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ClinicalTrials.gov public records Last synced Oct 21, 2026, 7:34 PM EDT

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Showing 169–192 of 10,540 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Disorders Clear all
Recruiting Phase 1 Interventional

Phase 1b Study of VERVE-201 in Patients With Refractory Hyperlipidemia

NCT06451770
View directory record Official record
Conditions
Hypercholesterolemia, Hypertriglyceridemia, Refractory Hyperlipidemia
Interventions
VERVE-201
Drug
Lead sponsor
Verve Therapeutics, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
4
States / cities
Clearwater, Florida • Miami Lakes, Florida • Port Orange, Florida + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 17, 2026 · Synced Oct 21, 2026, 7:34 PM EDT
Terminated Phase 4 Interventional

Assessment of the Risk of Inhibitor Formation in Previously Treated Patients With Severe Hemophilia A

NCT00621673
View directory record Official record
Conditions
Hemophilia A
Interventions
Kogenate (BAY 14-2222)
Drug
Lead sponsor
Bayer
Industry
Eligibility
12 Years to 60 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006
U.S. locations
2
States / cities
Detroit, Michigan • Las Vegas, Nevada
Source: ClinicalTrials.gov public record
Updated Dec 18, 2014 · Synced Oct 21, 2026, 7:34 PM EDT
Completed Phase 3 Interventional

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP

NCT06144840
View directory record Official record
Conditions
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Interventions
Dersimelagon, Placebo
Drug
Lead sponsor
Tanabe Pharma America, Inc.
Industry
Eligibility
12 Years to 75 Years
Enrollment
165 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
13
States / cities
Huntington Beach, California • San Francisco, California • Miami, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 21, 2026, 7:34 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

A Gender and Culturally Specific Approach to Reduce NAFLD in Mexican-American Men

NCT04186260
View directory record Official record
Conditions
NAFLD
Interventions
NAFLD-specific weight loss intervention, Wait-list control
Behavioral · Other
Lead sponsor
University of Arizona
Other
Eligibility
18 Years to 64 Years · Male only
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2025
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Aug 24, 2025 · Synced Oct 21, 2026, 7:34 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Dose Ranging Escalation Study of Tramadol Hydrochloride in Healthy Volunteers

NCT01947920
View directory record Official record
Conditions
Healthy
Interventions
Tramadol HCl, 50 mg, Placebo
Drug
Lead sponsor
Janssen Scientific Affairs, LLC
Industry
Eligibility
18 Years to 45 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Dec 16, 2014 · Synced Oct 21, 2026, 7:34 PM EDT
Completed No phase listed Observational

The Natural History and Pathogenesis of Mucolipidosis Type IV

NCT00015782
View directory record Official record
Conditions
Mucolipidosis Type IV
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 21, 2026, 7:34 PM EDT
Active, not recruiting No phase listed Observational

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

NCT05145062
View directory record Official record
Conditions
Blood and Lymphatic Diseases
Interventions
BIVV003, ST-400
Drug
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2038
U.S. locations
7
States / cities
Oakland, California • Sacramento, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 12, 2025 · Synced Oct 21, 2026, 7:34 PM EDT
Terminated Phase 2 Interventional

A Study of RO5186582 in Down Syndrome Among Children 6 to 11 Years of Age

NCT02484703
View directory record Official record
Conditions
Down Syndrome
Interventions
Placebo, RO5186582
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
6 Years to 11 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
9
States / cities
Phoenix, Arizona • Decatur, Georgia • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2017 · Synced Oct 21, 2026, 7:34 PM EDT
Terminated Phase 2Phase 3 Interventional Results available

Fenoldopam to Prevent Renal Dysfunction in Indomethacin Treated Preterm Infants

NCT02620761
View directory record Official record
Conditions
Patent Ductus Arteriosus (PDA), Acute Kidney Injury (AKI)
Interventions
Fenoldopam, 0.9%NS
Drug
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
0 Days to 28 Days
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Sep 16, 2022 · Synced Oct 21, 2026, 7:34 PM EDT
Completed Not applicable Interventional

Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.

NCT02654886
View directory record Official record
Conditions
Pompe Disease, Muscle Weakness
Interventions
Resistance Exercise Training
Other
Lead sponsor
University of California, Irvine
Other
Eligibility
16 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Sep 26, 2019 · Synced Oct 21, 2026, 7:34 PM 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 21, 2026, 7:34 PM EDT
Completed Phase 2Phase 3 Interventional

Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

NCT00125879
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
6
States / cities
Birmingham, Alabama • Gainesville, Florida • Miami, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2014 · Synced Oct 21, 2026, 7:34 PM EDT
Enrolling by invitation Phase 2 Interventional

Long-term Safety and Efficacy Evaluation of IMG-007 in Participants With Moderate-to-Severe Atopic Dermatitis

NCT07734415
View directory record Official record
Conditions
Atopic Dermatitis, Atopic Dermatitis Eczema, Eczema
Interventions
IMG-007
Drug
Lead sponsor
Inmagene LLC
Industry
Eligibility
18 Years to 75 Years
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
16
States / cities
Lancaster, California • Los Angeles, California • Ocala, Florida + 13 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 21, 2026, 7:34 PM EDT
Completed Phase 2 Interventional Results available

Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency

NCT03008915
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency, Emphysema, Chronic Obstructive Pulmonary Disease
Interventions
Aspirin, Placebo, Withdrawal from alpha1 antitrypsin replacement therapy
Drug · Other
Lead sponsor
Columbia University
Other
Eligibility
40 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 17, 2021 · Synced Oct 21, 2026, 7:34 PM EDT
Completed Phase 2 Interventional

Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis

NCT00578435
View directory record Official record
Conditions
Genetic Disorders, Sickle Cell Anemia
Interventions
Busulfan, Cyclophosphamide, BMD
Procedure
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
1 Year and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1994 – 2008
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 12, 2008 · Synced Oct 21, 2026, 7:34 PM EDT
Completed No phase listed Observational

MRI QSM Imaging for Iron Overload

NCT04631718
View directory record Official record
Conditions
Hemochromatosis, Iron Overload
Interventions
Magnetic Resonance Imaging (MRI)-based Quantitative Susceptibility Mapping (QSM)
Radiation
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
5 Years and older
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
2
States / cities
Stanford, California • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated May 9, 2024 · Synced Oct 21, 2026, 7:34 PM EDT
Completed Phase 2 Interventional Results available

Study to Determine the Safety and Effectiveness of Dupilumab for Treatment of Atopic Dermatitis (AD)

NCT01979016
View directory record Official record
Conditions
Atopic Dermatitis (AD)
Interventions
Dupilumab, Placebo, Background treatment
Drug · Other
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
3
States / cities
Chicago, Illinois • New York, New York • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 18, 2020 · Synced Oct 21, 2026, 7:34 PM EDT
Completed Phase 4 Interventional Results available

A Study to Evaluate the Mode of Administration and Safety of EUR-1008 (APT-1008) in Infants 1 to 12 Months of Age

NCT01100606
View directory record Official record
Conditions
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Interventions
EUR-1008 (APT-1008)
Drug
Lead sponsor
Forest Laboratories
Industry
Eligibility
1 Month to 12 Months
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010
U.S. locations
6
States / cities
Jacksonville, Florida • Las Vegas, Nevada • Akron, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 10, 2014 · Synced Oct 21, 2026, 7:34 PM EDT
Completed Phase 2 Interventional

Metabolic Mapping to Measure Retinal Metabolism

NCT00385333
View directory record Official record
Conditions
Macular Degeneration, Diabetic Retinopathy, Hippel-Landau Disease, Mitochondria
Interventions
Feasibility Study - Imaging System
Procedure
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 21, 2026, 7:34 PM EDT
Active, not recruiting Phase 2 Interventional

Study of UC-MSC in DMD Patients

NCT06579352
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC)
Biological
Lead sponsor
MED Institute Inc.
Industry
Eligibility
5 Years to 10 Years · Male only
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
2
States / cities
Dallas, Texas • Flower Mound, Texas
Source: ClinicalTrials.gov public record
Updated Aug 14, 2026 · Synced Oct 21, 2026, 7:34 PM EDT
Terminated Phase 2 Interventional Results available

Study to Evaluate Long-Term Safety of ASN002 in Subjects With Moderate to Severe Atopic Dermatitis

NCT03654755
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
ASN002
Drug
Lead sponsor
Asana BioSciences
Industry
Eligibility
18 Years to 75 Years
Enrollment
162 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
31
States / cities
Anniston, Alabama • Birmingham, Alabama • Fremont, California + 25 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2023 · Synced Oct 21, 2026, 7:34 PM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

The Measurement of Intraocular Pressure in Normal Children Under General Anesthesia With and Without Nitrous Oxide and Anesthetic Eye Drops

NCT01925391
View directory record Official record
Conditions
Glaucoma, Intraocular Pressure, Anesthesia
Interventions
Tono-pen XL Applanation tonometer + Ocu-film +
Drug
Lead sponsor
University of Miami
Other
Eligibility
1 Year to 18 Years
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2014
U.S. locations
2
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Jun 16, 2014 · Synced Oct 21, 2026, 7:34 PM EDT
Recruiting Phase 2 Interventional

Metyrapone Study for Patients Diagnosed With Mild Autonomous Cortisol Secretion - MACS

NCT07138274
View directory record Official record
Conditions
Mild Autonomous Cortisol Secretion (MACS), Autonomous Cortisol Secretion (ACS)
Interventions
Metyrapone 250 mg Oral Tablets, Placebo
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 25, 2026 · Synced Oct 21, 2026, 7:34 PM EDT
Completed Not applicable Interventional

Ensuring Patients' Informed Access to Noninvasive Prenatal Testing

NCT03420274
View directory record Official record
Conditions
Genetic Disease, Genetic Syndrome, Prenatal Disorder, Prenatal Maternal Abnormality, Pregnancy Complications
Interventions
NEST, Usual care
Behavioral
Lead sponsor
The Cleveland Clinic
Other
Eligibility
18 Years and older
Enrollment
691 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
2
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 21, 2026, 7:34 PM EDT