Condition
Gaucher Disease Type 1 clinical trials and research
As of November 12, 2026, Find a Trial lists 3 Gaucher Disease Type 1 clinical trials that are recruiting, at sites in 11 countries (most in Israel (2), Argentina (1), Australia (1)). By phase: Phase 3 (1), Phase 1/2 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 3Clinical trials
- 11Countries
Newest Gaucher Disease Type 1 clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| FLT201 gene therapy for adults with Gaucher disease type 1 | Phase 3 | Los Angeles, California and 31 other locations | Recruiting | Nov 3, 2025 |
| Evaluating LY-M001 Injection for Treating Type I Gaucher Disease | Phase 1/2 | Guangzhou, Guangdong and 2 other locations | Recruiting | Feb 11, 2025 |
| Identifying early signs of Parkinson's in Gaucher mutation carriers | — | Jerusalem, Please Select... | Recruiting | Feb 24, 2022 |
All recruiting trials (3)
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FLT201 gene therapy for adults with Gaucher disease type 1
This trial tests a one-time gene therapy called FLT201 in adults with Gaucher disease type 1 who have been on stable enzyme replacement or substrate reduction therapy to see if they can stop those treatments and keep their blood counts and disease control stable.
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Evaluating LY-M001 Injection for Treating Type I Gaucher Disease
This study is testing a new gene therapy injection called LY-M001 to see if it can safely help adults with Type I Gaucher Disease feel better.
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Identifying early signs of Parkinson's in Gaucher mutation carriers
This study is trying to find early signs of Parkinson's disease in people with a specific genetic mutation to see who might be at higher risk of developing the condition.
Quick answers
How many Gaucher Disease Type 1 clinical trials are recruiting right now?
3, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 11 countries. The most sites are in Israel (2 trials), Argentina (1 trial), Australia (1 trial), Brazil (1 trial), Canada (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 3: 1, Phase 1/2: 1. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.