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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 36 months

Awards & highlights

RISE Trial 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.Check my eligibility

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.See study design

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.

RISE Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a genetic condition with two missing SMN1 genes.

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

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

RISE Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 36 months

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Comparative intrasubject performance on nine hole peg test (NHPT)

Secondary outcome measures

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

100%

Pyrexia

57%

Cough

43%

Bronchitis

43%

Upper respiratory tract infection

43%

Nasopharyngitis

43%

Diarrhoea

43%

Gastroenteritis

29%

Ligament sprain

29%

Ear pain

29%

Pain in extremity

29%

Vomiting

29%

Respiratory tract infection

29%

Headache

14%

Abdominal pain

14%

Chronic respiratory failure

14%

Gastrointestinal infection

14%

Myalgia

14%

Malaise

14%

Influenza

14%

Urinary tract infection

14%

Oropharyngeal pain

14%

Rhinorrhoea

14%

Eczema

14%

Ear infection

14%

Respiratory tract inflammation

14%

Palmar erythema

14%

Skin exfoliation

14%

Hand-foot-and-mouth disease

14%

Pollakiuria

14%

Decreased appetite

14%

Tonsillitis

14%

Nausea

14%

Rash

14%

Upper respiratory tract inflammation

14%

Dehydration

14%

Dizziness

14%

Dry skin

14%

Nasal dryness

14%

Hypoglycaemia

14%

Fatigue

14%

Abdominal pain upper

14%

Contusion

14%

Allergy to arthropod bite

14%

Femur fracture

14%

Constipation

14%

Erythema

14%

Musculoskeletal chest pain

14%

Eczema eyelids

14%

Limb injury

14%

Erythema infectiosum

100%

80%

60%

40%

20%

Study treatment Arm

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: Risdiplam

Part 2: Placebo

RISE 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

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Clinic for Special ChildrenLead Sponsor

Genentech, Inc.Industry Sponsor

1,539 Previous Clinical Trials

567,745 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this medical research open to new participants?

"As indicated by the clinicaltrials.gov website, this medical trial is no longer actively seeking candidates to join; it was first posted on September 15th 2022 and last updated August 29th 2022. In its stead, there are 205 other studies recruiting patients at present."

Answered by AI

Is enrollment restricted to those below the age of 75 for this clinical investigation?

"This research protocol stipulates that the ideal participants are between 2 and 35 years of age. 47 studies have been conducted for minors, while 150 trials were designed for individuals over 65."

Answered by AI

May I participate in this research endeavor?

"This trial is recruiting 10 people, aged between 2 and 35 years old, with a diagnosis of spinal muscular atrophy. Applicants need to have Biallelic SMN1 deletions, 3 or 4 copies of the SMN2 gene and prior treatment with Nusinersen for at least 22 months."

Answered by AI