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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
Drug Has Already Been Approved
No Placebo-Only Group
Pivotal 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.
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.
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.

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.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of Participants With Adverse Events (AEs), Adverse Events of Special Interest (AESIs), and Serious Adverse Events (SAEs)
Secondary study objectives
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
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

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.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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
1,565 Previous Clinical Trials
569,556 Total Patients Enrolled
Clinical TrialsStudy DirectorHoffmann-La Roche
2,233 Previous Clinical Trials
901,773 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
~222 spots leftby Dec 2026
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