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ClinicalTrials.gov public records Last synced Oct 15, 2026, 6:23 PM EDT

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Showing 529–552 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Completed Phase 2 Interventional

Bucillamine for the Treatment of Acute Gout Flare in Subjects With Moderate to Severe Gout

NCT02330796
View directory record Official record
Conditions
Gout
Interventions
Bucillamine, Colchicine
Drug
Lead sponsor
Revive Therapeutics, Ltd.
Industry
Eligibility
18 Years to 80 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015
U.S. locations
3
States / cities
San Roman, California • Arlington, Texas • San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Oct 5, 2016 · Synced Oct 15, 2026, 6:23 PM EDT
Completed No phase listed Observational

23andMe IPF Research Study

NCT05028894
View directory record Official record
Conditions
Idiopathic Pulmonary Fibrosis
Interventions
No intervention
Other
Lead sponsor
23andMe, Inc.
Industry
Eligibility
18 Years and older
Enrollment
690 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Sunnyvale, California
Source: ClinicalTrials.gov public record
Updated Dec 9, 2024 · Synced Oct 15, 2026, 6:23 PM EDT
Withdrawn Phase 1 Interventional

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

NCT05238324
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Genetic HMI-203
Biological
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 45 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
5
States / cities
Oakland, California • New Haven, Connecticut • Hackensack, New Jersey + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2023 · Synced Oct 15, 2026, 6:23 PM EDT
Completed Phase 2 Interventional

Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria

NCT04534842
View directory record Official record
Conditions
Phenylketonuria
Interventions
SYNB1618, SYNB1934
Drug
Lead sponsor
Synlogic
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
11
States / cities
Aurora, Colorado • Boca Raton, Florida • Gainesville, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 17, 2022 · Synced Oct 15, 2026, 6:23 PM EDT
Completed Phase 2 Interventional Results available

Safety and Effect of Doxycycline in Patients With Amyloidosis

NCT01677286
View directory record Official record
Conditions
Amyloidosis
Interventions
Doxycycline 100 mg po bid x 12 months
Drug
Lead sponsor
Boston University
Other
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 1, 2017 · Synced Oct 15, 2026, 6:23 PM EDT
Completed Phase 2 Interventional Results available

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

NCT02678689
View directory record Official record
Conditions
Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
Interventions
BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1), Intracerebroventricular access device
Biological · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 17 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 17, 2025 · Synced Oct 15, 2026, 6:23 PM EDT
Enrolling by invitation No phase listed Observational

Long-Term Follow Up Study of Subjects Previously Administered HMI 102

NCT04348708
View directory record Official record
Conditions
Phenylketonuria, PAH Deficiency
Interventions
HMI-102
Genetic
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 30, 2022 · Synced Oct 15, 2026, 6:23 PM EDT
Completed Phase 1 Interventional

Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease

NCT01747135
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
VTS-270
Drug
Lead sponsor
Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
Industry
Eligibility
2 Years to 25 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 23, 2021 · Synced Oct 15, 2026, 6:23 PM EDT
Completed Phase 3 Interventional Results available

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

NCT00712348
View directory record Official record
Conditions
Gaucher Disease
Interventions
Taliglucerase alfa
Drug
Lead sponsor
Pfizer
Industry
Eligibility
2 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2018 · Synced Oct 15, 2026, 6:23 PM EDT
Completed Phase 3 Interventional Results available

Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks

NCT03180840
View directory record Official record
Conditions
Fabry Disease
Interventions
Pegunigalsidase alfa
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years to 60 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
7
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 15, 2026, 6:23 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria

NCT03116685
View directory record Official record
Conditions
Primary Hyperoxaluria
Interventions
Oxabact OC5 - Oxalobacter formigenes HC-1, Placebo
Biological · Other
Lead sponsor
OxThera
Industry
Eligibility
2 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Dec 10, 2021 · Synced Oct 15, 2026, 6:23 PM EDT
Terminated No phase listed Observational

