Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.

Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 14, 2026, 9:10 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 241–264 of 1,608 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolism, Inborn Errors Clear all
Completed Phase 2 Interventional

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

NCT04965597
View directory record Official record
Conditions
Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Shwachman-Diamond Syndrome, Hematologic Neoplasm With Germline GATA2 Mutation, Hematologic Neoplasm With Germline SAMD9 Mutation, Hematologic Neoplasm With Germline SAMD9L Mutation
Interventions
Treosulfan, Fludarabine Phosphate, Tacrolimus, Methotrexate, Lapine T-Lymphocyte Immune Globulin, Peripheral Blood Stem Cell Transplantation, Allogeneic Bone Marrow Transplantation, Quality-of-Life Assessment, Echocardiography, Multigated Acquisition Scan, Bone Marrow Biopsy, Bone Marrow Aspiration, Biospecimen Collection, X-Ray Imaging, Computed Tomography
Drug · Biological · Procedure + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
1 Year to 49 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
24
States / cities
Los Angeles, California • San Diego, California • San Francisco, California + 20 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Oct 14, 2026, 9:10 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.

NCT04526197
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, Celecoxib
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Aug 21, 2023 · Synced Oct 14, 2026, 9:10 AM EDT
Completed Phase 1 Interventional

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

NCT01917708
View directory record Official record
Conditions
Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions
Abatacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 26, 2019 · Synced Oct 14, 2026, 9:10 AM EDT
Recruiting Phase 1 Interventional

A Study of the c-Kit Specific Antibody-Drug Conjugate NN3201 for Advanced and/or Metastatic Solid Tumors Known to Express c-Kit

NCT06805825
View directory record Official record
Conditions
Gastrointestinal Stromal Tumors, Small-cell Lung Cancer, Adenoid Cystic Carcinoma, Uveal Melanoma, Neuroendocrine Tumors, Chromophobe Renal Cell Carcinoma, Clear Cell Renal Cell Carcinoma
Interventions
NN3201
Drug
Lead sponsor
Novelty Nobility, Inc.
Industry
Eligibility
18 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
5
States / cities
Ann Arbor, Michigan • Cleveland, Ohio • Portland, Oregon + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2025 · Synced Oct 14, 2026, 9:10 AM EDT
Recruiting Phase 1 Interventional

Assessing the Safety and Tolerability of NMN in DHDDS-CDG

NCT07572825
View directory record Official record
Conditions
DHDDS-Congenital Disorder of Glycosylation, DHDDS-CDG, Congenital Disorder of Glycosylation
Interventions
Nicotinamide Mononucleotide (NMN) Nucleosidase
Drug
Lead sponsor
Eva Morava-Kozicz
Other
Eligibility
4 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Oct 14, 2026, 9:10 AM 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 14, 2026, 9:10 AM EDT
Completed Phase 2 Interventional Results available

A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

NCT00214500
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 55 Years · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
5
States / cities
Los Angeles, California • Decatur, Georgia • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2018 · Synced Oct 14, 2026, 9:10 AM EDT
Not listed Phase 1 Interventional Accepts healthy volunteers

Safety and Efficacy Study of Intramuscular Uricase-PEG 20

NCT01038947
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
Uricase-PEG 20
Biological
Lead sponsor
EnzymeRx
Industry
Eligibility
40 Years to 75 Years
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2010
U.S. locations
2
States / cities
Miami, Florida • Duncansville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 24, 2009 · Synced Oct 14, 2026, 9:10 AM EDT
Completed Phase 2 Interventional Results available

A Study of Fabrazyme in Pediatric Patients With Fabry Disease

NCT00074958
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta)
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
7 Years to 15 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2005
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Apr 2, 2015 · Synced Oct 14, 2026, 9:10 AM EDT
Completed Phase 2 Interventional Results available

A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

NCT02963311
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
ALN-PCSSC, Standard of Care
Drug
Lead sponsor
The Medicines Company
Industry
Eligibility
12 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 18, 2020 · Synced Oct 14, 2026, 9:10 AM EDT
Active, not recruiting Phase 3 Interventional

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

NCT05222906
View directory record Official record
Conditions
Gaucher's Disease Type III
Interventions
Venglustat, imiglucerase
Drug
Lead sponsor
Sanofi
Industry
Eligibility
12 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
4
States / cities
New Haven, Connecticut • Iowa City, Iowa • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 14, 2026, 9:10 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Validation of Diagnostics to Identify Glucose-6-phosphate Dehydrogenase Activity in the US

