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ClinicalTrials.gov public records Last synced Oct 15, 2026, 7:48 PM EDT

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

Showing 553–576 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Recruiting No phase listed Observational

Clinical and Basic Investigations Into Congenital Disorders of Glycosylation

NCT04199000
View directory record Official record
Conditions
Congenital Disorders of Glycosylation
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2030
U.S. locations
12
States / cities
San Diego, California • Aurora, Colorado • Jacksonville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 3 Interventional Results available

Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis

NCT01438411
View directory record Official record
Conditions
Bile Acid Synthesis Defect
Interventions
Cholic Acid
Drug
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 3, 2023 · Synced Oct 15, 2026, 7:48 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

NCT03887533
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
VTS-270
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
3 Years to 60 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 30, 2022 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 3 Interventional Results available

Evolocumab Compared to LDL-C Apheresis in Patients Receiving LDL-C Apheresis Prior to Study Enrollment

NCT02585895
View directory record Official record
Conditions
Hypercholesterolemia
Interventions
Evolocumab, Low-density Lipoprotein Cholesterol (LDL-C) Apheresis
Biological · Procedure
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
4
States / cities
Boca Raton, Florida • Kansas City, Kansas • Grandville, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 30, 2022 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 4 Interventional Results available

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

NCT06019728
View directory record Official record
Conditions
Fabry's Disease
Interventions
AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine, Dexamethasone, Montelukast, Loratadine, Cetirizine, Fexofenadine
Drug
Lead sponsor
Sanofi
Industry
Eligibility
2 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
5
States / cities
Santa Monica, California • Atlanta, Georgia • Grand Rapids, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Bioavailability Study of Ciprofloxacin 1000 mg ER Tablets of Dr. Reddy's Under Non-Fasting Conditions

NCT01372358
View directory record Official record
Conditions
Fed
Interventions
CIPRO®XR
Drug
Lead sponsor
Dr. Reddy's Laboratories Limited
Industry
Eligibility
18 Years to 45 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 13, 2011 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 3 Interventional Results available

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

NCT02194985
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
7
States / cities
Atlanta, Georgia • Kansas City, Kansas • Grand Rapids, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2020 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells

NCT00001234
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase cDNA
Genetic
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1988 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 1 Interventional

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

NCT00151216
View directory record Official record
Conditions
Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
Interventions
AAV2CUhCLN2 (3x10^12 particle units)
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
3 Years to 18 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 24, 2020 · Synced Oct 15, 2026, 7:48 PM EDT
Completed No phase listed Observational

Estimation of the Carrier Frequency and Incidence of Smith-Lemli-Opitz Syndrome in African Americans

NCT00017732
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 15, 2026, 7:48 PM EDT
Active, not recruiting No phase listed Observational

Long Term Follow-up on Menkes Disease Patients

NCT04337684
View directory record Official record
Conditions
Menkes Disease
Interventions
Long Term Follow-Up
Drug
Lead sponsor
Sentynl Therapeutics, Inc.
Industry
Eligibility
Not listed
Enrollment
50 participants
Timeline
2019 – 2026
U.S. locations
1
States / cities
Solana Beach, California
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 15, 2026, 7:48 PM EDT
Completed No phase listed Observational

Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI

NCT01521429
View directory record Official record
Conditions
Mucopolysaccharidoses
Interventions
Not listed
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Other
Eligibility
5 Years to 35 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2019
U.S. locations
3
States / cities
Oakland, California • Torrance, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 22, 2024 · Synced Oct 15, 2026, 7:48 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

NCT04713475
View directory record Official record
Conditions
GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
Interventions
PBGM01
Biological
Lead sponsor
Gemma Biotherapeutics
Industry
Eligibility
1 Month to 24 Months
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
4
States / cities
Oakland, California • Minneapolis, Minnesota • New Brunswick, New Jersey + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 15, 2026, 7:48 PM EDT
Withdrawn No phase listed Observational

B7 Coreceptor Molecules in Hyper IgD Syndrome Form of Mevalonate Kinase Deficiency

NCT01568736
View directory record Official record
Conditions
Hyper IgD Syndrome, Mevalonate Kinase Deficiency
Interventions
Not listed
Lead sponsor
Michigan Technological University
Other
Eligibility
18 Years to 89 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
3
States / cities
Houghton, Michigan • Omaha, Nebraska • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jun 18, 2018 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

NCT01678898
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102
Drug
Lead sponsor
Protalix
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
9
States / cities
Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study of JR-171 in Patients With Mucopolysaccharidosis I

NCT04227600
View directory record Official record
Conditions
Mucopolysaccharidosis I
Interventions
JR-171 (lepunafusp alfa)
Drug
Lead sponsor
JCR Pharmaceuticals Co., Ltd.
Industry
Eligibility
0 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Mar 19, 2025 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth

NCT06193304
View directory record Official record
Conditions
Gaucher's Disease
Interventions
Eliglustat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 55 Years
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014
U.S. locations
1
States / cities
Evansville, Indiana
Source: ClinicalTrials.gov public record
Updated Jan 5, 2024 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 2 Interventional Results available

N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

NCT03759665
View directory record Official record
Conditions
GM2 Gangliosidosis, Tay-Sachs Disease, Sandhoff Disease
Interventions
IB1001
Drug
Lead sponsor
IntraBio Inc
Industry
Eligibility
6 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
3
States / cities
Los Angeles, California • Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 12, 2024 · Synced Oct 15, 2026, 7:48 PM EDT
Completed No phase listed Observational Results available

A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT01047306
View directory record Official record
Conditions
Sanfilippo Syndrome Type A
Interventions
assessment
Other
Lead sponsor
Shire
Industry
Eligibility
1 Year and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 11, 2021 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 3 Interventional Results available

Celebrex In Acute Gouty Arthritis Study

NCT00549549
View directory record Official record
Conditions
Arthritis, Gouty
Interventions
Indomethacin, Celecoxib
Drug
Lead sponsor
Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Industry
Eligibility
18 Years and older
Enrollment
402 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
43
States / cities
Glendale, Arizona • Mesa, Arizona • Paradise Valley, Arizona + 37 more
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Oct 15, 2026, 7:48 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Descriptive Analysis of Gut Microbiome Alterations in Hyperoxaluric Patients

NCT02794649
View directory record Official record
Conditions
Oxalate, Primary Hyperoxaluria, Microbiome
Interventions
Not listed
Lead sponsor
VA New York Harbor Healthcare System
Federal
Eligibility
18 Years to 80 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2018
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 26, 2017 · Synced Oct 15, 2026, 7:48 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 15, 2026, 7:48 PM EDT
Completed Phase 3 Interventional Results available

Long-Term Safety and Tolerability of Canakinumab Prefilled Syringes in Frequently Flaring Acute Gouty Arthritis Patients

NCT01431638
View directory record Official record
Conditions
Acute Gouty Arthritis
Interventions
Canakinumab 150mg in prefilled syringe
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
397 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
54
States / cities
Anniston, Alabama • Gulf Shores, Alabama • Mobile, Alabama + 50 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2021 · Synced Oct 15, 2026, 7:48 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 15, 2026, 7:48 PM EDT