Search | Clinical Trials Finder

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Nov 6, 2026, 6:59 AM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 961–984 of 1,513 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Inborn Errors of Metabolism Disorders Clear all

Conditions: Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Familial Hypercholesterolemia
Interventions: Screening for FH; Diagnostic Test
Lead sponsor: University of Wisconsin, Madison; Other
Eligibility: 18 Years and older
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2029
U.S. locations: 1
States / cities: Madison, Wisconsin

Conditions: Transthyretin Amyloidosis, ATTR-CM, ATTRv-PN, ATTR, ATTR-Mixed, hATTR
Interventions: Treatment of transthyretin (ATTR) amyloidosis in observational study setting; Drug
Lead sponsor: AstraZeneca; Industry
Eligibility: 18 Years to 130 Years
Enrollment: 1,850 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2031
U.S. locations: 33
States / cities: La Jolla, California • Los Angeles, California • San Francisco, California + 24 more

Conditions: Phenylketonurias
Interventions: PreKUnil® LNAA Medical Food for PKU; Other
Lead sponsor: University of Southern California; Other
Eligibility: 18 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2024
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Gaucher Disease Type 1, Gaucher Disease Type 3
Interventions: Velaglucerase alfa; Drug
Lead sponsor: Baylor Research Institute; Other
Eligibility: 4 Years to 14 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder, Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (sHTG), Mixed Hyperlipemia, Hypercholesterolaemia
Interventions: CTX310; Drug
Lead sponsor: CRISPR Therapeutics AG; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2028
U.S. locations: 8
States / cities: Hialeah, Florida • Jacksonville, Florida • Orlando, Florida + 5 more

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Foscarnet sodium; Drug
Lead sponsor: Astra USA; Industry
Eligibility: 18 Years to 65 Years
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Houston, Texas

Conditions: G(M2) Ganglioside, Tay-Sachs Disease Ganglioside, Sandhoff Disease Ganglioside
Interventions: Pyrimethamine; Drug
Lead sponsor: Exsar Corporation; Industry
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2008
U.S. locations: 2
States / cities: New York, New York • Cleveland, Ohio

Conditions: Hypercholesterolemia, Familial
Interventions: Torcetrapib/atorvastatin; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Lysosomal Acid Lipase Deficiency
Interventions: Not listed
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 2 Years and older
Enrollment: 640 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 15
States / cities: Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 12 more

Conditions: Hyperoxaluria
Interventions: Betaine, Placebo; Drug
Lead sponsor: Mayo Clinic; Other
Eligibility: 6 Years to 70 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2011
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Acute Myocardial Infarction, Restenoses, Coronary, Coronary Artery Lesions, Venous Embolism, Arterial Embolism
Interventions: Coronary and peripheral stents, Pacing catheters, Vascular plugs, Measurement and imaging (FFR and OCT), Peripheral dilatation catheters, Coronary dilatation catheters, Coronary and peripheral guidewires, Vessel closure/compression devices, Vascular access introducers; Device
Lead sponsor: Abbott Medical Devices; Industry
Eligibility: 18 Years and older
Enrollment: 3,784 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2031
U.S. locations: 7
States / cities: Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 4 more

Conditions: Mucopolysaccharidosis II
Interventions: Observational; Other
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 1 Month to 8 Years · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 2
States / cities: Oakland, California • Philadelphia, Pennsylvania

Conditions: Sexual Assault, Substance Use, Risky Health Behavior, Ema
Interventions: Ecological Momentary Intervention; Behavioral
Lead sponsor: University of New Mexico; Other
Eligibility: 18 Years to 24 Years · Female only
Enrollment: 183 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2023
U.S. locations: 1
States / cities: Albuquerque, New Mexico

Conditions: Mucopolysaccharidosis Type IIIA
Interventions: DNL126, Prophylactic immunomodulatory therapy; Drug
Lead sponsor: Denali Therapeutics Inc.; Industry
Eligibility: 0 Years to 18 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2032
U.S. locations: 4
States / cities: Oakland, California • Iowa City, Iowa • Chapel Hill, North Carolina + 1 more

