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ClinicalTrials.gov public records Last synced Oct 17, 2026, 1:08 AM EDT

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Showing 841–864 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Active, not recruiting Phase 3 Interventional

Long Term Extension Study in Patients With Primary Hyperoxaluria

NCT04042402
View directory record Official record
Conditions
Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases, Genetic Disease, Primary Hyperoxaluria Type 3 (PH3)
Interventions
DCR-PHXC
Drug
Lead sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Industry
Eligibility
Not listed
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2030
U.S. locations
4
States / cities
San Francisco, California • Boston, Massachusetts • Rochester, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 17, 2026, 1:08 AM EDT
Completed No phase listed Observational

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

NCT04470713
View directory record Official record
Conditions
GM1 Gangliosidosis, GM2 Gangliosidosis, Gaucher Disease, Type 2, Tay-Sachs Disease, AB Variant Gangliosidosis GM2, Sandhoff Disease
Interventions
Not listed
Lead sponsor
Idorsia Pharmaceuticals Ltd.
Industry
Eligibility
0 Months and older
Enrollment
226 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
3
States / cities
Chicago, Illinois • Rochester, Minnesota • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Nov 8, 2021 · Synced Oct 17, 2026, 1:08 AM EDT
Completed Phase 2Phase 3 Interventional Results available

A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome

NCT03098797
View directory record Official record
Conditions
Barth Syndrome
Interventions
Elamipretide, Placebo
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 16, 2024 · Synced Oct 17, 2026, 1:08 AM EDT
Completed Phase 3 Interventional Results available

A Study of PTC923 in Participants With Phenylketonuria

NCT05099640
View directory record Official record
Conditions
Phenylketonuria
Interventions
PTC923, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
12
States / cities
Stanford, California • Aurora, Colorado • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 10, 2024 · Synced Oct 17, 2026, 1:08 AM EDT
Completed Phase 2 Interventional

A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension

NCT00325962
View directory record Official record
Conditions
Hypertension
Interventions
6R-BH4 (sapropterin dihydrochloride)
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
24
States / cities
Hoover, Alabama • Huntsville, Alabama • Mobile, Alabama + 21 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2009 · Synced Oct 17, 2026, 1:08 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 17, 2026, 1:08 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

NCT03153319
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
Interventions
Adalimumab Injection [Humira], Saline Solution for Injection
Drug
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Other
Eligibility
5 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Feb 2, 2026 · Synced Oct 17, 2026, 1:08 AM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

CLinical Prediction Rule Score in Veterans

NCT01418781
View directory record Official record
Conditions
Gout
Interventions
Not listed
Lead sponsor
jasvinder singh
Federal
Eligibility
18 Years to 110 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 13, 2024 · Synced Oct 17, 2026, 1:08 AM EDT
Recruiting No phase listed Observational

Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)

NCT04404530
View directory record Official record
Conditions
Phenylketonurias
Interventions
Palynziq
Drug
Lead sponsor
Emory University
Other
Eligibility
16 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
2
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jan 14, 2026 · Synced Oct 17, 2026, 1:08 AM EDT
Completed Phase 3 Interventional Results available

An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia

NCT00694109
View directory record Official record
Conditions
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases, Hyperlipoproteinemia Type II, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Metabolic Disorder, Congenital Abnormalities, Hypercholesterolemia, Hyperlipoproteinemias, Dyslipidemias, Lipid Metabolism Disorders
Interventions
Mipomersen Sodium
Drug
Lead sponsor
Kastle Therapeutics, LLC
Industry
Eligibility
12 Years and older
Enrollment
144 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
20
States / cities
Mission Viejo, California • Newport Beach, California • Bridgeport, Connecticut + 17 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2016 · Synced Oct 17, 2026, 1:08 AM EDT
Completed Not applicable Interventional

Studies in Porphyria IV: Gonadotropin-Releasing Hormone (GnRH) Analogues for Prevention of Cyclic Attacks

NCT00004330
View directory record Official record
Conditions
Porphyria
Interventions
luteinizing hormone-releasing factor
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 55 Years · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1987
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 17, 2026, 1:08 AM EDT
Recruiting No phase listed Observational

GBE Deficiency (GSD IV and APBD) Natural History Study

NCT02683512
View directory record Official record
Conditions
Glycogen Storage Disease Type IV, Adult Polyglucosan Body Disease, GSD4, GSD IV, APBD
Interventions
No intervention
Other
Lead sponsor
Duke University
Other
Eligibility
0 Years to 90 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2035
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 17, 2026, 1:08 AM EDT
Not listed Phase 3 Interventional

AT-007 in Adult Subjects With Classic Galactosemia (CG)

