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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 belowSelect...
I have been diagnosed with 5q-autosomal recessive SMA.
WeSMA Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
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 • NCT0290868522%
Nasopharyngitis
15%
Upper respiratory tract infection
13%
Vomiting
13%
Pyrexia
10%
Headache
10%
Cough
9%
Diarrhoea
8%
Gastroenteritis
7%
Pneumonia
6%
Abdominal pain
6%
Respiratory tract infection
5%
Bronchitis
5%
Pharyngitis
5%
Rash
4%
Ear pain
3%
Constipation
3%
Nausea
3%
Influenza like illness
3%
Ear infection
3%
Influenza
3%
Limb injury
3%
Arthralgia
3%
Back pain
3%
Rhinorrhoea
3%
Eczema
3%
Oropharyngeal pain
2%
Gastritis
2%
Gastrointestinal infection
2%
Urinary tract infection
2%
Sinusitis
2%
Arthropod bite
2%
Contusion
2%
Decreased appetite
2%
Pain in extremity
2%
Dry skin
2%
Pruritus
2%
Erythema
1%
Dehydration
1%
Pneumonia mycoplasmal
1%
Device related infection
1%
Encephalitis
1%
Herpes zoster
1%
Infective thrombosis
1%
Post procedural infection
1%
Pyelonephritis
1%
Tonsillitis
1%
Viral upper respiratory tract infection
1%
Brain contusion
1%
Partial seizures
1%
Haematuria
1%
Atelectasis
1%
Pneumonitis aspiration
1%
Pneumothorax
1%
Tachycardia
1%
Ocular hyperaemia
1%
Abdominal pain upper
1%
Aphthous ulcer
1%
Asthenia
1%
Hypersensitivity
1%
Conjunctivitis
1%
Cystitis
1%
Groin infection
1%
Rhinitis
1%
Scarlet fever
1%
Asthma
1%
Epistaxis
1%
Productive cough
1%
Acne
1%
Alopecia
1%
Blister
1%
Neck pain
1%
Dizziness
1%
Amenorrhoea
1%
Dysmenorrhoea
1%
Dermatitis
1%
Nasal congestion
1%
Rhinitis allergic
1%
Seborrhoeic dermatitis
1%
Nephrolithiasis
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
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
1,541 Previous Clinical Trials
567,525 Total Patients Enrolled
Clinical TrialsStudy DirectorHoffmann-La Roche
2,202 Previous Clinical Trials
888,414 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have been diagnosed with 5q-autosomal recessive SMA.You have been part of a specific trial for risdiplam.I have been prescribed risdiplam after its FDA approval on 07 August 2020.You are allergic to risdiplam.
Research Study Groups:
This trial has the following groups:- Group 1: Risdiplam
Awards:
This trial has 3 awards, including:- 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.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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
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