Conditions: Danon Disease
Interventions: RP-A501; Genetic
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 8 Years and older · Male only
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2032
U.S. locations: 3
States / cities: La Jolla, California • Boston, Massachusetts • Philadelphia, Pennsylvania

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: zodasiran Injection, Placebo; Drug
Lead sponsor: Arrowhead Pharmaceuticals; Industry
Eligibility: 12 Years and older
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 4
States / cities: Park Ridge, Illinois • New York, New York • Cincinnati, Ohio + 1 more

Conditions: Fabry Disease
Interventions: migalastat hydrochloride; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 16 Years and older
Enrollment: 85 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2016
U.S. locations: 11
States / cities: Atlanta, Georgia • Chicago, Illinois • Kansas City, Kansas + 8 more

Conditions: Gaucher's Disease
Interventions: Eliglustat; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 6 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014
U.S. locations: 1
States / cities: Evansville, Indiana

Conditions: Gout
Interventions: Pegloticase, methotrexate, placebo, folic acid, gout flare prophylaxis regimen, fexofenadine, acetaminophen, methylprednisolone; Biological · Drug · Dietary Supplement
Lead sponsor: Amgen; Industry
Eligibility: 18 Years and older
Enrollment: 152 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 60
States / cities: Birmingham, Alabama • Anchorage, Alaska • Flagstaff, Arizona + 54 more

Conditions: Type 1 Gaucher Disease
Interventions: Safety and efficacy assessments; Other
Lead sponsor: AVROBIO; Industry
Eligibility: 18 Years to 99 Years
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 2
States / cities: Iowa City, Iowa • Hackensack, New Jersey

Conditions: Sphingolipidoses, Enzyme Deficiency
Interventions: no intervention; Other
Lead sponsor: University of California, San Francisco; Other
Eligibility: Not listed
Enrollment: 120 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2028
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Albinism, Inborn Errors of Metabolism, Oculocutaneous Albinism, Platelet Storage Pool Deficiency, Pulmonary Fibrosis
Interventions: Pirfenidone, Placebo; Drug
Lead sponsor: William Gahl, M.D.; NIH
Eligibility: 18 Years and older
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2016
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 13 Years and older
Enrollment: 225 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 19
States / cities: Berkeley, California • Los Angeles, California • San Diego, California + 12 more

Conditions: Lysosomal Acid Lipase Deficiency
Interventions: Sebelipase Alfa, Placebo; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 4 Years and older
Enrollment: 66 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2018
U.S. locations: 11
States / cities: Tucson, Arizona • Palo Alto, California • San Francisco, California + 8 more

Search by objective public record fields.

The ORIGIN-FH Study

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis

Additional Dietary Large Neutral Amino Acids (LNAA) for Improved Symptoms in Adult Classical Phenylktonuria (PKU)

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias

A Study of Foscarnet in the Treatment of Cytomegalovirus (CMV) of the Eyes in Patients With AIDS Who Have Not Had Success With Ganciclovir

A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis

Study To Evaluate The Effect Of Torcetrapib/Atorvastatin In Patients With Genetic High Cholesterol Disorder

A Study to Identify and Characterize LAL-D Patients in High-risk Populations

Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria

Abbott Vascular Medical Device Registry

Mucopolysaccharidosis Type II Observational

Nexus of Risk: Sexual Assault, Alcohol Use, and Risky Sex Among College Women

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)

Open-Label Phase 3 Long-Term Safety Study of Migalastat

A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth

Study of KRYSTEXXA® (Pegloticase) Plus Methotrexate in Participants With Uncontrolled Gout

A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02

SPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International)

Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

A Randomized Study Comparing the Safety and Efficacy of Two Regimens of Oral Ganciclovir to Intravenous Ganciclovir Maintenance Therapy for Cytomegalovirus Retinitis in People With AIDS Who Have Received Prior Ganciclovir Therapy

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency