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 2, 2026, 5:39 AM EDT

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

Showing 25–48 of 1,957 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Disorders of Metabolism Clear all
No Longer Available No phase listed Expanded access

Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

NCT02300753
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
EPI-743
Drug
Lead sponsor
Edison Pharmaceuticals Inc
Industry
Eligibility
8 Years to 65 Years
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 25, 2014 · Synced Oct 2, 2026, 5:39 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

NCT00787865
View directory record Official record
Conditions
Krabbe Disease
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
Up to 17 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 6, 2026 · Synced Oct 2, 2026, 5:39 AM EDT
Terminated Phase 4 Interventional Results available

Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias

NCT02443545
View directory record Official record
Conditions
Iron Overload, Sickle Cell Disease, Other Anemias
Interventions
Deferiprone
Drug
Lead sponsor
ApoPharma
Industry
Eligibility
3 Years and older
Enrollment
134 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
5
States / cities
Oakland, California • Ann Arbor, Michigan • Detroit, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 10, 2024 · Synced Oct 2, 2026, 5:39 AM EDT
Terminated Not applicable Interventional Results available

Methadone, Morphine, or Oxycodone in Treating Pain in Patients With Cancer

NCT00726830
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Pain, Precancerous Condition, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
methadone hydrochloride, morphine sulfate, oxycodone hydrochloride
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
2
States / cities
Spartanburg, South Carolina • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 24, 2020 · Synced Oct 2, 2026, 5:39 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Bioavailability Study of Ciprofloxacin 1000 mg ER Tablets of Dr. Reddy's Under Non-Fasting Conditions

NCT01372358
View directory record Official record
Conditions
Fed
Interventions
CIPRO®XR
Drug
Lead sponsor
Dr. Reddy's Laboratories Limited
Industry
Eligibility
18 Years to 45 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 13, 2011 · Synced Oct 2, 2026, 5:39 AM EDT
Terminated Phase 1 Interventional

12-O-Tetradecanoylphorbol-13-acetate in Treating Patients With Hematologic Cancer or Bone Marrow Disorder

NCT00004058
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Precancerous/Nonmalignant Condition
Interventions
tetradecanoylphorbol acetate
Drug
Lead sponsor
University of Medicine and Dentistry of New Jersey
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2004
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 26, 2010 · Synced Oct 2, 2026, 5:39 AM EDT
Completed Phase 2 Interventional

A Single-Dose Open-Label Study of XOMA 358 in Subjects With Congenital Hyperinsulinism

NCT02604485
View directory record Official record
Conditions
Congenital Hyperinsulinism
Interventions
Cohort 1, Cohort 2, Cohort 3, Cohort 4
Drug
Lead sponsor
XOMA (US) LLC
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 6, 2017 · Synced Oct 2, 2026, 5:39 AM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

NCT01994889
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Tafamidis, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 90 Years
Enrollment
441 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
50
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 24 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2019 · Synced Oct 2, 2026, 5:39 AM EDT
Completed Phase 3 Interventional Results available

Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

NCT01744782
View directory record Official record
Conditions
Cystinosis
Interventions
RP103
Drug
Lead sponsor
Amgen
Industry
Eligibility
Up to 6 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 2, 2026, 5:39 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study of the Absorption, Metabolism, and Excretion of GBT440 in Healthy Male Subjects

NCT02497924
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
GBT440
Drug
Lead sponsor
Global Blood Therapeutics
Industry
Eligibility
18 Years to 55 Years · Male only
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 12, 2017 · Synced Oct 2, 2026, 5:39 AM EDT
Completed Phase 2 Interventional Results available

Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria

NCT02180412
View directory record Official record
Conditions
Acute Porphyrias
Interventions
Panhematin, Glucose
Biological · Other
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
18 Years to 100 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 11, 2025 · Synced Oct 2, 2026, 5:39 AM EDT
Recruiting Phase 3 Interventional

Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia

NCT06597019
View directory record Official record
Conditions
Familial Hypercholesterolemia - Heterozygous
Interventions
Inclisiran, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
6 Years to 11 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
9
States / cities
San Francisco, California • Washington D.C., District of Columbia • Boca Raton, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 2, 2026, 5:39 AM EDT
Recruiting Phase 1Phase 2 Interventional

NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease

NCT07398508
View directory record Official record
Conditions
Menkes Disease
Interventions
Droxidopa Oral Product, Placebo Control
Drug · Other
Lead sponsor
Stephen G. Kaler
Other
Eligibility
7 Years to 17 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Oct 2, 2026, 5:39 AM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Acute Gouty Arthritis

NCT03002974
View directory record Official record
Conditions
Acute Gouty Arthritis
Interventions
Anakinra 100 mg, Triamcinolone Acetonide 40 mg, Placebo to Anakinra 100 mg, Placebo to Triamcinolone Acetonide 40 mg
Drug
Lead sponsor
Swedish Orphan Biovitrum
Industry
Eligibility
18 Years and older
Enrollment
165 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
37
States / cities
Birmingham, Alabama • Gulf Shores, Alabama • Mobile, Alabama + 32 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2020 · Synced Oct 2, 2026, 5:39 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Intranasal Oxytocin for Infants With Prader-Willi Syndrome

NCT03245762
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Oxytocin, Placebo
Drug
Lead sponsor
University of Florida
Other
Eligibility
1 Week to 6 Months
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Mar 26, 2020 · Synced Oct 2, 2026, 5:39 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Gene Function in Bone Marrow Cells From Patients With Fanconi Anemia and From Healthy Participants

NCT00896740
View directory record Official record
Conditions
Fanconi Anemia
Interventions
microarray analysis, biopsy
Genetic · Procedure
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
1 Year to 55 Years
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2007
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Dec 4, 2017 · Synced Oct 2, 2026, 5:39 AM EDT
Completed Phase 1 Interventional

Study to Evaluate Safety, Tolerability, PK and PD of DCR-PHXC in PH Type 3 Patients

NCT04555486
View directory record Official record
Conditions
Primary Hyperoxaluria Type 3
Interventions
DCR-PHXC, Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Industry
Eligibility
6 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
3
States / cities
Boston, Massachusetts • Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 2, 2026, 5:39 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

NCT04046224
View directory record Official record
Conditions
Fabry Disease
Interventions
ST-920
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
9
States / cities
Irvine, California • Tampa, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 2, 2026, 5:39 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome

NCT00467831
View directory record Official record
Conditions
Hermansky-Pudlak Syndrome (HPS), Pulmonary Fibrosis, Oculocutaneous Albinism, Platelet Storage Pool Deficiency, Metabolic Disease
Interventions
Losartan, Zileuton, N-Acetylcysteine, Pravastatin, Erythromycin
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 70 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 2, 2013 · Synced Oct 2, 2026, 5:39 AM EDT
Not listed Not applicable Interventional

Identification of Undiagnosed Gaucher Disease

NCT01716741
View directory record Official record
Conditions
Gaucher Disease
Interventions
Enzyme analysis
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 30, 2012 · Synced Oct 2, 2026, 5:39 AM EDT
Completed Not applicable Interventional Results available

Feasibility of Outpatient Closed Loop Control With the iLet Bionic Pancreas in Cystic Fibrosis Related Diabetes

NCT03258853
View directory record Official record
Conditions
Cystic Fibrosis-related Diabetes
Interventions
Bionic Pancreas, Usual Care
Device · Other
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
10 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 20, 2023 · Synced Oct 2, 2026, 5:39 AM EDT
Completed No phase listed Observational

Longitudinal Studies of Brain Structure and Function in MPS Disorders

NCT01870375
View directory record Official record
Conditions
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
6 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2019
U.S. locations
3
States / cities
Oakland, California • Minneapolis, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 6, 2019 · Synced Oct 2, 2026, 5:39 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Natural History of Apparent Mineralocorticoid Excess Syndrome

NCT00474942
View directory record Official record
Conditions
Apparent Mineralocorticoid Excess Syndrome
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2013
U.S. locations
2
States / cities
New York, New York • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 11, 2015 · Synced Oct 2, 2026, 5:39 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT04958642
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Adrabetadex
Drug
Lead sponsor
Mandos LLC
Industry
Eligibility
4 Years to 21 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Orange, California • San Francisco, California + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 29, 2023 · Synced Oct 2, 2026, 5:39 AM EDT