Gaucher disease trials in United States

In United States 17 Also abroad 31

17 trials · From ClinicalTrials.gov, checked 11 November 2026. Ask the study team whether a trial fits you.

No phaseTaking people
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Where
Bethesda
Age
1 day to 100 years
People
200
Run by
National Human Genome Research Institute (NHGRI)
See details and how to joinNCT00029965
No phaseTaking people
Parkinson's Disease in Type 1 Gaucher Disease
Where
New York
Age
40 years and over
People
100
Run by
Icahn School of Medicine at Mount Sinai
See details and how to joinNCT07886879
No phaseTaking people
Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease
Where
Minneapolis
Age
18 years to 75 years
People
34
Run by
University of Minnesota
See details and how to joinNCT02437396
No phaseEnrolling by invitation
Genetic Studies of Lysosomal Storage Disorders
Where
Bethesda
Age
1 week to 110 years
People
1,000
Run by
National Human Genome Research Institute (NHGRI)
See details and how to joinNCT00001215
No phaseTaking people
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
Where
Ann Arbor, Aurora, Baltimore, Beverly Hills, Boston, Camden, Charlotte, Charlottesville, Chicago, Cincinnati, Cleveland, Columbia, Columbus, Coral Gables, Coral Springs, Dallas, Decatur, Detroit, Dublin, Durham, Fairfax, Fort Worth, Grand Rapids, Greenville, Hackensack, Hershey, Huntington, Indianapolis, Iowa City, Jacksonville, Kansas City, Las Vegas, Little Rock, Los Angeles, Louisville, Milwaukee, Minneapolis, Morristown, Nashville, New Brunswick, New Haven, New Hyde Park, New Orleans, New York, Norfolk, Omaha, Orange, Paterson, Philadelphia, Phoenix, Pittsburgh, Portland, Providence, Rochester, Sacramento, Salt Lake City, San Diego, San Francisco, Seattle, Stanford, St Louis, Syracuse, Tacoma, Tampa, Tucson, Valhalla, Washington D.C.
People
12,000 worldwide
Run by
Genzyme, a Sanofi Company
See details and how to joinNCT00358943
Phase 3Taking people
A Gaucher Disease Gene Therapy Trial With FLT201
Where
Atlanta, Aurora, Cincinnati, Dallas, Durham, Fairfax, Hackensack, Iowa City, Los Angeles, Minneapolis, New York, Philadelphia, Pittsburgh, San Francisco
Age
18 years and over
People
45 worldwide
Run by
Spur Therapeutics
See details and how to joinNCT07223944 · EU 2025-520765-50-00
No phaseTaking people
Gaucher Disease Outcome Survey (GOS)
Where
Lexington
People
1,257
Run by
Shire
See details and how to joinNCT03291223
No phaseTaking people
A Long-term Follow-up Study of Gaucher Disease
Where
Durham
People
200
Run by
Duke University
See details and how to joinNCT03190837
No phaseTaking people
Advancing Integrated Therapies for Gaucher Disease
Where
New Haven
Age
3 months to 75 years
People
30
Run by
Yale University
See details and how to joinNCT07758816
No phaseTaking people
MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)
Where
Minneapolis
Age
18 years to 80 years
People
5
Run by
University of Minnesota
See details and how to joinNCT05586243
No phaseEnrolling by invitation
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Where
Brooklyn, Manhasset, New York, Queens, The Bronx
Age
Up to 4 weeks
People
100,000
Run by
Albert Einstein College of Medicine
See details and how to joinNCT05368038
No phaseTaking people
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Where
San Francisco
Age
Up to 64 years
People
250
Run by
University of California, San Francisco
See details and how to joinNCT05619900
No phaseTaking people
Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease
Where
Minneapolis
Age
18 years to 80 years
People
30
Run by
University of Minnesota
See details and how to joinNCT05843552
Phase 1Taking people
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Where
San Francisco
Age
18 years to 50 years
People
10
Run by
University of California, San Francisco
See details and how to joinNCT04532047
No phaseTaking people
Longitudinal Study of Neurodegenerative Disorders
Where
Pittsburgh
People
1,500
Run by
University of Pittsburgh
See details and how to joinNCT03333200
No phaseTaking people
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Where
Pittsburgh
Age
2 months to 60 years
People
50
Run by
Paul Szabolcs
See details and how to joinNCT04528355
Phase 2Taking people
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Where
Pittsburgh
Age
2 months to 55 years
People
100
Run by
Paul Szabolcs
See details and how to joinNCT01962415