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Venglustat for Tay-Sachs Disease (AMETHIST Trial)
AMETHIST Trial Summary
This trial is studying venglustat to see how well it works in treating late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or galactosialidosis.
AMETHIST Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowAMETHIST Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.AMETHIST Trial Design
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Who is running the clinical trial?
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- I do not have any health conditions that would make the trial unsafe for me.I can take pills, travel to the study site, and follow the study's requirements.My seizures are under control with medication that doesn't strongly affect CYP3A4.I am currently taking medication that could affect my vision or worsen cataracts.I have been diagnosed with a specific genetic disorder affecting my nervous system.I haven't taken any experimental drugs recently.I have symptoms of Tay-Sachs or Sandhoff disease not due to HEXA or HEXB gene mutations.I haven't taken certain drugs, including grapefruit products, before joining the study.I can complete a hand function test in less than or equal to 240 seconds.I am 18 years old or older.I am between 2 and 18 years old and weigh at least 10 kg.I have a significant cataract in my eye, not just in the center.My liver tests are within normal limits, or I have Gilbert syndrome with specific bilirubin levels.My kidney function is low, with an eGFR below 30 mL/min/1.73m2.I need a machine to help me breathe.You have been diagnosed with hepatitis B, hepatitis C, or HIV.If you're sexually active, you need to use birth control. You must not be pregnant, breastfeeding, or donating sperm.
- Group 1: GZ402671
- Group 2: Placebo
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- 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
Across how many different medical facilities is this research being conducted?
"There are 4 sites recruiting patients for this clinical trial, including Ataxia Center and HD Center of Excellence in Los Angeles, Emory Genetics in Atlanta, and NIH National Human Genome Research Institute in Bethesda. There are also other sites in 4 other locations."
What is the venglustat GZ402671 FDA approval status?
"There is clinical evidence backing up venglustat GZ402671's efficacy, making it a 3 on our team's safety scale."
What other research exists that is similar to this investigation?
"First studied in 2017, venglustat GZ402671's first clinical trial was sponsored by Genzyme. After the initial study with 13 participants, Phase 2 drug approval was received and now there are 14 different countries with 12 cities running active trials."
Are new patients currently being accepted into this trial?
"No, this specific trial is not looking for patients at the present moment. Although, according to the clinicaltrials.gov website, it was last edited on 11/2/2022. There are 9 other trials that might be seeking patients right now."
What goals does this experiment hope to realize?
"The purpose of this study, which will be conducted over the course of 104 weeks, is to evaluate change in cerebrospinal fluid (CSF) GM2 biomarker. Additionally, the investigation will assess venglustat's maximum concentration in plasma (Cmax), time 0 to 24 hour concentration versus time curve (AUC0-24h), and other pharmacokinetic parameters."
Are there other examples of venglustat GZ402671 being used in medical research?
"There are currently two ongoing clinical trials investigating venglustat GZ402671, with one trial in Phase 3. While several of the studies for venglustat GZ402671 are located in Praha 2 and Massachusetts, there are a total of 35 locations operating clinical trials for venglustat GZ402671."
What is the maximum capacity for this clinical trial?
"This particular trial is not enrolling patients at the moment. The listing was originally created on 6/29/2020 but was updated as recently as 11/2/2022. There are, however, 7 other trials for tay-sachs disease and 2 studies involving venglustat GZ402671 that are actively looking for participants."
Who else is applying?
What state do they live in?
What site did they apply to?
What portion of applicants met pre-screening criteria?
How many prior treatments have patients received?
Why did patients apply to this trial?
What questions have other patients asked about this trial?
How responsive is this trial?
Typically responds via
Most responsive sites:
- Emory Genetics - Investigational site number 8400006: < 48 hours
Average response time
- < 2 Days
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