Recruiting Studies
Advanced SearchData: ClinicalTrials.gov · Source of record for eligibility and locations
Top Cities for Gaucher Disease Clinical Trials
Gaucher Disease clinical trials are recruiting across 21 cities. Here are the cities with the most active studies:
Gaucher Disease Trial Phases
6 research organizations are sponsoring gaucher disease trials.
About Gaucher Disease
Gaucher disease is an inherited metabolic disorder caused by deficiency of the enzyme glucocerebrosidase, leading to accumulation of fatty substances in organs. It is the most common lysosomal storage disorder. Treatment includes enzyme replacement therapy and substrate reduction therapy.
Clinical trials are advancing new treatments for gaucher disease. Currently, 8 studies are recruiting a combined 13,586 participants across the United States. Research is being conducted by 6 organizations including Genzyme, a Sanofi Company, Shire, Duke University and 3 others.
2026 Gaucher Disease Research Landscape
As of July 2026, the gaucher disease clinical trial landscape includes 8 actively recruiting studies across 21 cities in the United States. These studies are collectively seeking 13,586 participants, with an average enrollment target of 1,698 per study.
The research spans multiple phases of development: 1 study is in PHASE3, 1 study is in PHASE1, 1 study is in PHASE2. The presence of Phase 3 trials indicates that some gaucher disease treatments are in late-stage testing and may be approaching regulatory review.
Research is being led by 6 different organizations, including Genzyme, a Sanofi Company, Shire, Duke University, Spur Therapeutics, University of Minnesota, and 1 others.
Geographically, gaucher disease trials are most concentrated in Minneapolis, Minnesota (4 trials); Los Angeles, California (3 trials); Durham, North Carolina (3 trials); San Francisco, California (2 trials); Phoenix, Arizona (1 trial) and 7 other cities.
Featured Gaucher Disease Studies
Highlighted recruiting studies for gaucher disease, selected by enrollment size and research scope.
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The objectives of the Re...
Gaucher Disease Outcome Survey (GOS)
The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received. No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician. The objectives of the registry includ...
A Long-term Follow-up Study of Gaucher Disease
The study aims are to: a) identify the long-term natural history of Gaucher disease, b) evaluate long-term treatment efficacy of enzyme replacement therapy (ERT) and substrate reduction therapy (SRT), and c) identify potential long-term complications of this disorder. These aims will be accomplished through long-term record review of individuals with all three types of Gaucher disease.
Frequently Asked Questions About Gaucher Disease Clinical Trials
Are there gaucher disease clinical trials near me?
Yes, there are 8 gaucher disease clinical trials currently recruiting across 21+ cities in the United States, including Minneapolis, Minnesota; Los Angeles, California; Durham, North Carolina. Browse the studies above to find one at a location convenient for you.
How do I join a gaucher disease clinical trial?
To join a gaucher disease clinical trial: 1) Browse the available studies on this page, 2) Click on a study that interests you, 3) Check the study locations to find a site near you, 4) Review the eligibility criteria, and 5) Contact the study site or complete the eligibility form. The process is free and you can withdraw at any time.
Are gaucher disease clinical trials free?
Yes, participation in gaucher disease clinical trials is free. Study-related treatments, medical tests, and doctor visits are provided at no cost to participants. Many studies also offer compensation for your time and travel expenses.
What types of gaucher disease treatments are being studied?
Current gaucher disease clinical trials are testing a range of approaches across PHASE3 (1 study), PHASE1 (1 study), PHASE2 (1 study). These include new drugs, combination therapies, medical devices, and other interventions sponsored by 6 research organizations.
Is it safe to participate in gaucher disease clinical trials?
Clinical trials are carefully regulated by the FDA and institutional review boards (IRBs). All trials must follow strict safety protocols, and participants receive close medical monitoring throughout the study. You can withdraw from a trial at any time without penalty.
Data updated July 16, 2026 from ClinicalTrials.gov
About This Data
Data: ClinicalTrials.gov · Source of record for eligibility and locations
Clinical trial information on this page is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health (NIH) and National Library of Medicine (NLM). Study data is refreshed every hour to ensure accuracy.
Medical Disclaimer: The information provided on this page is for informational purposes only and does not constitute medical advice. Always consult with a qualified healthcare provider before making decisions about clinical trial participation or changes to your treatment plan.
Page reviewed by the HelloStudys Research Team · Last updated July 16, 2026 · Data from ClinicalTrials.gov