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Substrate Reduction Therapy

Venglustat for Tay-Sachs Disease (AMETHIST Trial)

Q: 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.

Q: 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.

Q: 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.

Q: 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.

Q: 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.

Q: 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 <a href="https://www.withpower.com/clinical-trials/massachusetts">Massachusetts</a>, there are a total of 35 locations operating clinical trials for venglustat GZ402671.

Q: 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.

Phase 3

Waitlist Available

Research Sponsored by Genzyme, a Sanofi Company

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

For primary population, the participant has the ability to perform the 9-HPT at the screening visit in < = 240 seconds for the 2 consecutive trials of the dominant hand and the 2 consecutive trials of the nondominant hand.

Primary population and adult secondary population: age ≥ 18 years

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 104

Awards & highlights

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.

Who is the study for?

Adults and juveniles with late-onset GM2 gangliosidosis (Tay-Sachs or Sandhoff disease) can join this trial. Adults must be over 18, while juveniles should be at least 2 years old but under 18 and weigh more than 10 kg. Participants need to control any seizures with certain medications, not have other health issues that could interfere, and agree to use contraception if applicable.Check my eligibility

What is being tested?

The study is testing the effectiveness of a drug called Venglustat taken daily for two years compared to a placebo. It will look at how well it works (efficacy), its effects on the body (pharmacodynamics), how it's processed by the body (pharmacokinetics), and its safety in patients with Tay-Sachs or Sandhoff disease.See study design

What are the potential side effects?

While specific side effects are not listed here, common ones for oral medications like Venglustat may include stomach upset, headache, dizziness, fatigue or allergic reactions. Safety assessments will monitor any adverse effects throughout the trial.

AMETHIST Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I can complete a hand function test in less than or equal to 240 seconds.

Select...

I am 18 years old or older.

AMETHIST Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 104

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 104

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 104 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Assessment of PD response in CSF: GL-1 biomarker

Assessment of PD response in CSF: GL-1, GM1 biomarkers

Assessment of PD response in CSF: GL-1, GM1, GM3 biomarkers

+9 more

Secondary outcome measures

Absolute change in CSF GM2 biomarker

Acceptability assessment

Assessment of PK parameters in plasma: AUC0-24h

+8 more

AMETHIST Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: GZ402671Experimental Treatment1 Intervention

Primary population: participant will receive venglustat dose once daily during the primary analysis period (104 weeks) and the open-label extension period (104 weeks). Secondary population: participant will receive venglustat at various doses once daily during the primary analysis period open-label (104 weeks) and the open-label extension period (104 weeks).

Group II: PlaceboPlacebo Group1 Intervention

Primary population: participants will receive placebo once daily during the primary analysis period (104 weeks) and will receive venglustat dose once daily during the open-label extension period (104 weeks).

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Genzyme, a Sanofi CompanyLead Sponsor

524 Previous Clinical Trials

85,568 Total Patients Enrolled

Clinical Sciences & OperationsStudy DirectorSanofi

857 Previous Clinical Trials

2,018,881 Total Patients Enrolled

Media Library

Venglustat (Substrate Reduction Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04221451 — Phase 3

Tay-Sachs Disease Research Study Groups: GZ402671, Placebo

Tay-Sachs Disease Clinical Trial 2023: Venglustat Highlights & Side Effects. Trial Name: NCT04221451 — Phase 3

Venglustat (Substrate Reduction Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04221451 — Phase 3

Tay-Sachs Disease Patient Testimony for trial: Trial Name: NCT04221451 — Phase 3

Frequently Asked Questions

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

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

Who else is applying?

What state do they live in?

Georgia

What site did they apply to?

Emory Genetics - Investigational site number 8400006

What portion of applicants met pre-screening criteria?

Met criteria

How many prior treatments have patients received?

Why did patients apply to this trial?

We are looking for anything that will save our sons life. He is currenltly still talking and moving round, not walking, and seizures are under control and we would like to keep it that way.

PatientReceived 1 prior treatment