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SMN2 Splicing Modifier

Risdiplam for Presymptomatic Spinal Muscular Atrophy (Rainbowfish Trial)

Phase 2
Waitlist Available
Research Sponsored by Hoffmann-La Roche
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Body weight >= 3rd percentile for age, using appropriate country-specific guidelines
Able and expected to be able to safely travel to the study site for the entire duration of the study and in accordance to the frequency of required study visits, in the opinion of the investigator
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 7 years
Awards & highlights

Rainbowfish Trial Summary

This trial is testing a new drug for people with Spinal Muscular Dystrophy who don't have any symptoms yet.

Who is the study for?
This trial is for infants from birth to 6 weeks old with a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. They must be able to travel safely for study visits, have supportive caregivers, and not require invasive ventilation. Infants should weigh in the normal range for their age and have been born after a full-term pregnancy.Check my eligibility
What is being tested?
The trial is testing an oral medication called Risdiplam on infants who are genetically diagnosed with SMA but haven't shown symptoms. The goal is to see if early treatment can prevent or lessen muscle weakness caused by SMA.See study design
What are the potential side effects?
Risdiplam may cause sensitivity reactions, potential blood test abnormalities, and could interact with other medications affecting liver enzymes or those known for retinal toxicity. It's important that neither the infant nor breastfeeding mother takes certain drugs before joining.

Rainbowfish Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My body weight is in the normal range for my age.
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I can safely travel to the study site for all required visits.
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I am open to using breathing support if recommended.
Select...
My child is between 1 day and 6 weeks old and has not received the first dose before 7 days of age.
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I have a genetic form of spinal muscular atrophy linked to the SMN1 gene.
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I don't show strong signs of SMA according to my doctor's opinion.
Select...
My caregiver agrees to consider tube feeding if recommended.
Select...
I have a genetic form of spinal muscular atrophy linked to the SMN1 gene.
Select...
My child is between 1 day and 6 weeks old and has not received the first dose before 7 days of age.
Select...
My body weight is at or above the 3rd percentile for my age.

Rainbowfish Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 7 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 7 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
Percentage of Participants With Two Copies of the Survival Motor Neuron (SMN) 2 Gene (Excluding the Known SMN2 Gene Modifier Mutation c.859G>C) and Baseline Compound Muscle Action Potential (CMAP) >=1.5 Millivolt (mV) Who Are Sitting Without Support
Secondary outcome measures
Change From Baseline Percentiles for Head Circumference- For-age
Change From Baseline Percentiles for Weight-for-age, Length/Height-for-age, and weight-for- Length/Height
Change From Baseline Score in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale at Month 12
+24 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%
Ear pain
29%
Pain in extremity
29%
Ligament sprain
29%
Vomiting
29%
Respiratory tract infection
29%
Headache
14%
Abdominal pain
14%
Myalgia
14%
Gastrointestinal infection
14%
Chronic respiratory failure
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

Rainbowfish Trial Design

1Treatment groups
Experimental Treatment
Group I: Open-label RisdiplamExperimental Treatment1 Intervention
Participants will be enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range.
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?

Hoffmann-La RocheLead Sponsor
2,426 Previous Clinical Trials
1,089,143 Total Patients Enrolled
Clinical TrialsStudy DirectorHoffmann-La Roche
2,197 Previous Clinical Trials
888,571 Total Patients Enrolled

Media Library

Risdiplam (SMN2 Splicing Modifier) Clinical Trial Eligibility Overview. Trial Name: NCT03779334 — Phase 2
Spinal Muscular Atrophy Research Study Groups: Open-label Risdiplam
Spinal Muscular Atrophy Clinical Trial 2023: Risdiplam Highlights & Side Effects. Trial Name: NCT03779334 — Phase 2
Risdiplam (SMN2 Splicing Modifier) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03779334 — Phase 2

Frequently Asked Questions

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

Has the FDA issued a formal authorization for Risdiplam?

"The safety of risdiplam was evaluated as a 2 since it is in the Phase 2 trial stage, meaning that while there has been some data collected on its safety profile, no information exists yet confirming efficacy."

Answered by AI

To what extent have there been experiments conducted with Risdiplam?

"In 2016, the first clinical trials of risdiplam were conducted at Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie. So far 5 trails have been fully completed, with several others in progress across multiple locations including New york City."

Answered by AI

Is this an innovative research study?

"At present, there have been 6 clinical trials for Risdiplam conducted across 23 cities and 22 countries since its initial study in 2016. Sponsored by Hoffmann-La Roche, the original trial entailed 231 participants and concluded with both Phase 2 & 3 stages of drug approval. Subsequently, 5 other studies were carried out."

Answered by AI

Is there a possibility of me participating in this experimentation?

"This clinical trial seeks to enlist 25 neonates (1 Day - 6 Weeks) with muscular atrophy, spinal. These infants need to fulfill the following criteria: males and females aged from birth (1 day) to 6 weeks of age at first dose; a minimum age of 7 days for the primary enrollee; gestational ages ranging between 37-42 weeks for single births and 34-42 weeks for twins; body weight above 3rd percentile relative to their age group according to local guidelines; genetic diagnosis confirming homozygous deletion or compound heterozygosity associated with SMN1 gene loss of function; no visible signs or symptoms that could"

Answered by AI

Are there any available vacancies in this trial for participants?

"This clinical trial is no longer enrolling participants. It was initially posted on August 7th 2019 and was last edited September 29th 2022. Patients searching for alternative studies can consider 205 trials researching muscular atrophy or 6 Risdiplam studies actively seeking enrolment."

Answered by AI

Does this clinical research include participants aged thirty and above?

"Consistent with the entry requirements for this trial, the youngest participant can be 1 Day old and the oldest 6 Weeks."

Answered by AI

What is the upper limit of people participating in this exploration?

"This clinical trial is no longer accepting new participants. It was first uploaded on the 8th of July, 2019 and was last edited on September 29th, 2022. If you are seeking other options for medical studies, there are 205 active trials related to muscular atrophy currently recruiting patients as well as 6 Risdiplam trials welcoming enrollees."

Answered by AI

Who else is applying?

How old are they?
< 18
18 - 65
What site did they apply to?
London Health Sciences Centre; Children's Hospital; Pediatrics
What portion of applicants met pre-screening criteria?
Met criteria
What state do they live in?
California
~5 spots leftby Mar 2025