- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- Zodasiran
- Drug
- Lead sponsor
- Arrowhead Pharmaceuticals
- Industry
- Eligibility
- 12 Years to 17 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
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 1,608
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
- Interventions
- Not listed
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- Not listed
- Enrollment
- 1,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2033
- U.S. locations
- 13
- States / cities
- La Jolla, California • Los Angeles, California • Jacksonville, Florida + 10 more
- Conditions
- Iron Overload
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2010
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Familial Hypercholesterolemia - Heterozygous
- Interventions
- Inclisiran, Placebo
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 6 Years to 11 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 9
- States / cities
- San Francisco, California • Washington D.C., District of Columbia • Boca Raton, Florida + 4 more
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- Lomitapide
- Drug
- Lead sponsor
- Aegerion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 13 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2004
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Phenylketonuria, Hyperphenylalaninaemia
- Interventions
- Kuvan
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 1,887 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2021
- U.S. locations
- 45
- States / cities
- Los Angeles, California • Orange, California • San Diego, California + 38 more
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- Demographics, diagnosis type, genotype, lipid profile, treatment allocation, country of residence.
- Other
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2026
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
- Interventions
- Sonlicromanol, Placebo
- Drug
- Lead sponsor
- Khondrion BV
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 220 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Cleveland, Ohio • Houston, Texas
- Conditions
- Gaucher Disease, Bone Diseases
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- 5 Years to 21 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- 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
- Fabry Disease
- Interventions
- Venglustat (GZ402671), Placebo
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 122 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 11
- States / cities
- Birmingham, Alabama • Los Angeles, California • Orange, California + 8 more
- Conditions
- Hyper IgD Syndrome, Mevalonate Kinase Deficiency
- Interventions
- Not listed
- Lead sponsor
- Michigan Technological University
- Other
- Eligibility
- 18 Years to 89 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 3
- States / cities
- Houghton, Michigan • Omaha, Nebraska • Portland, Oregon
- Conditions
- Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis
- Interventions
- Acoramidis (AG10) oral tablet
- Drug
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2030
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Morquio A Syndrome, Mucopolysaccharidosis IV A
- Interventions
- Not listed
- Lead sponsor
- Nadia Ali, PhD
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Gaucher Disease
- Interventions
- AVR-RD-02
- Biological
- Lead sponsor
- John Bernat
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2037
- U.S. locations
- 1
- States / cities
- Iowa City, Iowa
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie's Syndrome
- Interventions
- laronidase
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2007
- U.S. locations
- 3
- States / cities
- Los Angles, California • Hartford, Connecticut • Milwaukee, Wisconsin
- Conditions
- Hereditary Amyloidosis, Transthyretin-Related
- Interventions
- neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
- Diagnostic Test
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 30 Years to 90 Years
- Enrollment
- 47 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- Evinacumab
- Drug
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 5 Years to 11 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 6
- States / cities
- Wilmington, Delaware • Boca Raton, Florida • Kansas City, Kansas + 3 more
- Conditions
- Mucopolysaccharidosis Type IIIA
- Interventions
- LYS-SAF302
- Drug
- Lead sponsor
- LYSOGENE
- Industry
- Eligibility
- 6 Months and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 4
- States / cities
- Orange, California • Minneapolis, Minnesota • New York, New York + 1 more
- Conditions
- Refractive Errors
- Interventions
- Acuity 200 (Fluoroxyfocon A) contact lens, Acuity 100 (Hexafocon A) contact lens
- Device
- Lead sponsor
- Acuity Polymers, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 74 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2020
- U.S. locations
- 3
- States / cities
- Honolulu, Hawaii • Edina, Minnesota • Pittsford, New York
- Conditions
- GSD1
- Interventions
- DTX401, steroid regimen
- Genetic · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 3
- States / cities
- Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
- Conditions
- Gaucher Disease, Type 1
- Interventions
- FLT201
- Genetic
- Lead sponsor
- Spur Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Fairfax, Virginia
- Conditions
- MDS, Anemia, Aplastic, Inborn Errors of Metabolism, Congenital Marrow Failure, Congenital Immunodeficiency Syndrome
- Interventions
- ALDHbr Umbilical Cord Blood Cells
- Biological
- Lead sponsor
- Joanne Kurtzberg, MD
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2012
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Gaucher Disease, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Interventions
- Not listed
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 12,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1991 – 2034
- U.S. locations
- 86
- States / cities
- Phoenix, Arizona • Tucson, Arizona • Little Rock, Arkansas + 65 more