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SMN2 Splicing Modifier

Long-Term Risdiplam for Spinal Muscular Atrophy (WeSMA Trial)

Phase 4
Recruiting
Research Sponsored by Genentech, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Confirmed diagnosis of 5q-autosomal recessive SMA
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

WeSMA Trial Summary

This trial will study the long-term safety and effectiveness of the drug risdiplam in patients with spinal muscular atrophy. The trial will last for up to 5 years, and patients will be followed even if they discontinue the drug.

Who is the study for?
This trial is for both adults and children with a confirmed diagnosis of 5q-autosomal recessive Spinal Muscular Atrophy (SMA). Participants must be prescribed risdiplam based on their doctor's judgment. Those allergic to risdiplam or who were in previous registrational trials for the drug cannot join.Check my eligibility
What is being tested?
The study is looking at the long-term safety and effectiveness of a medication called Risdiplam in patients with SMA. It's not comparing it to other treatments, but just following those taking it for up to five years to see how they do over time.See study design
What are the potential side effects?
While specific side effects are not listed here, Risdiplam may cause reactions that can vary from person to person. These could include typical drug-related side effects such as nausea, allergic reactions, or other symptoms depending on individual health conditions.

WeSMA Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been diagnosed with 5q-autosomal recessive SMA.

WeSMA Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Participants With Adverse Events (AEs), Adverse Events of Special Interest (AESIs), and Serious Adverse Events (SAEs)
Secondary outcome measures
Percentage of Participants Considered Improved on the Clinical Global Impression of Change (CGI-C) Scale

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%
0%
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

WeSMA Trial Design

1Treatment groups
Experimental Treatment
Group I: RisdiplamExperimental Treatment1 Intervention
Participants will receive risdiplam prescribed based on clinician judgment, as per the Evrysdi® USPI.
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?

Genentech, Inc.Lead Sponsor
1,540 Previous Clinical Trials
567,429 Total Patients Enrolled
Clinical TrialsStudy DirectorHoffmann-La Roche
2,201 Previous Clinical Trials
888,365 Total Patients Enrolled

Media Library

Risdiplam (SMN2 Splicing Modifier) Clinical Trial Eligibility Overview. Trial Name: NCT05232929 — Phase 4
Spinal Muscular Atrophy Research Study Groups: Risdiplam
Spinal Muscular Atrophy Clinical Trial 2023: Risdiplam Highlights & Side Effects. Trial Name: NCT05232929 — Phase 4
Risdiplam (SMN2 Splicing Modifier) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05232929 — Phase 4
Spinal Muscular Atrophy Patient Testimony for trial: Trial Name: NCT05232929 — Phase 4

Frequently Asked Questions

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

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
Central Texas Neurology Consultants
Childrens Hospital of Philadelphia
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
What state do they live in?
California
How many prior treatments have patients received?
1

Why did patients apply to this trial?

I got zolgensma at 6 months old. I am turning 3 in November. I am hoping the drug will help.
PatientReceived no prior treatments
Recent research and studies
clinicaltrials.govStudy
Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
2022. "Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05232929.
All > Spinal Muscular Atrophy
~333 spots leftby Jun 2029
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