NCT04010695
View directory record Official record
Conditions
G6PD Deficiency
Interventions
SD Biosensor G6PD Analyzer, Pointe Scientific Test Kit, HemoCue System
Diagnostic Test
Lead sponsor
PATH
Other
Eligibility
18 Years to 65 Years
Enrollment
250 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 10, 2022 · Synced Oct 14, 2026, 9:10 AM EDT
Completed Not applicable Interventional

MRI and Ultrasound Findings in Patients With Gout and Normal Plain Radiographs

NCT00584311
View directory record Official record
Conditions
Gout
Interventions
MRI and Ultrasound
Other
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 80 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jun 9, 2008 · Synced Oct 14, 2026, 9:10 AM EDT
Terminated Not applicable Interventional Results available

Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I

NCT02232477
View directory record Official record
Conditions
Mucopolysaccharidosis I, Cognitive Decline
Interventions
Intrathecal recombinant human alpha iduronidase
Drug
Lead sponsor
agnes chen
Other
Eligibility
6 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Oct 31, 2023 · Synced Oct 14, 2026, 9:10 AM EDT
Recruiting No phase listed Observational

Natural History of Wilson Disease

NCT03334292
View directory record Official record
Conditions
Wilson Disease
Interventions
Not listed
Lead sponsor
Yale University
Other
Eligibility
Not listed
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2029
U.S. locations
4
States / cities
New Haven, Connecticut • Orlando, Florida • Houston, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Oct 14, 2026, 9:10 AM EDT
Recruiting No phase listed Observational

RCDP Natural History Study

NCT04031287
View directory record Official record
Conditions
Rhizomelic Chondrodysplasia Punctata
Interventions
Not listed
Lead sponsor
MED-LIFE DISCOVERIES LP
Industry
Eligibility
6 Months to 21 Years
Enrollment
75 participants
Timeline
2019 – 2027
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated Oct 9, 2026 · Synced Oct 14, 2026, 9:10 AM EDT
Recruiting No phase listed Observational

Natural History of MADD

NCT07734090
View directory record Official record
Conditions
Multiple Acyl-CoA Dehydrogenase Deficiency
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Aurora, Colorado • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 14, 2026, 9:10 AM EDT
Completed No phase listed Observational

Alglucosidase Alfa Pompe Safety Sub-Registry

NCT01710813
View directory record Official record
Conditions
Pompe Disease
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
6
States / cities
Phoenix, Arizona • Durham, North Carolina • Hershey, Pennsylvania + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2021 · Synced Oct 14, 2026, 9:10 AM EDT
Completed No phase listed Observational Results available

The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment

NCT02763215
View directory record Official record
Conditions
Wilson Disease
Interventions
Standard of Care Medications
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
5
States / cities
New Haven, Connecticut • Chicago, Illinois • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2020 · Synced Oct 14, 2026, 9:10 AM EDT
Completed Not applicable Interventional

Open Label Ganciclovir Therapy for Sight- or Life-Threatening Cytomegalovirus Disease in the Immunocompromised Patient

NCT00002025
View directory record Official record
Conditions
Cytomegalovirus Infections, Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
3 Months and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 14, 2026, 9:10 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

NCT01942590
View directory record Official record
Conditions
Pompe Disease
Interventions
Clenbuterol, Placebo
Drug
Lead sponsor
Dwight Koeberl, M.D., Ph.D.
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 2, 2019 · Synced Oct 14, 2026, 9:10 AM EDT
Completed Phase 3 Interventional Results available

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

NCT02795676
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102 (pegunigalsidase alfa), agalsidase beta
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years to 60 Years
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Orange, California + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 14, 2026, 9:10 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Tweens to Teens Project at Penn State

NCT01184651
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
Questionnaires/Interviews
Behavioral
Lead sponsor
Penn State University
Other
Eligibility
10 Years to 13 Years · Female only
Enrollment
188 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2013
U.S. locations
1
States / cities
University Park, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 8, 2018 · Synced Oct 14, 2026, 9:10 AM EDT
Active, not recruiting Phase 2 Interventional

Metyrapone for Mild Autonomous Cortisol Secretion (MACS)

NCT06106295
View directory record Official record
Conditions
Autonomous Cortisol Secretion, Mild Autonomous Cortisol Secretion (MACS)
Interventions
Metyrapone
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 25, 2026 · Synced Oct 14, 2026, 9:10 AM EDT