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ClinicalTrials.gov public records Last synced Oct 15, 2026, 7:51 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 649–672 of 1,608 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolism, Inborn Errors Clear all
Completed Phase 2 Interventional Results available

Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)

NCT01163149
View directory record Official record
Conditions
Hypophosphatasia
Interventions
asfotase alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
13 Years to 65 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
2
States / cities
St Louis, Missouri • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 13, 2019 · Synced Oct 15, 2026, 7:51 AM EDT
Completed Phase 3 Interventional Results available

Hyper- and Hypokalemic Periodic Paralysis Study

NCT00494507
View directory record Official record
Conditions
Hyperkalemic Periodic Paralysis, Hypokalemic Periodic Paralysis
Interventions
Dichlorphenamide (double-blind), Placebo (double-blind), Dichlorphenamide (open-label)
Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years and older
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
10
States / cities
Los Angeles, California • San Francisco, California • Kansas City, Kansas + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2017 · Synced Oct 15, 2026, 7:51 AM EDT
Recruiting No phase listed Observational

Study of Alkaptonuria

NCT00005909
View directory record Official record
Conditions
Alkaptonuria
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 115 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 15, 2026, 7:51 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

NCT01907087
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 8, 2019 · Synced Oct 15, 2026, 7:51 AM EDT
Completed No phase listed Observational

"The MaP Study": Mapping the Patient Journey in MMA and PA

NCT03484767
View directory record Official record
Conditions
Methylmalonic Acidemia, Propionic Acidemia
Interventions
Not listed
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
Not listed
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
11
States / cities
Stanford, California • Atlanta, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 2, 2021 · Synced Oct 15, 2026, 7:51 AM EDT
Terminated No phase listed Observational

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

NCT00787995
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 8, 2014 · Synced Oct 15, 2026, 7:51 AM EDT
Completed Phase 2 Interventional

Phase II Randomized Study of Tin Mesoporphyrin for Neonatal Hyperbilirubinemia

NCT00004381
View directory record Official record
Conditions
Glucosephosphate Dehydrogenase Deficiency, Hyperbilirubinemia, Hemolytic Disease of Newborn
Interventions
tin mesoporphyrin
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
0 Years to 24 Hours
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 15, 2026, 7:51 AM EDT
Completed Phase 4 Interventional Results available

A Trial to Investigate the Non-inferiority of Pegloticase Administered Every 4 Weeks (Q4W) With MTX Compared With Every 2 Weeks (Q2W) With MTX in Participants With Uncontrolled Refractory Gout

NCT06229145
View directory record Official record
Conditions
Gout
Interventions
Pegloticase, Methotrexate
Biological · Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
306 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
74
States / cities
Birmingham, Alabama • Anchorage, Alaska • Chandler, Arizona + 63 more
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 15, 2026, 7:51 AM EDT
Recruiting Phase 1 Interventional

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

NCT07491172
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 15, 2026, 7:51 AM EDT
Completed No phase listed Observational

Bone Mineral Density in Adults With Hyperphenylalaninemia

NCT01209819
View directory record Official record
Conditions
Phenylketonurias
Interventions
Not listed
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 12, 2015 · Synced Oct 15, 2026, 7:51 AM EDT
Not listed Not applicable Interventional

Identification of Undiagnosed Lysosomal Acid Lipase Deficiency

NCT01716728
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease
Interventions
enzyme analysis
Other
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 13, 2013 · Synced Oct 15, 2026, 7:51 AM EDT
Recruiting Phase 3 Interventional

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

NCT06563895
View directory record Official record
Conditions
Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases, Polyneuropathies
Interventions
Acoramidis, Placebo oral tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 75 Years
Enrollment
587 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
40
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 31 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 15, 2026, 7:51 AM EDT
Completed Phase 2 Interventional Results available

Dexamethasone Treatment of Congenital Adrenal Hyperplasia

NCT00621985
View directory record Official record
Conditions
Adrenal Hyperplasia, Congenital
Interventions
dexamethasone, Hydrocortisone
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
2 Years to 9 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 23, 2011 · Synced Oct 15, 2026, 7:51 AM EDT
Completed No phase listed Observational