Natural History Study of Batten Disease

NCT04644549
View directory record Official record
Conditions
Batten Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis CLN3
Interventions
Not listed
Lead sponsor
Emily de los Reyes
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jul 30, 2025 · Synced Oct 15, 2026, 6:23 PM EDT
Completed Phase 1 Interventional Results available

A Gene Therapy Study in Patients With Gaucher Disease Type 1

NCT05324943
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
FLT201
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
2
States / cities
Los Angeles, California • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Oct 15, 2026, 6:23 PM EDT
Active, not recruiting No phase listed Observational

Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae

NCT03285425
View directory record Official record
Conditions
Batten Disease, CLN6
Interventions
Natural history
Other
Lead sponsor
Emily de los Reyes
Other
Eligibility
2 Years to 25 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 30, 2025 · Synced Oct 15, 2026, 6:23 PM EDT
Completed No phase listed Observational

Immune Response in Subjects With Fabry Disease Who Are Switching From Agalsidase Alfa to Agalsidase Beta

NCT01745185
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
7 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 4, 2017 · Synced Oct 15, 2026, 6:23 PM EDT
Completed Early Phase 1 Interventional

Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I

NCT03665636
View directory record Official record
Conditions
Glycogen Storage Disease Type I
Interventions
Triheptanoin
Drug
Lead sponsor
Areeg El-Gharbawy
Other
Eligibility
1 Month to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 15, 2021 · Synced Oct 15, 2026, 6:23 PM 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 15, 2026, 6:23 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ABP-671 in Healthy Subjects

NCT03906006
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
ABP-671, Placebo
Drug · Other
Lead sponsor
Atom Therapeutics Co., Ltd
Industry
Eligibility
18 Years to 45 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Tempe, Arizona
Source: ClinicalTrials.gov public record
Updated Jul 29, 2020 · Synced Oct 15, 2026, 6:23 PM EDT
Completed No phase listed Observational

PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

NCT00778206
View directory record Official record
Conditions
Phenylketonuria, Hyperphenylalaninaemia
Interventions
Kuvan
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
1,887 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2021
U.S. locations
45
States / cities
Los Angeles, California • Orange, California • San Diego, California + 38 more
Source: ClinicalTrials.gov public record
Updated Oct 12, 2022 · Synced Oct 15, 2026, 6:23 PM EDT
Withdrawn Phase 3 Interventional

Extension Study in Primary Distal Renal Tubular Acidosis

NCT03831152
View directory record Official record
Conditions
Acidosis, Renal Tubular
Interventions
ADV7103
Drug
Lead sponsor
Advicenne Pharma
Industry
Eligibility
4 Months to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Feb 9, 2026 · Synced Oct 15, 2026, 6:23 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®

NCT00920647
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg), Idursulfase IT (30 mg)
Other · Drug
Lead sponsor
Shire
Industry
Eligibility
3 Years to 18 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 28, 2021 · Synced Oct 15, 2026, 6:23 PM EDT
Completed Phase 3 Interventional Results available

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

NCT02432144
View directory record Official record
Conditions
Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
Interventions
UX003
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
5 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
4
States / cities
Oakland, California • Orange, California • Minneapolis, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Oct 15, 2026, 6:23 PM EDT
Recruiting Phase 1 Interventional

AAVrh10-PCCA Gene Therapy for Propionic Acidemia

NCT07643844
View directory record Official record
Conditions
Propionic Acidemia
Interventions
AAVrh10-PCCA low dose, AAVrh10-PCCA middle dose, AAVrh10-PCCA high dose
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
6 Months to 2 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2033
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 15, 2026, 6:23 PM EDT
Not listed Phase 2Phase 3 Interventional

N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia

NCT00843921
View directory record Official record
Conditions
Inborn Errors of Metabolism
Interventions
N-carbamylglutamate
Drug
Lead sponsor
Mendel Tuchman
Other
Eligibility
1 Day to 70 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2020
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 17, 2020 · Synced Oct 15, 2026, 6:23 PM EDT