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ClinicalTrials.gov public records Last synced Oct 21, 2026, 2:41 PM EDT

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Showing 241–264 of 1,510 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
Terminated Phase 1Phase 2 Interventional

A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency

NCT05154890
View directory record Official record
Conditions
Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
Interventions
Pegtarviliase IV, Pegtarviliase SC
Drug
Lead sponsor
Aeglea Biotherapeutics
Industry
Eligibility
12 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 27, 2023 · Synced Oct 21, 2026, 2:41 PM EDT
Withdrawn Phase 4 Interventional

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

NCT02528617
View directory record Official record
Conditions
Gaucher Disease Type 1, Gaucher Disease Type 3
Interventions
Velaglucerase alfa
Drug
Lead sponsor
Baylor Research Institute
Other
Eligibility
4 Years to 14 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 2, 2018 · Synced Oct 21, 2026, 2:41 PM EDT
Completed Phase 2 Interventional Results available

HSCT for High Risk Inherited Inborn Errors

NCT00383448
View directory record Official record
Conditions
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease, Sandhoffs Disease, Wolman Disease, I-Cell Disease, Sanfilippo Syndrome, GM1 Gangliosidosis
Interventions
Clofarabine, Total body Irradiation, Melphalan, Hematopoietic Stem Cell Transplantation, Alemtuzumab, mycophenylate mofetil, Cyclosporine A, Hydroxyurea
Drug · Procedure · Biological + 1 more
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 70 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 11, 2019 · Synced Oct 21, 2026, 2:41 PM EDT
Recruiting No phase listed Observational

Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study

NCT07301216
View directory record Official record
Conditions
Wilson Disease
Interventions
Not listed
Lead sponsor
Yale University
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Oct 21, 2026, 2:41 PM EDT
Terminated Phase 1 Interventional

Hepatocyte Transplantation for Liver Based Metabolic Disorders

NCT01345578
View directory record Official record
Conditions
Metabolic Diseases
Interventions
human hepatocyte transplantation, Preparative Radiation Therapy
Drug · Radiation
Lead sponsor
Ira Fox
Other
Eligibility
Up to 21 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 7, 2022 · Synced Oct 21, 2026, 2:41 PM EDT
Not yet recruiting Not applicable Interventional

Cascade Testing In Identifying At-risk Family Members Of Individuals With Familial Hypercholesterolemia (FH) In PCP

NCT07653555
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
Cascade testing, Standard of Care
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
1 Year and older
Enrollment
480 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 21, 2026, 2:41 PM 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 21, 2026, 2:41 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Intervention and Outcomes in Duarte Galactosemia

NCT02519504
View directory record Official record
Conditions
Duarte Galactosemia
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
6 Years to 12 Years
Enrollment
566 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 19, 2019 · Synced Oct 21, 2026, 2:41 PM EDT
Completed Phase 2 Interventional Results available

Theophylline as a Treatment for Children With Pseudohypoparathyroidism Type 1a (Albright Hereditary Osteodystrophy)

NCT02463409
View directory record Official record
Conditions
Pseudohypoparathyroidism Type 1a, Albright Hereditary Osteodystrophy
Interventions
Theophylline
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
10 Years to 21 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 26, 2017 · Synced Oct 21, 2026, 2:41 PM EDT
Recruiting No phase listed Observational

Longitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum Disorder

NCT06190626
View directory record Official record
Conditions
Zellweger Spectrum Disorder
Interventions
Not listed
Lead sponsor
McGill University Health Centre/Research Institute of the McGill University Health Centre
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 21, 2026, 2:41 PM EDT
Completed Phase 3 Interventional Results available

Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

NCT03156621
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
Alirocumab, Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
4
States / cities
Boca Raton, Florida • New York, New York • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 29, 2021 · Synced Oct 21, 2026, 2:41 PM EDT
Completed Phase 1Phase 2 Interventional Results available

