Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Sep 26, 2026, 3:02 AM EDT

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Showing 1–24 of 500 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Healthy
Interventions
Palbociclib administered Fasted, Palbociclib administered Fed high calorie, Palbociclib administered Fed low calorie, Palbociclib administered Fed moderate calorie
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 55 Years
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013
U.S. locations
1
States / cities
New Haven, Connecticut
Active, not recruiting Phase 1 Interventional

Study for the Treatment for CLN7 Disease

NCT04737460
Conditions
CLN7
Interventions
AAV9/CLN7
Genetic
Lead sponsor
Benjamin Greenberg
Other
Eligibility
1 Year to 18 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
1
States / cities
Dallas, Texas
Conditions
Infantile Neronal Ceroid Lipofuscinosis
Interventions
Cystagon
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Months to 3 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
200 participants
Timeline
2017 – 2028
U.S. locations
1
States / cities
Durham, North Carolina
Completed No phase listed Observational Accepts healthy volunteers

PET Scans in Normal Volunteers and Patients With Fabry Disease

NCT00005111
Conditions
Fabry Disease, Healthy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Completed Early Phase 1 Interventional Accepts healthy volunteers

Apolipoprotein CIII Reduction Via Colchicine

NCT02083510
Conditions
Hypertriglyceridemia, Gout, Pericarditis
Interventions
Colchicine
Drug
Lead sponsor
Scripps Translational Science Institute
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014
U.S. locations
1
States / cities
La Jolla, California
Conditions
Severe Familial Hypercholesterolemia
Interventions
Evolocumab
Biological
Lead sponsor
Amgen
Industry
Eligibility
12 Years to 80 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
6
States / cities
Los Angeles, California • New York, New York • Cincinnati, Ohio + 1 more
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Hemoglobinopathy

NCT00176852
Conditions
Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
Interventions
Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem cell infusion
Drug · Radiation · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 50 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 8 Months
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Phoenix, Arizona
Conditions
GM1 Gangliosidosis
Interventions
LYS-GM101
Genetic
Lead sponsor
LYSOGENE
Industry
Eligibility
Up to 3 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Orange, California
Conditions
Gaucher's Disease, Osteopenia
Interventions
alendronate sodium, calcium carbonate, cholecalciferol
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 50 Years
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2002
U.S. locations
2
States / cities
Cincinnati, Ohio • Dayton, Ohio
Conditions
Niemann-Pick Disease, Type C1
Interventions
Hydroxypropyl-beta-cyclodextrin
Drug
Lead sponsor
Cyclo Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
2
States / cities
Oakland, California • Morristown, New Jersey
Conditions
Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions
Abatacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Atlanta, Georgia
Conditions
Familial Hypoalphalipoproteinemia
Interventions
CER-001, Placebo
Drug
Lead sponsor
Cerenis Therapeutics, SA
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Salt Lake City, Utah
Conditions
Familial Hypercholesterolemia
Interventions
Implitapide
Drug
Lead sponsor
Medical Research Laboratories International
Industry
Eligibility
18 Years to 70 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
Cincinnati, Ohio
Conditions
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions
CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone
Device · Drug
Lead sponsor
Diane George
Other
Eligibility
Up to 22 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Conditions
Familial Hypercholesterolemia, Adherence, Medication
Interventions
Monetary incentives
Behavioral
Lead sponsor
Boston Children's Hospital
Other
Eligibility
12 Years to 21 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Gaucher Disease Type 1
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
16 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Conditions
GM1 Gangliosidosis
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
2
States / cities
Oakland, California • Philadelphia, Pennsylvania
Conditions
Fabry Disease, Proteinuria
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
14 Years to 95 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Birmingham, Alabama
Conditions
Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy
Interventions
Not listed
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
1
States / cities
Iowa City, Iowa
Conditions
Gaucher Disease Type 1
Interventions
FLT201
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
16
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 11 more
Conditions
Non-ischemic Cardiomyopathy, Cardiomyopathies, Hypertrophic Cardiomyopathy, Right Ventricular Arrhythmogenic Cardiomyopathy, Cardiac Amyloidosis, Anderson Fabry Disease, Sarcoidosis, Cancer Therapy-related Cardiac Dysfunction, Ventricular Arrythmia, Heart Failure
Interventions
Not listed
Lead sponsor
Montefiore Medical Center
Other
Eligibility
18 Years and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
1
States / cities
The Bronx, New York
Conditions
Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
Interventions
Sebelipase alfa 0.35 mg/kg, Sebelipase alfa 1 mg/kg, Sebelipase alfa 3 mg/kg
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
3
States / cities
Stanford, California • New York, New York • Pittsburgh, Pennsylvania