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ClinicalTrials.gov public records Last synced Oct 22, 2026, 3:40 PM EDT

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Showing 337–360 of 1,510 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
Terminated Phase 4 Interventional Results available

Immune Tolerance Induction Study

NCT00701701
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
Myozyme® (alglucosidase alfa)
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
1 Month and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2020
U.S. locations
4
States / cities
Louisville, Kentucky • Durham, North Carolina • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2022 · Synced Oct 22, 2026, 3:40 PM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

CLinical Prediction Rule Score in Veterans

NCT01418781
View directory record Official record
Conditions
Gout
Interventions
Not listed
Lead sponsor
jasvinder singh
Federal
Eligibility
18 Years to 110 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 13, 2024 · Synced Oct 22, 2026, 3:40 PM EDT
Completed Phase 3 Interventional Results available

Lesinurad and Febuxostat Combination Extension Study in Gout

NCT01808144
View directory record Official record
Conditions
Gout
Interventions
lesinurad, febuxostat
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 85 Years
Enrollment
196 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
55
States / cities
Birmingham, Alabama • Tempe, Arizona • Glendale, California + 50 more
Source: ClinicalTrials.gov public record
Updated Jan 30, 2018 · Synced Oct 22, 2026, 3:40 PM EDT
Completed Phase 3 Interventional Results available

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

NCT00146770
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 3, 2015 · Synced Oct 22, 2026, 3:40 PM EDT
Completed Phase 2 Interventional Results available

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

NCT00925054
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG 0.001 mg/kg, rAvPAL-PEG 0.003 mg/kg, rAvPAL-PEG 0.01 mg/kg, rAvPAL-PEG 0.03 mg/kg, rAvPAL-PEG 0.1 mg/kg
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 55 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
11
States / cities
Aurora, Colorado • Decatur, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2019 · Synced Oct 22, 2026, 3:40 PM EDT
Recruiting Phase 1Phase 2 Interventional

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

NCT06839456
View directory record Official record
Conditions
Leukemia, High Risk Acute Lymphoblastic Leukemia, High Risk Acute Myeloid Leukemia, Relapse Leukemia, MDS (Myelodysplastic Syndrome), Relapsed Non-Hodgkin Lymphoma, Acquired Aplastic Anemia, Inherited BMF Syndrome, Immunodeficiency, Primary Immune Regulatory Disorder, Hemoglobinopathies, Bone Marrow Failure, Inborn Errors of Metabolism, HLH
Interventions
Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3, Phase 2 Maximum Tolerated Dose determined in Phase 1, Phase 2 Established Dose from prior study, NCT03810196
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
1 Month to 25 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Oct 22, 2026, 3:40 PM EDT
Recruiting Phase 4 Interventional Accepts healthy volunteers

Effects of Bisphosphonates on OI-Related Hearing Loss

NCT04152551
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Risedronate Oral Tablet
Drug
Lead sponsor
Hospital for Special Surgery, New York
Other
Eligibility
6 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 28, 2026 · Synced Oct 22, 2026, 3:40 PM EDT
Completed Phase 1 Interventional

Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease

NCT00004294
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase gene into autologous peripheral blood stem cells
Genetic
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 22, 2026, 3:40 PM EDT
Completed No phase listed Observational

Natural Evolution of AV Conduction Disorders in Patients Implanted With Cardiac Pacemakers

NCT00531037
View directory record Official record
Conditions
Patients Implanted With a Pacemaker
Interventions
Not listed
Lead sponsor
LivaNova
Industry
Eligibility
18 Years and older
Enrollment
1,440 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
7
States / cities
Birmingham, Alabama • Sheffield, Alabama • Mesa, Arizona + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 14, 2017 · Synced Oct 22, 2026, 3:40 PM EDT
Not listed Phase 2Phase 3 Interventional

An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III

NCT06614894
View directory record Official record
Conditions
Sanfilippo Syndrome, MPS3
Interventions
Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day
Drug
Lead sponsor
Ozlem Goker-Alpan
Other
Eligibility
18 Years to 99 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 20, 2025 · Synced Oct 22, 2026, 3:40 PM 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 22, 2026, 3:40 PM EDT
Completed Phase 2 Interventional Results available

