- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- evinacumab, Placebo
- Drug
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 65 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 6
- States / cities
- Boca Raton, Florida • Boston, Massachusetts • New York, New York + 3 more
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 501
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Familial Partial Lipodystrophy
- Interventions
- Metreleptin
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- 13 Years and older
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 7
- States / cities
- Birmingham, Alabama • Boca Raton, Florida • Boston, Massachusetts + 4 more
- Conditions
- Dyslipidemias, High Cholesterol, Hypercholesterolemia, Familial Hypercholesterolemia, Lipid Metabolism Disorder, Metabolic Disease, Lipid Metabolism, Inborn Errors, Genetic Disease, Inborn, Hyperlipoproteinemias
- Interventions
- Obicetrapib, Placebo
- Drug
- Lead sponsor
- NewAmsterdam Pharma
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 354 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 20
- States / cities
- Jonesboro, Arkansas • Toluca Lake, California • Sarasota, Florida + 17 more
- Conditions
- Fabry Disease
- Interventions
- Replagal
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 2
- States / cities
- Iowa City, Iowa • Fairfax, Virginia
- Conditions
- Juvenile Neuronal Ceroid Lipofuscinosis
- Interventions
- Mycophenolate mofetil, Liquid Placebo
- Drug
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 6 Years to 25 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Fabry Disease
- Interventions
- migalastat HCl
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 55 Years · Male only
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Decatur, Georgia • Bethesda, Maryland + 2 more
- Conditions
- Gaucher Disease, Type 1
- Interventions
- FLT201
- Genetic
- Lead sponsor
- Spur Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Healthy Volunteers, Fasting, Fed
- Interventions
- Apixaban 5 mg Oral Dissolving Film (TAH3311) and Tablet (Eliquis®)
- Drug
- Lead sponsor
- TAHO Pharmaceuticals Ltd.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- Las Vegas, Nevada
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- VTS-270
- Drug
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- Up to 6 Months
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2024
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- 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
- Fabry Disease, Pregnancy, Pregnancy Complications
- Interventions
- Not applicable- observational study
- Other
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- Female only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2034
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Niemann-Pick Disease, Type C1
- Interventions
- VTS-270
- Drug
- Lead sponsor
- Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
- Industry
- Eligibility
- 2 Years to 25 Years
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
- Interventions
- bempedoic acid, placebo
- Drug
- Lead sponsor
- Esperion Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 779 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 2
- States / cities
- Clearwater, Florida • Georgetown, Texas
- Conditions
- Neutropenia
- Interventions
- Mavorixafor, Placebo
- Drug
- Lead sponsor
- X4 Pharmaceuticals
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 176 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 22
- States / cities
- Phoenix, Arizona • Irvine, California • La Jolla, California + 19 more
- Conditions
- Barth Syndrome
- Interventions
- Not listed
- Lead sponsor
- Duke University
- Other
- Eligibility
- 8 Years to 35 Years · Male only
- Enrollment
- 64 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2020
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Krabbe Disease
- Interventions
- FBX-101
- Biological
- Lead sponsor
- Forge Biologics, Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- Conditions
- Hypercholesterolemia, Familial
- Interventions
- Not listed
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Sjogren-Larsson Syndrome (SLS)
- Interventions
- Not listed
- Lead sponsor
- University of Nebraska
- Other
- Eligibility
- Not listed
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2023
- U.S. locations
- 2
- States / cities
- Omaha, Nebraska • Pittsburgh, Pennsylvania
- Conditions
- Homozygous Familial Hypercholesterolemia (HoFH)
- Interventions
- AAV directed hLDLR gene therapy
- Genetic
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2020
- U.S. locations
- 5
- States / cities
- Boca Raton, Florida • Kansas City, Kansas • Portland, Oregon + 2 more
- Conditions
- Tay-Sachs Disease, Porphyria, Erythropoietic, Leukodystrophy, Globoid Cell, Metabolism, Inborn Errors
- Interventions
- Not listed
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 0 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1999
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia, Heme Malignancy, Shwachman Syndrome, Inherited Bone Marrow Failure, Exocrine Pancreatic Insufficiency, WHIM, ELANE, SBDS Gene Mutation, EFL1 Gene Mutation, DNAJC21 Gene Mutation, SRP54 Gene Mutation, Inherited Cancer Syndrome, Inherited Cancer-Predisposing Syndrome, Neutropenia, Severe Chronic, Neutropenia Other, Neutropenia Chronic Benign, Ribosome Alteration, Ribosomopathy, Immune Deficiency, Inherited BMF Syndrome, Inherited Immunodeficiency Diseases, Cognitive Delay, Mild, Myelodysplastic Syndromes, Pancytopenia
- Interventions
- Not listed
- Lead sponsor
- Shwachman-Diamond Syndrome Alliance Inc
- Other
- Eligibility
- Not listed
- Enrollment
- 8,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2088
- U.S. locations
- 1
- States / cities
- Woburn, Massachusetts
- Conditions
- Parkinson Disease
- Interventions
- LY3884961, Methylprednisolone, Sirolimus
- Biological · Drug
- Lead sponsor
- Prevail Therapeutics
- Industry
- Eligibility
- 35 Years to 80 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2031
- U.S. locations
- 11
- States / cities
- Scottsdale, Arizona • Murrieta, California • Englewood, Colorado + 7 more
- Conditions
- Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease
- Interventions
- BMN 190
- Biological
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 3 Years to 15 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 1
- States / cities
- Columbus, 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