Clinical trials
Gaucher Disease Type 1 clinical trials
Below are recruiting gaucher disease type 1 clinical trials, each written for real people, not researchers. We’re tracking 10 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT04532047RecruitingPhase 1
Fetal enzyme therapy trial for certain inherited storage disorders
This Phase 1 trial tests an enzyme replacement treatment given to a developing baby before birth for specific inherited “lysosomal storage” diseases. The goal is to see if the treatment is safe and can improve outcomes for babies diagnosed in pregnancy.
San Francisco, CaliforniaAges 18–50 - NCT02437396Recruiting
Blood tests in stable adults with Gaucher disease
This study measures inflammation and “oxidative stress” (cell stress caused by imbalance in the body) using blood biomarkers in adults with Gaucher disease. It may help researchers understand what’s happening in the body and how treatment status relates to these blood signals.
Minneapolis, MinnesotaAges 18–75 - NCT05253560Recruiting
Study prodromal Parkinson signs in GBA1 mutation carriers
This study looks at early (before full disease) Parkinson-like symptoms in people who carry a specific genetic change called GBA1. It may help researchers understand who could develop Parkinson’s and how early signs can be tracked.
Jerusalem, Please Select...Ages 40–75 - NCT06162338Recruiting
Study of LY-M001 for Gaucher Disease Type I
This study tests an injection called LY-M001 for adults with Gaucher disease type I. The goal is to see if it can safely help replace the missing enzyme and improve symptoms.
Hanzhou, ZhejiangAges 18–60 - NCT06272149RecruitingEarly Phase 1
Gene Therapy for Infants With Type 2 Gaucher Disease
This trial tests a new gene therapy (VGN-R08b) for infants with Type 2 Gaucher disease, a rare genetic condition that affects the brain and body. The goal is to see if the treatment is safe and can help improve symptoms.
Shanghai, Shanghai MunicipalityAges birth–2 years - NCT06818838RecruitingPhase 1/Phase 2
Gene therapy for Type 1 Gaucher disease
This study tests a new gene therapy called LY-M001 for adults with Type 1 Gaucher disease. It aims to help the body produce the missing enzyme and reduce symptoms like low blood counts or enlarged organs.
Guangzhou, GuangdongAges 18–60 - NCT07223944RecruitingPhase 3
Gene therapy trial for Gaucher disease type 1
This trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. It aims to provide a long-term treatment option for people who have been stable on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years.
Los Angeles, CaliforniaAges 18 years+ - NCT03240653Recruiting
Study to sort people with Gaucher disease into groups
This study looks at people with Gaucher disease (types I, II, or III) to better “group” patients for understanding and future research. If you (or your child) have confirmed Gaucher disease, you may be asked to share information and follow a study schedule.
BirminghamAges Any age - NCT06627543Enrolling by invitation
Enzyme replacement therapy for heart health in Gaucher type 3
This study looks at how enzyme replacement therapy affects heart function in children with Gaucher disease type 3. It aims to see if the therapy helps keep the heart healthy.
AsyutAges Any age - NCT04388969Recruiting
Ambroxol study for Gaucher disease and Parkinson symptoms
This trial looks at ambroxol, a medicine, for people who have Gaucher disease and Parkinson disease that is linked to Gaucher. If you fit the condition group, the study may help doctors learn whether ambroxol can improve Parkinson-related symptoms in this specific situation.
Jerusalem, Please Select...Ages Up to 100 years
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Common questions
- Are there clinical trials for gaucher disease type 1?
- Yes. Clin2 currently lists 10 recruiting gaucher disease type 1 studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a gaucher disease type 1 trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a gaucher disease type 1 trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.