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Risdiplam for Spinal Muscular Atrophy (Jewelfish Trial)
Jewelfish Trial Summary
This trial is exploring the safety and effectiveness of a new drug for Spinal Muscular Atrophy, a rare disease that causes muscle weakness.
Jewelfish Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowJewelfish 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 • NCT02908685Jewelfish Trial Design
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- I have a history of cancer that is not considered cured.I have previously used chloroquine, hydroxychloroquine, retigabin, vigabatrin, or thioridazine.I have had an eye disease in the last year.You are suspected of using drugs regularly.You have recently tested positive for drugs or alcohol in your urine.I haven't had a serious illness or fever in the last month.I started treatment for spinal muscular atrophy with a specific medication less than 6 weeks ago.You are allergic to risdiplam or any ingredients in it.I have previously participated in the Moonfish study or have been treated with specific drugs for my condition.I agree to not have sex or use birth control and not donate sperm.You cannot meet the requirements of the study.I have not had gene or cell therapy, except for AVXS-101.I am willing to consider tube feeding and non-invasive ventilation for my child under 2, as recommended.I have heart issues shown on an ECG or have cardiovascular disease.As a caregiver, I am open to considering tube feeding and non-invasive ventilation for my child if recommended.I have been part of the Moonfish study or treated with Nusinersen, Olesoxime, or AVXS-101.I agree to either not have sex or use birth control, and not donate sperm.My child under 2 was hospitalized for a lung problem recently and hasn’t fully recovered.I don't have major issues with my digestive, kidney, liver, hormone, or heart systems.I am over 6 years old and at high risk for suicidal behavior.I haven't taken any drugs affecting FMO1 or FMO3 enzymes in the last 2 weeks.I have been diagnosed with 5q-autosomal recessive SMA.
- Group 1: 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
What is the sample size of participants taking part in this trial?
"Unfortunately, no more participants are being solicited for this investigation. First posted on March 3rd 2017 and last revised October 26th 2022, recruitment is now closed. However, if you’re looking to join another study related to muscular atrophy or spinal cord injury there are 205 trials still open and 6 clinical trials recruiting patients with Risdiplam as a treatment option."
Are there any particular demographics that are more suitable for enrollment in this clinical trial?
"The medical study is searching for 174 patients between 6 months and 60 years of age who are afflicted with muscular atrophy. Furthermore, these individuals must satisfy the following prerequisites: Previous treatment or enrollment in Study BP29420 (Moonfish) with splicing modifier RO6885247, recuperation from illness during screening time period according to Investigator's opinion; Women of childbearing potential will need negative pregnancy test results alongside agreeing to abstain from sexual intercourse using contraception measures as well as refraining from donating eggs 28 days after final drug dose; Men that meet criteria require abstinence through contraceptive methods while also declining sperm donation offers; Lastly, participants"
How many facilities are currently hosting this research trial?
"Patients are able to enroll in this clinical trial at multiple sites, including Nemours Children's Hospital (Orlando, Florida), Boston Childrens Hospital (Boston, Massachusetts), and Columbia University Medical Center; The Neurological Institute of New york (New York, New York)."
Is eligibility for this medical experiment restricted to adults above a certain age?
"This clinical trial only admits participants aged between 6 months and 60 years. There are 52 trials for those younger than 18, while 150 are available to the elderly population over 65."
To what extent has Risdiplam been proved to be innocuous for patients?
"Our team at Power rated the safety of Risdiplam a 2, as this is presently in Phase 2 trials and only preliminary data exists confirming its security but not efficacy."
Is there still capacity for participation in this experiment?
"This medical trial is no longer accepting patients, as it was last updated on October 26th 2022. If you are looking for alternative research opportunities, 205 trials related to muscular atrophy and 6 studies concerning Risdiplam are currently recruiting participants."
Could you please describe any other experiments involving Risdiplam?
"Risdiplam was initially trialled in 2016 at Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie, and 5 studies have since been completed. At present, 6 trials are live worldwide with a concentration of sites located in Orlando Florida."
Does this research project present any groundbreaking discoveries?
"Presently, there are 6 live trials involving Risdiplam that span 22 countries and 23 cities. This drug was initially tested in a Phase 2 & 3 study sponsored by Hoffmann-La Roche which included 231 patients and concluded in 2016 - since then 5 other studies have been completed."
Who else is applying?
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