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

NCT02675465
View directory record Official record
Conditions
Pompe Disease
Interventions
ATB200, AT2221
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
12
States / cities
Phoenix, Arizona • Orange, California • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced Oct 21, 2026, 2:41 PM EDT
No Longer Available No phase listed Expanded access

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

NCT01461304
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
1 Month and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 10, 2021 · Synced Oct 21, 2026, 2:41 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCT04093349
View directory record Official record
Conditions
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Interventions
SPK-3006
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2032
U.S. locations
10
States / cities
Phoenix, Arizona • Orange, California • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 27, 2024 · Synced Oct 21, 2026, 2:41 PM EDT
Active, not recruiting No phase listed Observational

Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

NCT05634512
View directory record Official record
Conditions
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
Interventions
Laronidase therapy and a stem cell transplant
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
0 Years to 3 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Oct 21, 2026, 2:41 PM EDT
Recruiting Phase 1 Interventional

A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease

NCT06164730
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease
Interventions
VERVE-102
Drug
Lead sponsor
Verve Therapeutics, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
10
States / cities
Dothan, Alabama • Pomona, California • Boca Raton, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 21, 2026, 2:41 PM EDT
Completed Phase 3 Interventional Results available

A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)

NCT00730236
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
AEGR-733
Drug
Lead sponsor
Aegerion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
2
States / cities
Los Angeles, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 20, 2018 · Synced Oct 21, 2026, 2:41 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers

Curcumin (Tumeric) in the Treatment of Irritable Bowel Syndrome: A Randomized-Controlled Trial

NCT00779493
View directory record Official record
Conditions
Irritable Bowel Syndrome
Interventions
curcumin, placebo
Dietary Supplement
Lead sponsor
Kaiser Permanente
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 11, 2015 · Synced Oct 21, 2026, 2:41 PM EDT
Completed No phase listed Observational

Mitochondrial Video Assessment Source Material Study

NCT06180421
View directory record Official record
Conditions
PolG, Primary Mitochondrial Myopathy
Interventions
Not listed
Lead sponsor
The Emmes Company, LLC
Industry
Eligibility
16 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Kingston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Oct 21, 2026, 2:41 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Clinical Evaluation of NeoPlex4 Assay and NeoPlex System

NCT01488721
View directory record Official record
Conditions
Adrenal Hyperplasia, Congenital, Congenital Hypothyroidism, Cystic Fibrosis
Interventions
Not listed
Lead sponsor
Luminex Corporation
Industry
Eligibility
1 Day to 7 Days
Enrollment
7,462 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2012
U.S. locations
3
States / cities
Albany, New York • Nashville, Tennessee • Taylorsville, Utah
Source: ClinicalTrials.gov public record
Updated Aug 17, 2012 · Synced Oct 21, 2026, 2:41 PM EDT
Enrolling by invitation Phase 4 Interventional

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

NCT05492799
View directory record Official record
Conditions
MPS III B
Interventions
AX 250
Drug
Lead sponsor
Allievex Corporation
Industry
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
2
States / cities
Oakland, California • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 9, 2023 · Synced Oct 21, 2026, 2:41 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

NCT06181136
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIA
Interventions
DNL126
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
0 Years to 18 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
4
States / cities
Oakland, California • Iowa City, Iowa • Chapel Hill, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 21, 2026, 2:41 PM EDT
Completed Phase 2 Interventional Results available

A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria

NCT00879034
View directory record Official record
Conditions
Progeria, Hutchinson-Gilford Syndrome
Interventions
Lonafarnib, Zoledronic Acid, Pravastatin
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 13, 2019 · Synced Oct 21, 2026, 2:41 PM EDT
Not listed Phase 2 Interventional

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

NCT03784287
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIB, MPS III B
Interventions
AX 250
Drug
Lead sponsor
Allievex Corporation
Industry
Eligibility
0 Years to 18 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
2
States / cities
Oakland, California • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 18, 2023 · Synced Oct 21, 2026, 2:41 PM EDT