Clinical trials · California
Gaucher Disease clinical trials in California
We’re tracking 5 recruiting gaucher disease studies with a site in California — including Phase 1 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies in California
- 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 - NCT00358943Recruiting
Gaucher disease registry and pregnancy tracking study
This study creates a large, long-term record of people with Gaucher disease and details about pregnancy outcomes. It helps researchers better understand Gaucher over a person’s life and during pregnancy.
Irvine, CaliforniaAges Any age - NCT06539169Recruiting
Following people with rare diseases over time
This study follows people with rare diseases over time to learn more about how these conditions progress and how they are treated. Joining may help researchers understand your disease better and find better ways to care for others.
Los Altos, CaliforniaAges Any age - 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+ - NCT05619900Recruiting
Register people with lysosomal storage diseases
This study is a registry that collects information from people diagnosed with lysosomal storage diseases. It helps researchers better understand these conditions and support future studies.
San Francisco, CaliforniaAges Up to 64 years
Where these studies are running in California
Institutions with a site for the recruiting gaucher disease studies listed above.
- University of California, San Francisco · 3 studies
- Children's Hospital of Orange County
- Kaiser Permanente Los Angeles Medical Center
- Kaiser Permanente Sacramento Medical Center
- Southern California Permanente Medical Group
- Stanford University
- Tower Hematology Oncology Medical Group
- University of California
What taking part in a gaucher disease study involves
A screening visit first
Before anything else, the study team checks whether you fit — usually a visit with some tests. You can stop at any point, and screening is typically free.
Care at a nearby site
Study visits happen at a clinic or hospital taking part. Many studies cover the cost of the study treatment and related visits, and some reimburse travel.
You stay in control
Taking part is voluntary and you can leave a study at any time, for any reason, without affecting your regular care.
The team decides eligibility
Our fit check is a helpful first read, not a decision. The study team makes the final call after reviewing your health history.
Gaucher Disease trials in other states
Common questions
- Are there gaucher disease clinical trials in California?
- Yes. We're currently tracking 5 recruiting gaucher disease studies with a site in California, each rewritten in plain language so you can see what it's testing and who it's for.
- How do I find out if I qualify for a gaucher disease study in California?
- Each study lists its eligibility rules — age, diagnosis, prior treatments. On every trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Do I have to live in California to take part?
- Not necessarily. These studies have a site in California, but eligibility is about your health, not your address — some people travel to take part, and a few studies reimburse travel. The study team can tell you what's required.
- Does it cost anything to join a gaucher disease trial?
- Using Clin2 is always free. Many trials cover the study treatment and related visits; some reimburse travel. The study team explains exactly what's covered before you decide.
Not the right time?
New gaucher disease studies open in California regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.
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.