Condition

Gaucher Disease clinical trials and research

• 9Clinical trials
• 38Countries

Newest Gaucher Disease clinical trials

All recruiting trials (9)

• Gaucher Disease: Following Patients on Standard Care to See How It Progresses

  This study will observe people with Gaucher disease who are receiving standard care to see how their condition changes over time and to explore the biology behind persistent or worsening disease.

  Recruiting Yale University · New Haven, Connecticut · NCT07758816

• Registry for Rare Diseases

  This study is creating a detailed registry to collect information on rare diseases like amyloidosis and sarcoidosis to help improve how they are diagnosed and treated.

  Recruiting Hospital Italiano de Buenos Aires · Buenos Aires, Buenos Aires · NCT06573723

• Prevalence of Acid Sphingomyelinase Deficiency and Gaucher Disease in Patients with Monoclonal Gammopathies

  This study is trying to see how common Acid Sphingomyelinase Deficiency and Gaucher Disease are in patients with monoclonal gammopathies, especially those with an enlarged spleen or who have had their spleen removed.

  Recruiting at 23 sites Fundación Española de Hematología y Hemoterapía · Vitoria-Gasteiz, Alava and 22 other locations · NCT05992532

• Exploring extracellular vesicles in Gaucher Disease

  This study is trying to see how certain tiny particles in the body behave differently in people with Gaucher Disease compared to healthy individuals to understand their role in communication between cells and possible links to brain inflammation.

  Recruiting University of Minnesota · Minneapolis, Minnesota · NCT05843552

• Ambroxol for people with Gaucher disease or GBA-related Parkinson's disease

  This project tests whether ambroxol is safe and may help people with Gaucher disease or Parkinson's disease caused by GBA gene changes.

  Recruiting at 3 sites Shaare Zedek Medical Center · Jerusalem, Please Select... and 2 other locations · NCT04388969

• Long-term observation of rare genetic brain disorders

  This study is tracking how rare genetic brain disorders progress over time to better understand their effects on patients.

  Recruiting University of Pittsburgh · Pittsburgh, Pennsylvania · NCT03333200

• Registry for patients with Gaucher Disease

  This study is collecting information from people with Gaucher Disease to see how well different treatments work and to learn more about the disease over time.

  Recruiting Takeda · Lexington, Massachusetts · NCT03291223

• Long-term follow-up for Gaucher disease

  This study looks at how well enzyme replacement therapy and substrate reduction therapy work for people with Gaucher disease over the long term by tracking their health records.

  Recruiting Duke University · Durham, North Carolina · NCT03190837

• Registry tracking outcomes for patients with Gaucher disease

  This study tracks the health and progress of people with Gaucher disease to help improve treatment and care for them, including those who are pregnant.

  Recruiting at 238 sites Sanofi · Phoenix, Arizona and 316 other locations · NCT00358943

Related topics

Quick answers

How many Gaucher Disease clinical trials are recruiting right now?

9, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 38 countries. The most sites are in United States (5 trials), Argentina (2 trials), Israel (2 trials), Australia (1 trial), Belgium (1 trial). Each study page lists its sites and which ones are recruiting.

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.

Describe your situation →