Phase 2 Study of Obeticholic Acid for Lipodystrophy Patients

NCT02430077
View directory record Official record
Conditions
Familial Partial Lipodystrophy
Interventions
Obeticholic Acid, Placebo
Drug
Lead sponsor
Abhimanyu Garg
Other
Eligibility
18 Years to 70 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
2
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Aug 27, 2024 · Synced Oct 22, 2026, 3:40 PM EDT
Completed Phase 2 Interventional

SCH-58235 (Ezetimibe) to Treat Homozygous Sitosterolemia

NCT00045812
View directory record Official record
Conditions
Lipoidosis
Interventions
SCH-58235
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 22, 2026, 3:40 PM EDT
Completed Phase 2 Interventional Results available

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease

NCT00304512
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years · Female only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Oct 3, 2018 · Synced Oct 22, 2026, 3:40 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism

NCT01780168
View directory record Official record
Conditions
Oxidative Phosphorylation Deficiencies, Electron Transport Chain Disorders, Mitochondrial, Mitochondrial Disorders, Leigh Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
4 Weeks to 115 Years
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 22, 2026, 3:40 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy Volunteers

NCT03500627
View directory record Official record
Conditions
Healthy
Interventions
OP-101
Drug
Lead sponsor
Orpheris, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 17, 2018 · Synced Oct 22, 2026, 3:40 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Caregiving Networks Across Disease Context and the Life Course

NCT05007990
View directory record Official record
Conditions
Inherited Metabolic Disorders, Undiagnosed Diseases, Batten's Disease, Tay Sachs, Diabetes
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
2,800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 22, 2026, 3:40 PM EDT
Active, not recruiting Phase 3 Interventional

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

NCT06255782
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
Interventions
ECUR-506
Genetic
Lead sponsor
iECURE, Inc.
Industry
Eligibility
24 Hours to 7 Months · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
6
States / cities
Los Angeles, California • Aurora, Colorado • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 22, 2026, 3:40 PM EDT
Completed Phase 3 Interventional

Lesinurad and Allopurinol Combination Extension Study in Gout

NCT01808131
View directory record Official record
Conditions
Gout
Interventions
Lesinurad
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 85 Years
Enrollment
717 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
151
States / cities
Birmingham, Alabama • Gulf Shores, Alabama • Pinson, Alabama + 125 more
Source: ClinicalTrials.gov public record
Updated Jan 24, 2017 · Synced Oct 22, 2026, 3:40 PM EDT
Terminated Phase 1Phase 2 Interventional

Gene Therapy Clinical Study in Adult PKU

NCT03952156
View directory record Official record
Conditions
Phenylketonurias, PAH Deficiency
Interventions
HMI-102
Genetic
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
13
States / cities
Los Angeles, California • Orange, California • Tampa, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 29, 2023 · Synced Oct 22, 2026, 3:40 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Migalastat Food Effect Study

NCT01489995
View directory record Official record
Conditions
Fabry Disease
Interventions
A (migalastat), B (migalastat), C (migalastat), D (migalastat), E (migalastat)
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Dec 18, 2013 · Synced Oct 22, 2026, 3:40 PM EDT
Completed Phase 4 Interventional Results available

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

NCT03635957
View directory record Official record
Conditions
Gout
Interventions
Pegloticase, Methotrexate (MTX), Standard Gout Flare Prophylaxis, Infusion Reaction (IR) Prophylaxis, Folic Acid
Biological · Drug · Dietary Supplement
Lead sponsor
Amgen
Industry
Eligibility
18 Years to 65 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
6
States / cities
Anchorage, Alaska • Glendale, Arizona • Mesa, Arizona + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 26, 2024 · Synced Oct 22, 2026, 3:40 PM EDT
Completed Phase 2 Interventional Results available

Safety Study of Replagal® Therapy in Children With Fabry Disease

NCT01363492
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal (agalsidase alfa)
Biological
Lead sponsor
Shire
Industry
Eligibility
7 Years to 17 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
5
States / cities
Decatur, Georgia • Durham, North Carolina • Dallas, Texas + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Oct 22, 2026, 3:40 PM EDT
Completed No phase listed Observational

Data Collection in Women With Fabry Disease

NCT00030134
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Female only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 22, 2026, 3:40 PM EDT