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ClinicalTrials.gov public records Last synced Oct 21, 2026, 1:36 AM EDT

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Showing 145–168 of 1,509 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
Enrolling by invitation Phase 2 Interventional

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

NCT07393373
View directory record Official record
Conditions
Hypochondroplasia
Interventions
Infigratinib
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 18 Years
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2036
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Oct 21, 2026, 1:36 AM EDT
Completed No phase listed Observational

Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

NCT03609827
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
Interventions
Melphalan
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 4, 2021 · Synced Oct 21, 2026, 1:36 AM EDT
Completed No phase listed Observational Results available

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

NCT00937794
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Neurobehavioral testing, Visual and auditory assessments
Behavioral · Other
Lead sponsor
Shire
Industry
Eligibility
32 Months to 18 Years · Male only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 14, 2021 · Synced Oct 21, 2026, 1:36 AM 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 21, 2026, 1:36 AM EDT
Completed No phase listed Observational

Fabry Outcome Survey (FOS)

NCT03289065
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Not listed
Enrollment
4,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2021
U.S. locations
1
States / cities
Lexington, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 16, 2021 · Synced Oct 21, 2026, 1:36 AM EDT
Completed Not applicable Interventional

A Randomized, Double-Blind Study of the Efficacy and Safety of Oral Ganciclovir for the Prevention of CMV Disease in People Infected With the Human Immunodeficiency Virus

NCT00002095
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years and older
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
19
States / cities
Berkeley, California • Los Angeles, California • San Diego, California + 12 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 21, 2026, 1:36 AM 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 21, 2026, 1:36 AM EDT
Completed Phase 2 Interventional Results available

Dosing Study of Replagal in Patients With Fabry Disease

NCT00068107
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
Baylor Research Institute
Other
Eligibility
Male only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 31, 2015 · Synced Oct 21, 2026, 1:36 AM EDT
Terminated Phase 2 Interventional Results available

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

NCT00668564
View directory record Official record
Conditions
Hurler's Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Sphingolipidoses, Krabbe Disease, Wolman's Disease, Niemann-Pick Disease Type B, Niemann-Pick Disease, Type C
Interventions
Stem Cell Transplantation, Cyclophosphamide, Campath-1H, Busulfan
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 28, 2017 · Synced Oct 21, 2026, 1:36 AM EDT
Completed No phase listed Observational

Genetic Analysis of the Chiari I Malformation

NCT00004738
View directory record Official record
Conditions
Syringomyelia, Type I Arnold Chiari Malformation
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
1 Year and older
Enrollment
152 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 21, 2026, 1:36 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)

NCT05394064
View directory record Official record
Conditions
AMN, AMN Gene Mutation, X-ALD
Interventions
SBT101 Treatment
Genetic
Lead sponsor
SwanBio Therapeutics, Inc.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Oct 21, 2026, 1:36 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Inborn Errors of Metabolism

NCT00176904
View directory record Official record
Conditions
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions
Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 28, 2017 · Synced Oct 21, 2026, 1:36 AM EDT
Completed No phase listed Observational Results available

Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09

NCT02235493
View directory record Official record
Conditions
Hypophosphatasia, Bone Disease
Interventions
Retrospective Case Only
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
5 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 13, 2019 · Synced Oct 21, 2026, 1:36 AM EDT
Completed Phase 2 Interventional Results available

Long-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)

NCT02312687
View directory record Official record
Conditions
X-Linked Hypophosphatemia
Interventions
KRN23
Biological
Lead sponsor
Kyowa Kirin, Inc.
Industry
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
5
States / cities
San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
Source: ClinicalTrials.gov public record
Updated May 6, 2024 · Synced Oct 21, 2026, 1:36 AM EDT
Completed Phase 1 Interventional

A Phase I Study of the Safety of Proleukin (Aldesleukin) in Combination With Ganciclovir and Antiretroviral Therapy in HIV Seropositive Patients With Cytomegalovirus (CMV) Retinitis

NCT00002321
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Aldesleukin
Drug
Lead sponsor
Chiron Corporation
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Toledo, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 21, 2026, 1:36 AM EDT
Not listed Phase 1 Interventional Accepts healthy volunteers

Study of Treatment and Metabolism in Patients With Urea Cycle Disorders

NCT00004307
View directory record Official record
Conditions
Amino Acid Metabolism, Inborn Errors
Interventions
Protein and calorie controlled diet, Ornithine transcarbamylase vector
Behavioral · Genetic
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
6 Months to 64 Years
Enrollment
66 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1999
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 21, 2026, 1:36 AM EDT
Terminated Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Manipulating the Gut Microbiome Study

NCT03181828
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
Acetohydroxamic Acid Oral Tablet, No treatment
Drug · Other
Lead sponsor
Nicholas Ah Mew
Other
Eligibility
18 Years to 60 Years
Enrollment
4 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Dec 15, 2021 · Synced Oct 21, 2026, 1:36 AM EDT
Completed Phase 3 Interventional Results available

PREventative Study Against URate-lowering Drug-induced Gout Exacerbations 1

NCT00829829
View directory record Official record
Conditions
Intercritical Gout
Interventions
Placebo, Rilonacept 80 mg, Rilonacept 160 mg
Other · Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years to 80 Years
Enrollment
241 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
67
States / cities
Birmingham, Alabama • Gilbert, Arizona • Peoria, Arizona + 61 more
Source: ClinicalTrials.gov public record
Updated Apr 28, 2017 · Synced Oct 21, 2026, 1:36 AM EDT
Completed Phase 1 Interventional

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

NCT01898364
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD II), Acid Maltase Deficiency
Interventions
GZ402666
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
7
States / cities
Phoenix, Arizona • Jacksonville, Florida • Kansas City, Kansas + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2023 · Synced Oct 21, 2026, 1:36 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI

NCT02437253
View directory record Official record
Conditions
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI
Interventions
Adalimumab, Placebo
Drug · Other
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Other
Eligibility
5 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Sep 15, 2017 · Synced Oct 21, 2026, 1:36 AM EDT
Completed Not applicable Interventional

Evaluation of PKU Sphere

NCT03419819
View directory record Official record
Conditions
Phenylketonurias
Interventions
PKU Sphere
Dietary Supplement
Lead sponsor
Vitaflo International, Ltd
Industry
Eligibility
3 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Feb 16, 2024 · Synced Oct 21, 2026, 1:36 AM EDT
Recruiting Phase 1Phase 2 Interventional

A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia

NCT06525636
View directory record Official record
Conditions
X-linked Hypophosphatemia
Interventions
KK8123
Drug
Lead sponsor
Kyowa Kirin Co., Ltd.
Industry
Eligibility
18 Years to 65 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
5
States / cities
San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 21, 2026, 1:36 AM EDT
Completed Phase 3 Interventional

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

NCT05166161
View directory record Official record
Conditions
Phenylketonuria
Interventions
PTC923
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
247 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
7
States / cities
Aurora, Colorado • Gainesville, Florida • Indianapolis, Indiana + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 21, 2026, 1:36 AM EDT
Completed No phase listed Observational

Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients

NCT04148001
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia (HoFH)
Interventions
Not listed
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Boca Raton, Florida
Source: ClinicalTrials.gov public record
Updated Feb 2, 2021 · Synced Oct 21, 2026, 1:36 AM EDT