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ClinicalTrials.gov public records Last synced Oct 30, 2026, 4:46 PM EDT

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

Showing 769–792 of 2,426 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolic Abnormalities Clear all
Completed Phase 3 Interventional Accepts healthy volunteers Results available

Safety of Inhaled Insulin With Type 1 and Type 2 Diabetes

NCT00308737
View directory record Official record
Conditions
Diabetes, Type 1, Diabetes, Type 2
Interventions
Technosphere® Insulin Inhalation Powder, Usual Care
Drug
Lead sponsor
Mannkind Corporation
Industry
Eligibility
18 Years to 70 Years
Enrollment
2,053 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2008
U.S. locations
82
States / cities
Phoenix, Arizona • Tempe, Arizona • Tucson, Arizona + 64 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2014 · Synced Oct 30, 2026, 4:46 PM EDT
Completed No phase listed Observational

Visual Function and Ocular Pigmentation in Albinism

NCT00001153
View directory record Official record
Conditions
Albinism, Albinism, Ocular, Albinism, Oculocutaneous
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1976 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 30, 2026, 4:46 PM EDT
Recruiting Phase 1Phase 2 Interventional

DFT383 in Pediatric Participants With Nephropathic Cystinosis

NCT06910813
View directory record Official record
Conditions
Nephropathic Cystinosis
Interventions
DFT383
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 5 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2044
U.S. locations
6
States / cities
Phoenix, Arizona • San Diego, California • Stanford, California + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 30, 2026, 4:46 PM EDT
Completed No phase listed Observational

Cardiac Function in Patients With Hereditary Hemochromatosis

NCT00068159
View directory record Official record
Conditions
Hereditary Hemochromatosis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
21 Years to 100 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 3, 2024 · Synced Oct 30, 2026, 4:46 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

AAV Gene Therapy Study for Subjects with PKU

NCT04480567
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
BMN 307
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
15 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
2
States / cities
Tampa, Florida • Morristown, New Jersey
Source: ClinicalTrials.gov public record
Updated Dec 12, 2024 · Synced Oct 30, 2026, 4:46 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1

NCT04152200
View directory record Official record
Conditions
Primary Hyperoxaluria Type 1, Primary Hyperoxaluria
Interventions
Lumasiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
2
States / cities
Rochester, Minnesota • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 18, 2025 · Synced Oct 30, 2026, 4:46 PM EDT
Recruiting No phase listed Observational

Cardiovascular Multimodality Imaging Study

NCT06610019
View directory record Official record
Conditions
Non-ischemic Cardiomyopathy, Cardiomyopathies, Hypertrophic Cardiomyopathy, Right Ventricular Arrhythmogenic Cardiomyopathy, Cardiac Amyloidosis, Anderson Fabry Disease, Sarcoidosis, Cancer Therapy-related Cardiac Dysfunction, Ventricular Arrythmia, Heart Failure
Interventions
Not listed
Lead sponsor
Montefiore Medical Center
Other
Eligibility
18 Years and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Oct 30, 2026, 4:46 PM EDT
Completed Phase 1 Interventional

Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

NCT00243399
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Oxandrolone
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 15, 2011 · Synced Oct 30, 2026, 4:46 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Early Detection of Neuropathy in ATTRv

NCT05311488
View directory record Official record
Conditions
Hereditary Amyloidosis, Transthyretin-Related
Interventions
neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
Diagnostic Test
Lead sponsor
University of Pennsylvania
Other
Eligibility
30 Years to 90 Years
Enrollment
47 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Oct 30, 2026, 4:46 PM EDT
Active, not recruiting No phase listed Observational

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

NCT03694561
View directory record Official record
Conditions
Pompe Disease, Pompe Disease (Late-onset), GAA Deficiency
Interventions
Observational
Other
Lead sponsor
Duke University
Other
Eligibility
3 Months to 54 Months
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 30, 2026, 4:46 PM EDT
Recruiting Phase 2Phase 3 Interventional

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

NCT02716246
View directory record Official record
Conditions
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Interventions
UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy
Biological · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2029
U.S. locations
2
States / cities
Columbus, Ohio • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 30, 2026, 4:46 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)

NCT07748403
View directory record Official record
Conditions
Wilson Disease, Wilson's Disease, Wilsons Disease
Interventions
PM577a
Drug
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
12 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
New Haven, Connecticut • Chicago, Illinois • San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 30, 2026, 4:46 PM EDT
Not yet recruiting No phase listed Observational

