- Conditions
- Gaucher Disease
- Interventions
- Enzyme analysis
- Other
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2012
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 289–312
of 2,421
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Gaucher Disease
- Interventions
- Standard of Care
- Other
- Lead sponsor
- Takeda
- Industry
- Eligibility
- Up to 5 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Osteogenesis Imperfecta
- Interventions
- Risedronate Oral Tablet
- Drug
- Lead sponsor
- Hospital for Special Surgery, New York
- Other
- Eligibility
- 6 Years to 100 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2027
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
- Interventions
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Drug
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 3 Months to 39 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2023
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Cardiovascular Diseases, Cardiovascular Abnormalities, Calcifications, Vascular, Endstage Renal Disease, ESRD, Coronary Artery Calcification
- Interventions
- SNF472, Placebo
- Drug
- Lead sponsor
- Sanifit Therapeutics S. A.
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 274 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 46
- States / cities
- Bakersfield, California • Chula Vista, California • Escondido, California + 42 more
- Conditions
- Gout
- Interventions
- DYV700, Placebo, Colchicine 0.6 mg
- Drug
- Lead sponsor
- Dyve Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 284 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2021
- U.S. locations
- 1
- States / cities
- Poway, California
- Conditions
- Wilson Disease
- Interventions
- Penicillamine (D1-W12), TETA 4HCL (W12-60), Penicillamine (W12-W60), TETA 4HCL (60-<W108)
- Drug
- Lead sponsor
- Orphalan
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 77 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 1
- States / cities
- New Haven, Connecticut
- Conditions
- Phenylketonuria, Maternal
- Interventions
- Pegvaliase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Female only
- Enrollment
- 50 participants
- Timeline
- 2022 – 2032
- U.S. locations
- 1
- States / cities
- Wilmington, North Carolina
- Conditions
- Hyperuricemia, Gout
- Interventions
- ALLN-346, Placebo
- Drug
- Lead sponsor
- Allena Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 18 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Fanconi Anemia
- Interventions
- Cyclophosphamide, Fludarabine, Total Body Irradiation, Bone Marrow Transplantation, Mycophenolate Mofetil, Sirolimus
- Drug · Procedure
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 83 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2020
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Rare Kidney Stone Diseases
- Interventions
- Not listed
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- Not listed
- Enrollment
- 6,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2028
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Menkes Disease
- Interventions
- Droxidopa Oral Product, Placebo Control
- Drug · Other
- Lead sponsor
- Stephen G. Kaler
- Other
- Eligibility
- 7 Years to 17 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2030
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Apparent Mineralocorticoid Excess (AME)
- Interventions
- Glycyrrhetic Acid, Placebo
- Drug
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2002 – 2009
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease, Wolman Disease, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2, Acid Lipase Deficiency, LIPA Deficiency, LAL-Deficiency
- Interventions
- Not listed
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2029
- U.S. locations
- 19
- States / cities
- Phoenix, Arizona • Stanford, California • Miramar, Florida + 14 more
- Conditions
- HIV Infections, Lipodystrophy, Insulin Resistance
- Interventions
- Avandia administration for 6-12 weeks
- Drug
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- NIH
- Eligibility
- 18 Years to 55 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1999 – 2003
- U.S. locations
- 1
- States / cities
- Stony Brook, New York
- Conditions
- MELAS
- Interventions
- IW-6463 Tablets
- Drug
- Lead sponsor
- Tisento Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2022
- U.S. locations
- 5
- States / cities
- Washington D.C., District of Columbia • Baltimore, Maryland • Boston, Massachusetts + 2 more
- Conditions
- Pseudohypoparathyroidism and Pseudopseudohypoparathyroidism, Pseudohypoparathyroidism Type Ia, Albright Hereditary Osteodystrophy
- Interventions
- Not listed
- Lead sponsor
- Vanderbilt University Medical Center
- Other
- Eligibility
- 6 Years to 50 Years
- Enrollment
- 14 participants
- Timeline
- 2019 – 2021
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- Conditions
- Nephropathic Cystinosis
- Interventions
- DFT383
- Genetic
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 2 Years to 5 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2044
- U.S. locations
- 6
- States / cities
- Phoenix, Arizona • San Diego, California • Stanford, California + 3 more
- Conditions
- Phenylketonurias, PKU
- Interventions
- Not listed
- Lead sponsor
- Homology Medicines, Inc
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Orange, California • Tampa, Florida + 5 more
- Conditions
- Pompe Disease
- Interventions
- S-606001, Placebo
- Drug
- Lead sponsor
- Shionogi
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 9
- States / cities
- Irvine, California • Gainesville, Florida • Atlanta, Georgia + 6 more
- Conditions
- Obesity Associated With Defects in Leptin-melanocortin Pathway
- Interventions
- Setmelanotide
- Drug
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 13
- States / cities
- Scottsdale, Arizona • San Diego, California • Gainesville, Florida + 10 more
- Conditions
- Fabry Disease
- Interventions
- Pegunigalsidase-alfa
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 6
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 3 more
- Conditions
- Adrenal Hyperplasia, Congenital
- Interventions
- Not listed
- Lead sponsor
- Office of Rare Diseases (ORD)
- NIH
- Eligibility
- 20 Years to 35 Years · Female only
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2009
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- RCDP - Rhizomelic Chondrodysplasia Punctata, RCDP1, RCDP2, RCDP3, RCDP4, RCDP5
- Interventions
- Not listed
- Lead sponsor
- Nemours Children's Clinic
- Other
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2030
- U.S. locations
- 1
- States / cities
- Wilmington, Delaware