NCT05418829
View directory record Official record
Conditions
Classic Galactosemia
Interventions
AT-007
Drug
Lead sponsor
Applied Therapeutics, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Berlin, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 14, 2022 · Synced Oct 17, 2026, 1:08 AM EDT
Completed Phase 3 Interventional Results available

Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

NCT00925301
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat hydrochloride, Placebo
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
16 Years to 74 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
13
States / cities
Los Angeles, California • San Francisco, California • Decatur, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2018 · Synced Oct 17, 2026, 1:08 AM EDT
Recruiting Phase 2 Interventional

Safety and Efficacy of ABP-745 in Participants With an Acute Gout Flare

NCT07145229
View directory record Official record
Conditions
Acute Gouty Arthritis, Gout Flare, Gout Flares, Acute Gout Flare
Interventions
ABP-745 Dose A, ABP-745 Dose B, Colchicine, Placebo
Drug
Lead sponsor
Atom Therapeutics Co., Ltd
Industry
Eligibility
18 Years to 70 Years
Enrollment
380 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
14
States / cities
Peoria, Arizona • La Mesa, California • Norcross, Georgia + 11 more
Source: ClinicalTrials.gov public record
Updated Dec 8, 2025 · Synced Oct 17, 2026, 1:08 AM EDT
Terminated Phase 3 Interventional Results available

CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases

NCT02697136
View directory record Official record
Conditions
Familial Hypoalphalipoproteinemia
Interventions
CER-001, Placebo
Drug
Lead sponsor
Cerenis Therapeutics, SA
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jul 22, 2025 · Synced Oct 17, 2026, 1:08 AM EDT
Completed No phase listed Observational

Antiproteinuric Agents and Fabry Disease

NCT00343577
View directory record Official record
Conditions
Fabry Disease, Proteinuria
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
14 Years to 95 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Nov 19, 2013 · Synced Oct 17, 2026, 1:08 AM EDT
Terminated Phase 2 Interventional Results available

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

NCT01609062
View directory record Official record
Conditions
Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
Interventions
BMN 110
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
7 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
4
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2016 · Synced Oct 17, 2026, 1:08 AM EDT
Recruiting Phase 2 Interventional

A Study to Investigate the Clinical Effect and the Safety of PRX-115 Infused Intravenously at Different Dosing Regimens, With and Without Methotrexate, Versus Placebo in Adults Gout Patients (RELEASE)

NCT07280156
View directory record Official record
Conditions
Gout
Interventions
PRX-115, Methotrexate (MTX), PRX-115 placebo, Placebo-Methotrexate
Biological · Drug · Other
Lead sponsor
Protalix
Industry
Eligibility
18 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
26
States / cities
Glendale, Arizona • Little Rock, Arkansas • Anaheim, California + 20 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 17, 2026, 1:08 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Study to Investigate the Effects of Food on Relative Bioavailability of ABP-671 Tablets in Healthy Subjects

NCT04303039
View directory record Official record
Conditions
Gout, Food-drug Interaction
Interventions
ABP-671
Drug
Lead sponsor
Atom Therapeutics Co., Ltd
Industry
Eligibility
18 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 23, 2020 · Synced Oct 17, 2026, 1:08 AM 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 17, 2026, 1:08 AM EDT
Completed Phase 2 Interventional Results available

RDEA3170 and Febuxostat Combination Study in Gout Subjects

NCT02246673
View directory record Official record
Conditions
Gout
Interventions
RDEA3170 15 mg, RDEA3170 10 mg, RDEA3170 2.5, RDEA3170 5 mg, Febuxostat 40 mg, Febuxostat 80 mg
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
6
States / cities
Anaheim, California • Glendale, California • South Miami, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2017 · Synced Oct 17, 2026, 1:08 AM EDT
Suspended Not applicable Interventional

The PhOCus Trial: Implementation of Pharmacogenomic Testing in Oncology Care

NCT04541381
View directory record Official record
Conditions
Gastrointestinal Cancer, Head and Neck Cancer, Dihydropyrimidine Dehydrogenase Deficiency, UGT1A1 Gene Mutation, Breast Cancer
Interventions
Availability of clinical decision support based on pharmacogenomic results.
Other
Lead sponsor
University of Chicago
Other
Eligibility
18 Years and older
Enrollment
860 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Oct 17, 2026, 1:08 AM EDT
Completed Not applicable Interventional Results available

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

NCT01626092
View directory record Official record
Conditions
Lysosomal Storage Disease, Peroxisomal Disorder
Interventions
Campath-1H, Clofarabine, Melphalan, Total Body Irradiation with Marrow Boosting, Hematopoietic stem cell transplantation, Cyclosporine A, Mycophenolate mofetil
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 5, 2017 · Synced Oct 17, 2026, 1:08 AM EDT