Impact of Unapproved Drug Initiative on Colchicine Use

NCT02000232
View directory record Official record
Conditions
Gout
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
185 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Aug 10, 2022 · Synced Oct 15, 2026, 7:51 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Single and Multiple Dose Study in Japanese Subjects

NCT01872832
View directory record Official record
Conditions
Gout
Interventions
RDEA3170 and Placebo
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
20 Years to 55 Years · Male only
Enrollment
46 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013
U.S. locations
1
States / cities
Glendale, California
Source: ClinicalTrials.gov public record
Updated Jan 9, 2014 · Synced Oct 15, 2026, 7:51 AM EDT
Completed Phase 1 Interventional Results available

Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease

NCT01065558
View directory record Official record
Conditions
Lesch-Nyhan Disease
Interventions
Ecopipam
Drug
Lead sponsor
Psyadon Pharma
Industry
Eligibility
6 Years to 65 Years · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
2
States / cities
San Diego, California • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 18, 2013 · Synced Oct 15, 2026, 7:51 AM EDT
Completed Phase 2 Interventional

Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias

NCT00061763
View directory record Official record
Conditions
Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
175 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
6
States / cities
Oakland, California • Stanford, California • Arlington Heights, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 22, 2017 · Synced Oct 15, 2026, 7:51 AM EDT
Withdrawn No phase listed Observational

A Study of the Natural Course of SURF1 Deficiency

NCT05277363
View directory record Official record
Conditions
Leigh Syndrome
Interventions
None (Observational study)
Other
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
Up to 18 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated May 20, 2022 · Synced Oct 15, 2026, 7:51 AM EDT
Completed Phase 2 Interventional

Open Label Safety/Efficacy Study of Arhalofenate in Combination With Febuxostat for Hyperuricemia in Gout Patients

NCT01416402
View directory record Official record
Conditions
Hyperuricemia, Gout
Interventions
Arhalofenate, Febuxostat, Colchicine
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
18 Years to 75 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Apr 3, 2015 · Synced Oct 15, 2026, 7:51 AM EDT
Approved For Marketing No phase listed Expanded access

Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

NCT06360237
View directory record Official record
Conditions
Familial Chylomicronemia Syndrome
Interventions
Olezarsen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
U.S. locations
1
States / cities
Carlsbad, California
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 15, 2026, 7:51 AM EDT
Completed Phase 2 Interventional Results available

PTG-300 in Subjects With Hereditary Hemochromatosis

NCT04202965
View directory record Official record
Conditions
Hereditary Hemochromatosis
Interventions
PTG-300
Drug
Lead sponsor
Protagonist Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
9
States / cities
Fayetteville, Georgia • Bethesda, Maryland • Charlotte, North Carolina + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2025 · Synced Oct 15, 2026, 7:51 AM EDT
Completed Phase 3 Interventional Results available

Combining Lesinurad With Allopurinol in Inadequate Responders

NCT01493531
View directory record Official record
Conditions
Gout
Interventions
Lesinurad, Placebo, Allopurinol
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 85 Years
Enrollment
610 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
88
States / cities
Mobile, Alabama • Chandler, Arizona • Mesa, Arizona + 76 more
Source: ClinicalTrials.gov public record
Updated May 26, 2016 · Synced Oct 15, 2026, 7:51 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Habitual Diet and Avocado Trial

NCT03528031
View directory record Official record
Conditions
Intra-abdominal Fat, Metabolic Syndrome, High Cholesterol, Triglycerides High, Diet Habit, High Blood Sugar, Liver Fat, Dietary Modification, HDL Cholesterol, Low Serum, Cardiovascular Diseases, High Density Lipoprotein Deficiency, Low-density-lipoprotein-type, Cardiovascular Risk Factor, Diabetes
Interventions
Intervention Daily Avocado
Other
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
25 Years and older
Enrollment
1,008 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
4
States / cities
Loma Linda, California • Los Angeles, California • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 22, 2023 · Synced Oct 15, 2026, 7:51 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Hemoglobinopathy

NCT00176852
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
Interventions
Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem cell infusion
Drug · Radiation · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 50 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 27, 2020 · Synced Oct 15, 2026, 7:51 AM EDT