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ClinicalTrials.gov public records Last synced Oct 17, 2026, 9:32 PM EDT

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

Showing 985–1008 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Completed No phase listed Observational

Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease

NCT00244582
View directory record Official record
Conditions
Type 2 Gaucher Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Up to 5 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 17, 2026, 9:32 PM EDT
Recruiting Not applicable Interventional

Elevated Rate Pacing of Cardiac Amyloidosis

NCT07583823
View directory record Official record
Conditions
Cardiac Amyloidosis, Pacemaker
Interventions
Pacemaker Rate
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 17, 2026, 9:32 PM EDT
Terminated Phase 2 Interventional Results available

Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia

NCT01597440
View directory record Official record
Conditions
Propionic Acidemia, Methylmalonic Acidemia
Interventions
N-carbamylglutamate, Standard of Care
Drug · Other
Lead sponsor
Mendel Tuchman
Other
Eligibility
1 Hour to 4 Weeks
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
7
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 26, 2017 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Phase 3 Interventional

Study To Evaluate The Safety And Efficacy Of Torcetrapib/Atorvastatin In Subjects With Familial Hypercholerolemia

NCT00134485
View directory record Official record
Conditions
Hypercholesterolemia, Familial, Hyperlipidemia
Interventions
torcetrapib/atorvastatin, atorvastatin
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
14
States / cities
San Diego, California • Washington D.C., District of Columbia • Lutz, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2007 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.

NCT04526197
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, Celecoxib
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Aug 21, 2023 · Synced Oct 17, 2026, 9:32 PM EDT
Recruiting No phase listed Observational

Hepatic Histopathology in Urea Cycle Disorders

NCT04908319
View directory record Official record
Conditions
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia, Carbamyl Phosphate Synthetase Deficiency, NAGS Deficiency
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 20, 2025 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Not applicable Interventional

GoalKeeper: Intelligent Information Sharing for Children With Medical Complexity

NCT03620071
View directory record Official record
Conditions
Childhood Cancer, Cerebral Palsy, Chronic Lung Disease, Congenital Heart Disease, Congenital Metabolic Disorder, Gastrostomy
Interventions
GoalKeeper, Standard Care
Other
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Sep 26, 2023 · Synced Oct 17, 2026, 9:32 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy Study for Children With CLN5 Batten Disease

NCT05228145
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis CLN5
Interventions
NGN-101
Genetic
Lead sponsor
Neurogene Inc.
Industry
Eligibility
3 Years to 9 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Aug 12, 2024 · Synced Oct 17, 2026, 9:32 PM EDT
Recruiting No phase listed Observational

Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

NCT00029965
View directory record Official record
Conditions
Neurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Day to 100 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Phase 3 Interventional Results available

Effectiveness of Paricalcitol in Reducing Parathyroid Hormone (PTH) Levels in X-linked Hypophosphatemic Rickets

NCT00417612
View directory record Official record
Conditions
Hypophosphatemia, Familial, Hyperparathyroidism
Interventions
Paricalcitol, Placebo
Drug · Other
Lead sponsor
Yale University
Other
Eligibility
9 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 17, 2020 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Phase 2 Interventional Results available

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

NCT00813865
View directory record Official record
Conditions
Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1
Interventions
afegostat tartrate
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 15, 2018 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Phase 3 Interventional Results available

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

NCT01074944
View directory record Official record
Conditions
Gaucher Disease
Interventions
Eliglustat tartrate
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
170 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
10
States / cities
San Diego, California • New Haven, Connecticut • Decatur, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2017 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Phase 4 Interventional Results available

Late-Onset Treatment Study Extension Protocol

NCT00455195
View directory record Official record
Conditions
Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II, Acid Maltase Deficiency (AMD)
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
8 Years and older
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
26
States / cities
Anchorage, Alaska • Phoenix, Arizona • Beverly Hills, California + 23 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2014 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Phase 2 Interventional Results available

Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840

NCT04573309
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jun 24, 2024 · Synced Oct 17, 2026, 9:32 PM EDT
Suspended Phase 1Phase 2 Interventional

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

NCT04771416
View directory record Official record
Conditions
Leukodystrophy, Globoid Cell
Interventions
PBKR03
Biological
Lead sponsor
Gemma Biotherapeutics
Industry
Eligibility
1 Month to 9 Months
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
4
States / cities
Chicago, Illinois • New York, New York • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Phase 3 Interventional

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

NCT00134511
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 7, 2007 · Synced Oct 17, 2026, 9:32 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

NCT05845749
View directory record Official record
Conditions
MPS IVA, MPS VI
Interventions
Vosoritide Injection [Voxzogo]
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
5 Years to 10 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Phase 1 Interventional

Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)

NCT00337636
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis
Interventions
Surgery to implant human CNS stem cells (HuCNS-SC), Medication to suppress the immune system
Procedure · Drug
Lead sponsor
StemCells, Inc.
Industry
Eligibility
18 Months to 12 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jan 15, 2015 · Synced Oct 17, 2026, 9:32 PM EDT
Not listed No phase listed Observational

A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders

NCT00596999
View directory record Official record
Conditions
Hematologic Malignancies
Interventions
UCB and HPDSC
Procedure
Lead sponsor
Celgene Corporation
Industry
Eligibility
Up to 55 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated Apr 10, 2012 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Phase 2 Interventional Results available

Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)

NCT01205152
View directory record Official record
Conditions
Hypophosphatasia
Interventions
asfotase alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
24 Weeks to 42 Months
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
5
States / cities
Wilmington, Delaware • Springfield, Missouri • Omaha, Nebraska + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2019 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Phase 1 Interventional

Human Placental-Derived Stem Cell Transplantation

NCT01586455
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Interventions
Human Placental Derived Stem Cell
Drug
Lead sponsor
New York Medical College
Other
Eligibility
0 Years to 55 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
3
States / cities
Denver, Colorado • Valhalla, New York • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 25, 2022 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Phase 4 Interventional Results available

Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

NCT00364858
View directory record Official record
Conditions
Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
Interventions
Cerezyme
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2007
U.S. locations
18
States / cities
Washington D.C., District of Columbia • Coral Springs, Florida • Atlanta, Georgia + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2015 · Synced Oct 17, 2026, 9:32 PM EDT
Completed Phase 3 Interventional

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

NCT06846515
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 17, 2026, 9:32 PM EDT
Recruiting No phase listed Observational

Rare Kidney Stone Consortium Patient Registry

NCT00588562
View directory record Official record
Conditions
Primary Hyperoxaluria, Dent Disease, Cystinuria, APRT Deficiency
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
0 Years to 100 Years
Enrollment
730 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2028
U.S. locations
3
States / cities
Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 17, 2026, 9:32 PM EDT