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 15, 2026, 3:57 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 1105–1128 of 1,513 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Inborn Errors of Metabolism Disorders Clear all

Conditions: Propionic Acidemia
Interventions: Not listed
Lead sponsor: University of Pittsburgh; Other
Eligibility: Not listed
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2022
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Gout, Arthritis, Gouty, Hyperuricemia, Gout Chronic
Interventions: AR882 50 mg, AR882 75 mg, Placebo; Drug
Lead sponsor: Arthrosi Therapeutics; Industry
Eligibility: 18 Years to 85 Years
Enrollment: 866 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2026
U.S. locations: 83
States / cities: Birmingham, Alabama • Huntsville, Alabama • Peoria, Arizona + 74 more

Conditions: Hunter Syndrome
Interventions: idursulfase-IT, No IT treatment; Biological · Other
Lead sponsor: Shire; Industry
Eligibility: Up to 18 Years · Male only
Enrollment: 58 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2017
U.S. locations: 3
States / cities: Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina

Conditions: Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)
Interventions: Stem Cell Transplant, Busulfan, Cyclophosphamide, ATG; Procedure · Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Not listed
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1999 – 2010
U.S. locations: 1
States / cities: Minneapolis, Minnesota

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: 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: Hunter Syndrome
Interventions: Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg), Idursulfase IT (30 mg); Other · Drug
Lead sponsor: Shire; Industry
Eligibility: 3 Years to 18 Years · Male only
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2012
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Malaria, Glucose 6 Phosphate Dehydrogenase Deficiency
Interventions: RPQ, SPQ, Primaquine Phosphate, Placebo; Drug
Lead sponsor: University of Mississippi, Oxford; Other
Eligibility: 18 Years to 65 Years
Enrollment: 36 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2019
U.S. locations: 1
States / cities: University, Mississippi

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: CER-001; Drug
Lead sponsor: Cerenis Therapeutics, SA; Industry
Eligibility: 12 Years and older
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2014
U.S. locations: 2
States / cities: Hartford, Connecticut • North Massapequa, New York

Conditions: COPD, Tetrahydrobiopterin Deficiency, Oxidative Stress
Interventions: Tetrahydrobiopterin, Placebo oral tablet; Drug
Lead sponsor: University of Massachusetts, Amherst; Other
Eligibility: 18 Years to 85 Years
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2022
U.S. locations: 1
States / cities: Amherst, Massachusetts

Conditions: Fructose Intolerance
Interventions: Placedo, Alanine; Dietary Supplement
Lead sponsor: Augusta University; Other
Eligibility: 18 Years and older
Enrollment: 65 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2020
U.S. locations: 1
States / cities: Augusta, Georgia

Conditions: Cytomegalovirus Retinitis, HIV Infections, Gastrointestinal Diseases
Interventions: Ganciclovir; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 13 Years and older
Enrollment: 850 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Ends 1995
U.S. locations: 17
States / cities: San Francisco, California • Denver, Colorado • Wilmington, Delaware + 12 more

Conditions: MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
Interventions: Glycerol Tributyrate; Drug
Lead sponsor: George Washington University; Other
Eligibility: 18 Years to 65 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2027 – 2028
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

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: Adrenal Hyperplasia, Congenital
Interventions: Not listed
Lead sponsor: Office of Rare Diseases (ORD); NIH
Eligibility: 20 Years to 35 Years · Female only
Enrollment: 40 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2008 – 2009
U.S. locations: 1
States / cities: New York, New York

Conditions: Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Interventions: AG10, Placebo; Drug
Lead sponsor: Eidos Therapeutics, a BridgeBio company; Industry
Eligibility: 18 Years to 90 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2024
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Gout
Interventions: RDEA3170 4.5 mg, RDEA3170 6 mg, RDEA3170 12 mg; Drug
Lead sponsor: Ardea Biosciences, Inc.; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 40 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2016
U.S. locations: 1
States / cities: Austin, Texas

Conditions: Pancreatic Cancer
Interventions: Gemcitabine, TT vaccine booster; Drug · Biological
Lead sponsor: Albert Einstein College of Medicine; Other
Eligibility: 18 Years to 100 Years
Enrollment: 10 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2017 – 2019
U.S. locations: 1
States / cities: The Bronx, New York

Conditions: Myalgia, Hypercholesterolemia, Hyperlipidemia
Interventions: PPD10558, Atorvastatin, Placebo; Drug
Lead sponsor: Furiex Pharmaceuticals, Inc; Industry
Eligibility: 40 Years and older
Enrollment: 282 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011
U.S. locations: 67
States / cities: Anniston, Alabama • Phoenix, Arizona • Huntington Park, California + 60 more

Conditions: Congenital Adrenal Hyperplasia, Healthy
Interventions: Not listed
Lead sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); NIH
Eligibility: 9 Years to 40 Years
Enrollment: 61 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2011
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Maple Syrup Urine Disease
Interventions: Phenylbutyrate, Placebo powder; Drug
Lead sponsor: Brendan Lee; Other
Eligibility: 3 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2017
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
Interventions: atumelnant (CRN04894); Drug
Lead sponsor: Crinetics Pharmaceuticals Inc.; Industry
Eligibility: 16 Years to 74 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2029
U.S. locations: 3
States / cities: Minneapolis, Minnesota • Morehead City, North Carolina • Philadelphia, Pennsylvania

Conditions: MPS IV A
Interventions: BMN 110 Weekly, Placebo, BMN 110 Every Other Week; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 5 Years and older
Enrollment: 177 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2012
U.S. locations: 6
States / cities: Oakland, California • Wilmington, Delaware • Washington D.C., District of Columbia + 3 more

Conditions: Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
Interventions: Aldurazyme, placebo; Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2005
U.S. locations: 8
States / cities: Mobile, Alabama • Atlanta, Georgia • Rochester, New York + 5 more

Search by objective public record fields.

Review of Charts From Amish/Mennonite Variant PA Patients

Phase 3 Evaluation of Efficacy and Safety of AR882 in Gout Patients (AR882-301)

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment

Stem Cell Transplantation for Hurler

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

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

A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®

Metabolism and Pharmacokinetics of Primaquine Enantiomers in Human Volunteers Receiving a Seven Day Dose Regimen

Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects

O2 Transport and Utilization in Health and Lung Disease

Investigation of Supplemental L-alanine in the Management of Dietary Fructose Intolerance

The Safety and Effectiveness of Ganciclovir in the Prevention of Cytomegalovirus (CMV) of the Eyes and Disease of the Stomach and Intestines in Patients With HIV

A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)

Diagnostic and Screening Study of Genetic Disorders

Role of the Protein Osteoprotegerin in the Bone Health of Women With Congenital Adrenal Hyperplasia

Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy

RDEA3170 PK/PD Study

Analyzing Childhood Recall Antigens in Patients With Pancreatic Cancer

Study of the Safety and Tolerability Associated With PPD10558 Versus Atorvastatin in Patients Previously Intolerant to Statins Due to Statin-associated Myalgia (SAM)

Catecholamine Reserve and Exercise Tolerance in Healthy Volunteers and Patients With Congenital Adrenal Hyperplasia

Phenylbutyrate Therapy for Maple Syrup Urine Disease

An Extension Study to Evaluate Safety and Efficacy of Atumelnant in Participants With Congenital Adrenal Hyperplasia

A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients