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Risdiplam for Presymptomatic Spinal Muscular Atrophy (Rainbowfish Trial)
Rainbowfish Trial Summary
This trial is testing a new drug for people with Spinal Muscular Dystrophy who don't have any symptoms yet.
Rainbowfish Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowRainbowfish Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Side effects data
From 2023 Phase 2 trial • 231 Patients • NCT02908685Rainbowfish Trial Design
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- My body weight is in the normal range for my age.I have received treatments targeting the SMN2 gene.I can safely travel to the study site for all required visits.My baby or I (if breastfeeding) haven't taken specific drugs affecting liver enzymes recently.I am not taking oral salbutamol or similar drugs for SMA, but I may use inhaled versions.My veins are not suitable for the required blood tests.You have other serious health conditions at the same time.I was born with joint stiffness and/or hip issues.I am open to using breathing support if recommended.My child is between 1 day and 6 weeks old and has not received the first dose before 7 days of age.I need a machine to help me breathe.My oxygen levels drop below 95% when I'm awake, with or without a breathing machine.I have a genetic form of spinal muscular atrophy linked to the SMN1 gene.You have certain heart rhythm problems that could be risky for you to take the study drug.Participants must be within 37 to 42 weeks pregnant for one baby or 34 to 42 weeks pregnant for twins.I have been diagnosed with an eye disease.I don't show strong signs of SMA according to my doctor's opinion.Pregnant women must be between 37-42 weeks pregnant with one baby, or 34-42 weeks pregnant with two babies.I have not shown signs or symptoms of SMA according to my doctor's opinion.My child was not exposed to drugs harmful to the eyes during pregnancy or breastfeeding.My caregiver agrees to consider tube feeding if recommended.You are allergic to risdiplam or any of the ingredients in its formulation.I have a genetic form of spinal muscular atrophy linked to the SMN1 gene.My child is between 1 day and 6 weeks old and has not received the first dose before 7 days of age.Your lab test results show important health problems.My body weight is at or above the 3rd percentile for my age.Your blood pressure or heart rate is too high or too low, according to the doctor.
- Group 1: Open-label Risdiplam
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- 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.
Frequently Asked Questions
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."
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."
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."
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"
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."
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."
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."
Who else is applying?
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What portion of applicants met pre-screening criteria?
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