiplam is already approved in Brazil, China for the following indications:

Approved in Brazil as Evrysdi for:

• Spinal muscular atrophy (SMA)

Approved in China as Evrysdi for:

• Spinal muscular atrophy (SMA)

Who Is Running the Clinical Trial?

Clinic for Special Children

Lead Sponsor

Trials

2

Recruited

50+

Genentech, Inc.

Industry Sponsor

Trials

1,578

Recruited

569,000+

Ashley Magargee

Genentech, Inc.

Chief Executive Officer since 2024

MBA from Harvard University, BA from Princeton University

Levi Garraway

Genentech, Inc.

Chief Medical Officer since 2021

MD, PhD