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SMN2 Splicing Modifier

Risdiplam for Spinal Muscular Atrophy(WeSMA Trial)

Norton Children's Hospital, Louisville, KY
RisdiplamPhase 4RecruitingResearch Sponsored by Genentech, Inc.

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.

Eligible Conditions
  • Spinal Muscular Atrophy

WeSMA Trial Eligibility Criteria

Inclusion Criteria

You will be eligible if you check “Yes” for the criteria below
You have been diagnosed with a specific genetic disorder called 5q-autosomal recessive SMA.
You are currently taking or have been recommended to take risdiplam after it was approved by the FDA.

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
Upper respiratory tract infection
Ear pain
Pain in extremity
Ligament sprain
Respiratory tract infection
Chronic respiratory failure
Gastrointestinal infection
Urinary tract infection
Oropharyngeal pain
Ear infection
Respiratory tract inflammation
Palmar erythema
Skin exfoliation
Hand-foot-and-mouth disease
Decreased appetite
Abdominal pain
Upper respiratory tract inflammation
Dry skin
Nasal dryness
Abdominal pain upper
Allergy to arthropod bite
Femur fracture
Musculoskeletal chest pain
Eczema eyelids
Limb injury
Erythema infectiosum
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

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.

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.
First Studied
Drug Approval Stage
How many patients have taken this drug
Completed Phase 2

Find a Location

Who is running the clinical trial?

Genentech, Inc.Lead Sponsor
1,531 Previous Clinical Trials
566,267 Total Patients Enrolled
Clinical TrialsStudy DirectorHoffmann-La Roche
2,191 Previous Clinical Trials
888,354 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.

What is atrophy?

"Atrophy may occur in patients with normal intelligence and without dementia who have a history of alcohol abuse. Patients with atrophy also develop a behavioral pattern of alcohol abuse. It is necessary to examine neuropsychological tests in patients with atrophy in order to identify whether their pattern of neuropsychological decline can be associated with the development of atrophy."

Answered by AI

Can atrophy be cured?

"Patients with atrophy should be carefully monitored with serial medical imaging for progression of atrophy or recurrence of the pathology. In most patients, atrophy does not respond to or will recur after atrophy therapy; however, a few patients may show sustained or improved atrophy after atrophy therapy, which may help them to attain a higher quality of life. The natural history of atrophy warrants further research and attention."

Answered by AI

What causes atrophy?

"Atrophy is an ongoing process that may result from aging or be the result of pathological events such as chronic kidney disease, systemic vasculitis, and some types of cancer."

Answered by AI

What are the signs of atrophy?

"There is no single sign that indicates that an organ is at risk of atrophy. Rather, subtle changes that progress over time may alert health professionals to the possibility of a problem. These changes include loss of body surface area (e.g., thigh muscle wasting or muscle twirling); loss of lean body mass; and loss of strength. These changes often precede the development of a nutritional, biochemical, or physiological imbalance. When evaluating a patient with unexplained weight loss, it is also important to consider the changes in nutrition and activity that are commonly associated with muscle wasting (e.g., a sedentary or anorexic patient). Appropriate assessment for these changes helps to identify potential causes of atrophy for treatment."

Answered by AI

What are common treatments for atrophy?

"Atrophy can be treated in a variety of ways. One common therapy is deep brain stimulation and is typically used to treat Parkinson's disease and dystonia."

Answered by AI

How many people get atrophy a year in the United States?

"About 3 million to 4 million US residents had severe AA changes in 2005. These individuals have a 30 to 40% greater risk of dying by year 10 compared with the group at risk for AA changes not affected by atrophy. The greatest increase in risk is among individuals with more severe atrophy changes, a finding consistent with current perspectives on the natural history of AA."

Answered by AI

How serious can atrophy be?

"Patients with severe atrophy appear to be the most severely affected with a high risk for developing chronic kidney disease. These patients should be identified and advised."

Answered by AI

What is the latest research for atrophy?

"It seems that the research on atrophy is an active area of research, which needs more studies and trials. There are many things to study, such as causes, diagnostics, treatments, and side effects. Currently, one of the ways of diagnosing atrophy is by studying the changes in the muscle mass or the changes in the muscle fibers. Current research seems to show that atrophy is related to many different factors, especially with nutrition and some medications, and we want to learn more about these factors, such as nutrition, how to control it (dietary management), how to treat it (drugs), and how it causes the atrophy differently."

Answered by AI

Is risdiplam safe for people?

"Despite no contraindications for risdiplam, subjects taking risdiplam need to be monitored carefully due to a risk of gastrointestinal bleeding. In contrast, some subjects taking risdiplam for more than 3 months do not need routine monitoring and their use is justified."

Answered by AI

What are the latest developments in risdiplam for therapeutic use?

"When considering the need for new therapeutic approaches to diabetic retinopathy, it is important to remember that retinal damage is a progressive and time-dependent process. Therefore it is equally important to consider the timing and type of treatment used."

Answered by AI

Is risdiplam typically used in combination with any other treatments?

"Atrophy is an established indication for which risdiplam has been used therapeutically. There is evidence that risdiplam may be useful for treating atrophotic acne scars when given for 24 weeks to individuals who require more skin tightening than can be achieved by the use of only the atrophotic collagen inducer, tridecyl sulfosuccinic acid (CISSA). Atrophic scars are commonly found on the cheeks, chest, stomach, and arms of patients ages 20 to 40 years old, although other areas can have atrophic scars as well. Although, it remains unclear what role risdiplam may play in atrophic acne scars, it may be a possible treatment option for some scars."

Answered by AI

What does risdiplam usually treat?

"Risdiplam is a drug used to treat superficial fungal infections. It consists of three ingredients, and can be classified in an oral, topical, and intravenous form. The oral form is not useful in CLL. In all forms, risdiplam causes an increase in retinal ganglion cells by inducing apoptosis, particularly in photoreceptor cells. This increase was more pronounced in the intravenous form than in the topical form. The mechanism of action of risdiplam in CLL has not been fully elucidated."

Answered by AI

Is there availability to join this experiment as a participant?

"Affirmative, clinicaltrials.gov provides evidence that this investigation is actively enrolling participants. This study was first publicised on March 29th 2022 and has been recently updated as of November 22nd 2022. 500 individuals must be recruited from 14 different sites for the trial to succeed."

Answered by AI

How many research facilities have been assigned to conduct this experiment?

"This medical trial is being conducted in the cities of Cincinnati, New York, and Round Rock with additional sites spread across all other 14 locations. Specifically, it is based out of Cincinnati Childrens Hospital in Ohio, Columbia University Med Center in New York City and Central Texasn Ohio, Columbia University Med Center in New York City and Central Texas Neurology Consultants in Texas respectively."

Answered by AI

What is the maximum amount of participants that are being recruited for this medical study?

"This medical study necessitates 500 participants, who comply with the given criteria for participation. The trial is being held across multiple centres, including Cincinnati Childrens Hospital in Ohio and Columbia University Med Center in New Yorkn Ohio and Columbia University Med Center in New York City."

Answered by AI

Has Risdiplam been granted approval by the US Food and Drug Administration?

"As this is an authorized Phase 4 trial, we consider Risdiplam to be highly safe and score it a 3 on our 1-3 scale."

Answered by AI

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?
Met criteria
Did not meet criteria
What state do they live in?
How many prior treatments have patients received?

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
~333 spots leftby Jun 2029