Risdiplam for Spinal Muscular Atrophy (RISE Trial)
Palo Alto (17 mi)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
I have previously received SMN gene therapy.
Treatment Details
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.
1Treatment groups
Experimental Treatment
Group I: Open-label crossoverExperimental Treatment1 Intervention
Participants crossover to 36 months of open-label risdiplam mono therapy following a comparable period of nusinersen treatment.
Risdiplam is already approved in United States, European Union, Brazil, China for the following indications:
🇺🇸 Approved in United States as Evrysdi for:
- Spinal muscular atrophy (SMA) in patients 2 months of age and older
🇪🇺 Approved in European Union as Evrysdi for:
- 5q-autosomal recessive SMA with a clinical diagnosis of SMA types 1, 2, or 3 or with one to four survival motor neuron 2 (SMN2) copies
🇧🇷 Approved in Brazil as Evrysdi for:
- Spinal muscular atrophy (SMA)
🇨🇳 Approved in China as Evrysdi for:
- Spinal muscular atrophy (SMA)
Find a clinic near you
Research locations nearbySelect from list below to view details:
Clinic for Special ChildrenStrasburg, PA
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Who is running the clinical trial?
Clinic for Special ChildrenLead Sponsor
Genentech, Inc.Industry Sponsor