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ClinicalTrials.gov public records Last synced Oct 21, 2026, 10:58 AM EDT

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Showing 217–240 of 1,510 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
Completed No phase listed Observational

Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

NCT04041102
View directory record Official record
Conditions
GM1 Gangliosidosis
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
2
States / cities
Oakland, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 24, 2025 · Synced Oct 21, 2026, 10:58 AM EDT
Terminated Phase 2 Interventional Results available

A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease

NCT02473445
View directory record Official record
Conditions
Mitochondrial Diseases
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
5
States / cities
San Diego, California • Stanford, California • Akron, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 21, 2026, 10:58 AM EDT
Terminated Phase 2 Interventional

Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders

NCT01195753
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HHLivC
Biological
Lead sponsor
Cytonet GmbH & Co. KG
Industry
Eligibility
1 Day to 5 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
4
States / cities
Palo Alto, California • San Diego, California • New Haven, Connecticut + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 8, 2016 · Synced Oct 21, 2026, 10:58 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

NCT00872729
View directory record Official record
Conditions
Cystinosis
Interventions
Cystagon®, RP103
Drug
Lead sponsor
Amgen
Industry
Eligibility
Not listed
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Dec 19, 2024 · Synced Oct 21, 2026, 10:58 AM EDT
Recruiting No phase listed Observational

The Myelin Disorders Biorepository Project

NCT03047369
View directory record Official record
Conditions
Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Not listed
Enrollment
12,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
23
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 18 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced Oct 21, 2026, 10:58 AM EDT
Terminated Phase 4 Interventional Results available

Cardiac Uptake of 18F Florbetapir in Patients Undergoing Chemotherapy

NCT03333551
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
F18 Florbetapir (amyvid) cardiac PET/CT imaging
Drug
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Oct 21, 2026, 10:58 AM EDT
Recruiting Phase 1Phase 2 Interventional

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

NCT06270316
View directory record Official record
Conditions
Fabry Disease
Interventions
AMT-191
Drug
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
10
States / cities
Birmingham, Alabama • Los Angeles, California • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Oct 21, 2026, 10:58 AM EDT
Enrolling by invitation Phase 1 Interventional

PET Imaging With [18F]Fluselenamyl in People With Cardiac Amyloidosis

NCT07771621
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
[18F]Fluselenamyl (FSA)
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 21, 2026, 10:58 AM EDT
Active, not recruiting No phase listed Observational

Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial

NCT07226622
View directory record Official record
Conditions
Wilson Disease
Interventions
Not listed
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
7
States / cities
Los Angeles, California • Sacramento, California • New Haven, Connecticut + 4 more
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Oct 21, 2026, 10:58 AM EDT
Terminated Phase 1 Interventional Results available

Efficacy, Safety And Tolerability Of PF-06743649 In Gout Subjects.

NCT02187029
View directory record Official record
Conditions
Gout
Interventions
PF-06743649, Placebo
Drug · Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 60 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
4
States / cities
Miami, Florida • South Miami, Florida • Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Nov 11, 2016 · Synced Oct 21, 2026, 10:58 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease

NCT04055831
View directory record Official record
Conditions
Gaucher Disease Type 1
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
16 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 15, 2019 · Synced Oct 21, 2026, 10:58 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Natural History of Apparent Mineralocorticoid Excess Syndrome

NCT00474942
View directory record Official record
Conditions
Apparent Mineralocorticoid Excess Syndrome
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2013
U.S. locations
2
States / cities
New York, New York • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 11, 2015 · Synced Oct 21, 2026, 10:58 AM EDT
Completed Phase 4 Interventional

Initiation of Allopurinol at First Medical Contact for Acute Attacks of Gout

NCT01310673
View directory record Official record
Conditions
Gout, Gout Acute
Interventions
Allopurinol, Placebo
Drug
Lead sponsor
White River Junction Veterans Affairs Medical Center
Federal
Eligibility
18 Years and older · Male only
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2009
U.S. locations
1
States / cities
White River Junction, Vermont
Source: ClinicalTrials.gov public record
Updated Mar 8, 2011 · Synced Oct 21, 2026, 10:58 AM EDT
Completed Phase 3 Interventional Results available

Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

NCT01347073
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100
Drug
Lead sponsor
Amgen
Industry
Eligibility
29 Days to 6 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
8
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Portland, Maine + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2024 · Synced Oct 21, 2026, 10:58 AM 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 21, 2026, 10:58 AM EDT
Recruiting Phase 4 Interventional

Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis

NCT07695701
View directory record Official record
Conditions
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis
Interventions
Acoramidis (AG10) oral tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 80 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 21, 2026, 10:58 AM 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 21, 2026, 10:58 AM EDT
Completed No phase listed Observational

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

NCT04804566
View directory record Official record
Conditions
Fabry Disease
Interventions
This is a non-interventional study
Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
Eagan, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 3, 2024 · Synced Oct 21, 2026, 10:58 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

NCT02651675
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia (HoFH)
Interventions
AAV directed hLDLR gene therapy
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
5
States / cities
Boca Raton, Florida • Kansas City, Kansas • Portland, Oregon + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2023 · Synced Oct 21, 2026, 10:58 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Intracardiac Flow Assessment in Cardiac Amyloidosis

NCT05379101
View directory record Official record
Conditions
Amyloidosis Cardiac, Healthy Adults
Interventions
Cardiac magnetic resonance image (CMR), Transthoracic Echocardiogram (TTE), Six-minute Walk Test
Diagnostic Test
Lead sponsor
Mayo Clinic
Other
Eligibility
40 Years and older
Enrollment
81 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Oct 21, 2026, 10:58 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac Amyloidosis

NCT06034405
View directory record Official record
Conditions
Lumbar Spinal Stenosis, Cardiac Amyloidosis, ATTR Amyloidosis Wild Type, ATTR Gene Mutation, ATTRV122I Amyloidosis, Cardiomyopathy, Hypertrophic
Interventions
Tc99-PYP or Tc99-HDMP Scan
Diagnostic Test
Lead sponsor
Columbia University
Other
Eligibility
50 Years and older
Enrollment
1,663 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2028
U.S. locations
6
States / cities
Beverly Hills, California • Boston, Massachusetts • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 21, 2026, 10:58 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Taste Evaluation of Different Liquid Formulations With Eliglustat

NCT02422654
View directory record Official record
Conditions
Gaucher Disease
Interventions
eliglustat
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 55 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015
U.S. locations
1
States / cities
Evansville, Indiana
Source: ClinicalTrials.gov public record
Updated May 27, 2015 · Synced Oct 21, 2026, 10:58 AM EDT
Withdrawn Phase 1 Interventional Accepts healthy volunteers

O2 Transport and Utilization in Health and Lung Disease

NCT04014712
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 19, 2023 · Synced Oct 21, 2026, 10:58 AM EDT
Completed No phase listed Observational Results available

Chronic Liver Disease in Urea Cycle Disorders

NCT03721367
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
Diagnostic Ultrasound
Other
Lead sponsor
Baylor College of Medicine
Other
Eligibility
5 Years to 60 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 10, 2023 · Synced Oct 21, 2026, 10:58 AM EDT