Natural History to Assess Disease in Patients With MPS IIIC

NCT07712003
View directory record Official record
Conditions
Sanfilippo Syndrome Type C, Mucopolysaccharidosis (MPS) IIIC
Interventions
Not listed
Lead sponsor
Phoenix Nest
Industry
Eligibility
1 Year to 25 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 17, 2026 · Synced Oct 30, 2026, 4:46 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Li-Fraumeni Syndrome/TP53 Biobank

NCT04367246
View directory record Official record
Conditions
Li-Fraumeni Syndrome, Li-Fraumeni-Like Syndrome
Interventions
No Intervention
Other
Lead sponsor
Abramson Cancer Center at Penn Medicine
Other
Eligibility
Not listed
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2029
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Phildelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 4, 2026 · Synced Oct 30, 2026, 4:46 PM EDT
Completed Phase 3 Interventional Results available

Re-exposure Study of Pegloticase Intravenous (i.v.) in Symptomatic Gout Patients

NCT00675103
View directory record Official record
Conditions
Chronic Gout Refractory to Conventional Therapy
Interventions
pegloticase 8 mg i.v.
Drug
Lead sponsor
Savient Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
4
States / cities
Chicago, Illinois • Wheaton, Maryland • Durham, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 28, 2011 · Synced Oct 30, 2026, 4:46 PM EDT
Completed Phase 1Phase 2 Interventional Results available

NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome

NCT04977388
View directory record Official record
Conditions
Menkes Disease, Occipital Horn Syndrome
Interventions
Droxidopa, Placebo
Drug · Other
Lead sponsor
Stephen G. Kaler, MD
Other
Eligibility
18 Years to 50 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 30, 2026, 4:46 PM EDT
Completed Phase 3 Interventional Results available

Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia

NCT02415127
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Interferon γ-1b, Placebo
Drug
Lead sponsor
Amgen
Industry
Eligibility
10 Years to 25 Years
Enrollment
92 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
4
States / cities
Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 19, 2024 · Synced Oct 30, 2026, 4:46 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease

NCT02725580
View directory record Official record
Conditions
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
Interventions
AT-GTX-501
Genetic
Lead sponsor
Emily de los Reyes
Other
Eligibility
1 Year and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 24, 2025 · Synced Oct 30, 2026, 4:46 PM EDT
Withdrawn Phase 2 Interventional

Sirolimus Prophylaxis for aGVHD in TME SCID

NCT02177760
View directory record Official record
Conditions
Severe Combined Immunodeficiency, Transplacental Maternal Engraftment, Stem Cell Transplant
Interventions
Sirolimus
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
1 Day to 1 Year
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Dec 3, 2015 · Synced Oct 30, 2026, 4:46 PM EDT
Completed Phase 4 Interventional Results available

An Assessment of Chronic Synovial-Based Inflammation and Its Role With Serum Urate Levels.

NCT01112982
View directory record Official record
Conditions
Gout
Interventions
Magnetic Resonance Imaging, Febuxostat, Colchicine
Other · Drug
Lead sponsor
University of South Florida
Other
Eligibility
18 Years and older
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Aug 7, 2019 · Synced Oct 30, 2026, 4:46 PM EDT
Completed Phase 4 Interventional

Replagal Enzyme Replacement Therapy for Adults With Fabry Disease

NCT00097890
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal (Agalsidase Alfa), Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 30, 2026, 4:46 PM EDT
Terminated Phase 3 Interventional

Carotid B-mode Ultrasound Study to Compare Anti-Atherosclerotic Effect of Torcetrapib/Atorvastatin to Atorvastatin.

NCT00134238
View directory record Official record
Conditions
Mixed Hyperlipidemia
Interventions
torcetrapib/atorvastatin, atorvastatin
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 70 Years
Enrollment
755 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
40
States / cities
Gilbert, Arizona • Phoenix, Arizona • Scottsdale, Arizona + 37 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2012 · Synced Oct 30, 2026, 4:46 PM EDT
Completed Phase 3 Interventional Results available

A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

NCT05218655
View directory record Official record
Conditions
Inherited Mitochondrial Disease
Interventions
Vatiquinone
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
101 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
14
States / cities
La Jolla, California • Stanford, California • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 22, 2025 · Synced Oct 30, 2026, 4:46 PM EDT
Completed No phase listed Observational

Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation

NCT01316549
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Fludarabine
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 4, 2021 · Synced Oct 30, 2026, 4:46 PM EDT