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Small Molecule

Risdiplam for Spinal Muscular Atrophy (RISE Trial)

Phase 4
Waitlist Available
Research Sponsored by Clinic for Special Children
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Biallelic SMN1 deletions
3 or 4 copies of SMN2
Must not have
Prior treatment with SMN gene replacement therapy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 36 months
Awards & highlights
Pivotal Trial
Drug Has Already Been Approved
No Placebo-Only Group

Summary

This trial is for SMA patients who have been treated with nusinersen and are now crossover to 36 months of risdiplam monotherapy. The trial will track the most informative outcomes from the nusinersen trial, while adding the Box and Block Test as an additional measure of upper limb endurance and function.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While specific side effects are not listed here, risdiplam may cause similar side effects as other medications used to treat SMA which can include fever, headache, vomiting, back pain, joint pain and respiratory infections.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have a genetic condition with two missing SMN1 genes.
Select...
My genetic test shows I have 3 or 4 copies of the SMN2 gene.
Select...
I have been treated with nusinersen for at least 22 months.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have previously received SMN gene therapy.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~36 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 36 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Comparative intrasubject performance on nine hole peg test (NHPT)
Secondary study objectives
Comparative intrasubject change in grip strength
Frequency and type of adverse events
Intrasubject change in pulmonary function
+2 more

Side effects data

From 2023 Phase 2 trial • 231 Patients • NCT02908685
22%
Nasopharyngitis
15%
Upper respiratory tract infection
13%
Vomiting
13%
Pyrexia
10%
Headache
10%
Cough
9%
Diarrhoea
8%
Gastroenteritis
7%
Pneumonia
6%
Respiratory tract infection
6%
Abdominal pain
5%
Bronchitis
5%
Pharyngitis
5%
Rash
4%
Ear pain
3%
Nausea
3%
Back pain
3%
Arthralgia
3%
Influenza
3%
Limb injury
3%
Oropharyngeal pain
3%
Rhinorrhoea
3%
Eczema
3%
Constipation
3%
Influenza like illness
3%
Ear infection
2%
Contusion
2%
Erythema
2%
Urinary tract infection
2%
Sinusitis
2%
Arthropod bite
2%
Gastritis
2%
Decreased appetite
2%
Pain in extremity
2%
Dry skin
2%
Pruritus
2%
Gastrointestinal infection
1%
Ocular hyperaemia
1%
Infective thrombosis
1%
Encephalitis
1%
Brain contusion
1%
Nephrolithiasis
1%
Atelectasis
1%
Neck pain
1%
Amenorrhoea
1%
Dysmenorrhoea
1%
Pneumonia mycoplasmal
1%
Rhinitis
1%
Scarlet fever
1%
Tonsillitis
1%
Dehydration
1%
Device related infection
1%
Herpes zoster
1%
Post procedural infection
1%
Pyelonephritis
1%
Viral upper respiratory tract infection
1%
Partial seizures
1%
Haematuria
1%
Pneumonitis aspiration
1%
Pneumothorax
1%
Dizziness
1%
Nasal congestion
1%
Rhinitis allergic
1%
Tachycardia
1%
Asthma
1%
Epistaxis
1%
Productive cough
1%
Acne
1%
Alopecia
1%
Blister
1%
Dermatitis
1%
Abdominal pain upper
1%
Aphthous ulcer
1%
Asthenia
1%
Seborrhoeic dermatitis
1%
Hypersensitivity
1%
Conjunctivitis
1%
Cystitis
1%
Groin infection
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part 2 OLT: Risdiplam/Risdiplam
Part 2 OLT: Placebo/Risdiplam
Part 2 OLE: Risdiplam
Part 1 Group B: Children (0.25 mg/kg Risdiplam)
Part 1 Group A: Adolescents and Adults (3 mg Risdiplam)
Part 1 Group A: Adolescents and Adults (5 mg Risdiplam)
Part 1 Group B: Children (Placebo-Control Period Pooled)
Part 1 Group A: Adolescents and Adults (Placebo-Control Period Pooled)
Part 1 Group B: Children (0.02 mg/kg Risdiplam)
Part 1 Group B: Children (0.05 mg/kg Risdiplam)
Part 1 Group B: Children (0.15 mg/kg Risdiplam)
Part 1 Group A: OLE
Part 1 Group B: OLE
Part 2 Placebo-Controlled: Risdiplam
Part 2 Placebo-Controlled: Placebo

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Drug Has Already Been Approved
The FDA has already approved this drug, and is just seeking more data.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Risdiplam
2016
Completed Phase 2
~420

Find a Location

Who is running the clinical trial?

Clinic for Special ChildrenLead Sponsor
1 Previous Clinical Trials
40 Total Patients Enrolled
Genentech, Inc.Industry Sponsor
1,565 Previous Clinical Trials
570,046 Total Patients Enrolled

Media Library

Risdiplam (Small Molecule) Clinical Trial Eligibility Overview. Trial Name: NCT05522361 — Phase 4
Spinal Muscular Atrophy Research Study Groups: Open-label crossover
Spinal Muscular Atrophy Clinical Trial 2023: Risdiplam Highlights & Side Effects. Trial Name: NCT05522361 — Phase 4
Risdiplam (Small Molecule) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05522361 — Phase 4
Spinal Muscular Atrophy Patient Testimony for trial: Trial Name: NCT05522361 — Phase 4
~3 spots leftby Dec 2025
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