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ClinicalTrials.gov public records Last synced Nov 7, 2026, 10:08 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 1033–1056 of 1,513 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Inborn Errors of Metabolism Disorders Clear all

Conditions: Gout, Arthritis, Gouty, Hyperuricemia, Gout Chronic
Interventions: AR882 Dose 1, AR882 Dose 2, Placebo; Drug
Lead sponsor: Arthrosi Therapeutics; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 140 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 16
States / cities: Gilbert, Arizona • Sun City, Arizona • Tucson, Arizona + 12 more

Conditions: Hermansky-Pudlak Syndrome (HPS)
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Month to 115 Years
Enrollment: 600 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 1995
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Hypertension
Interventions: 6R-BH4 (sapropterin dihydrochloride); Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years and older
Enrollment: 84 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2008
U.S. locations: 24
States / cities: Hoover, Alabama • Huntsville, Alabama • Mobile, Alabama + 21 more

Conditions: Adrenal Hyperplasia, Congenital
Interventions: Hydrocortisone sodium acetate; Drug
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 3 Years to 18 Years
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2008
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Cardiac Amyloidosis
Interventions: AI ECG Amyloid algorithm; Other
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years and older
Enrollment: 20,143 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 4
States / cities: Scottsdale, Arizona • Jacksonville, Florida • Rochester, Minnesota + 1 more

Conditions: Parkinson Disease, Gaucher Disease
Interventions: 15-0 H20; Drug
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 18 Years to 110 Years
Enrollment: 64 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2006
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: 21-hydroxylase Deficiency
Interventions: Hydrocortisone withdrawal; Procedure
Lead sponsor: Maria I. New; Other
Eligibility: 18 Years to 50 Years
Enrollment: 99 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2009
U.S. locations: 2
States / cities: New York, New York • Dallas, Texas

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: 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

Conditions: Pseudohypoparathyroidism, Albright Hereditary Osteodystrophy, Pseudohypoparathyroidism Type 1a
Interventions: Theophylline, Placebo; Drug
Lead sponsor: Vanderbilt University Medical Center; Other
Eligibility: 2 Years to 12 Years
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Elevated LDL Cholesterol
Interventions: Cardiovascular fixed dose combination pill (acetylsalicylic acid, simvastatin and ramipril),, Simvastatin; Drug
Lead sponsor: Ferrer Internacional S.A.; Industry
Eligibility: 18 Years and older
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2011
U.S. locations: 1
States / cities: New York, New York

Conditions: Congenital Adrenal Hyperplasia
Interventions: Crinecerfont, Placebo; Drug
Lead sponsor: Neurocrine Biosciences; Industry
Eligibility: 2 Years to 17 Years
Enrollment: 103 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 22
States / cities: Birmingham, Alabama • Los Angeles, California • Orange, California + 19 more

Conditions: Kidney Calculi
Interventions: Potassium Citrate, Acetazolamide, Diet low in oxalate and purines; Drug · Behavioral
Lead sponsor: Children's Mercy Hospital Kansas City; Other
Eligibility: 5 Years to 17 Years
Healthy volunteers: Accepts healthy volunteers
Timeline: 2005 – 2007
U.S. locations: 1
States / cities: Kansas City, Missouri

Conditions: MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
Interventions: Not listed
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: Not listed
Enrollment: 353 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2014
U.S. locations: 2
States / cities: Oakland, California • Chicago, Illinois

Conditions: Darier Disease, Hailey-Hailey Disease, Hyperkeratosis, Epidermolytic, Ichthyosis, Ichthyosis, Lamellar, Ichthyosis, X-Linked, Keratoderma
Interventions: Not listed
Lead sponsor: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS); NIH
Eligibility: Not listed
Enrollment: 610 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1994 – 2004
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Phenylketonurias, PAH Deficiency, Phenylketonuria
Interventions: HMI-103; Drug
Lead sponsor: Homology Medicines, Inc; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 2
States / cities: Topeka, Indiana • Lancaster, Pennsylvania

Conditions: Lactose Intolerance
Interventions: Milk containing A1 and A2 beta-casein, Milk containing only A2 beta-casein; Other
Lead sponsor: Purdue University; Other
Eligibility: 18 Years to 65 Years
Enrollment: 24 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2025
U.S. locations: 1
States / cities: West Lafayette, Indiana

Conditions: Cystinosis
Interventions: HIIT Exercise; Other
Lead sponsor: Stanford University; Other
Eligibility: 18 Years to 60 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2024
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
Interventions: BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1), Intracerebroventricular access device; Biological · Device
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: Up to 17 Years
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2022
U.S. locations: 1
States / cities: Columbus, Ohio

Conditions: Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
Interventions: Idursulfase; Biological
Lead sponsor: Shire; Industry
Eligibility: 5 Years and older · Male only
Enrollment: 94 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2008
U.S. locations: 16
States / cities: Phoenix, Arizona • Oakland, California • Denver, Colorado + 13 more

Conditions: Gout, Moderate Renal Impairment
Interventions: Febuxostat IR, Febuxostat XR, Febuxostat placebo, Colchicine, Naproxen, Lansoprazole; Drug
Lead sponsor: Takeda; Industry
Eligibility: 18 Years and older
Enrollment: 189 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 89
States / cities: Birmingham, Alabama • Muscle Shoals, Alabama • Phoenix, Arizona + 75 more

Conditions: Hypophosphatasia
Interventions: ALXN1850, asfotase alfa; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 2 Years to 11 Years
Enrollment: 43 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2028
U.S. locations: 6
States / cities: Hartford, Connecticut • Baltimore, Maryland • Minneapolis, Minnesota + 3 more

Conditions: Cardiac Amyloidosis
Interventions: FNOS, Florbetaben; Drug
Lead sponsor: University of Pennsylvania; Other
Eligibility: 18 Years and older
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2024
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: MPS II, Hunter Syndrome (MPS II)
Interventions: RGX-121-3102; Genetic
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 4 Months to 5 Years · Male only
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2031
U.S. locations: 1
States / cities: New Brunswick, New Jersey

Search by objective public record fields.

Phase 2b Evaluation of Efficacy and Safety of AR882 in Gout Patients

Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome

A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension

Research Study for Children With Salt Wasting Congenital Adrenal Hyperplasia

Artificial Intelligence Enhanced ECG to Detect Cardiac Amyloidosis

Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations

Examining Genetic Differences Among People With 21-Hydroxylase Deficiency

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old

A Pharmacodynamic Study to Evaluate the Effect of a Fixed Dose Combination Pill on Low Density Lipoprotein (LDL) Cholesterol

Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

Effects of Potassium Citrate in Urine of Children With Elevated Calcium in Urine and Kidney Stones

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

National Registry for Ichthyosis and Related Disorders

Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

Comparing the Adaptation of Commercial Milk and A2 Milk in Lactose Maldigesters

The Effect of Exercise on Muscle Dysfunction in Cystinosis

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

Efficacy and Safety of Extended Release and Immediate Release Febuxostat in Participants With Gout and Moderate Renal Impairment

Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa

Molecular Imaging of the Underlying Mechanism of Cardiotoxicity in Patients With Light Chain Amyloidosis Using PET/CT

RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)