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ClinicalTrials.gov public records Last synced Nov 8, 2026, 10:20 AM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 3073–3096 of 10,825 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Developmental Defect Clear all

Conditions: Coarctation of Aorta
Interventions: Pressure-volume loop catheter; Diagnostic Test
Lead sponsor: University of Minnesota; Other
Eligibility: 6 Years and older
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 7
States / cities: Los Angeles, California • Boston, Massachusetts • Grand Rapids, Michigan + 4 more

Conditions: Congenital Disorders
Interventions: Not listed
Lead sponsor: Children's Healthcare of Atlanta; Other
Eligibility: Up to 21 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: X-Linked Combined Immunodeficiency Diseases
Interventions: CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT, Palifermin, Busulfan; Biological · Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 2 Years to 40 Years
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2022
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Cystic Fibrosis
Interventions: Lumacaftor, Ivacaftor; Drug
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 6 Years to 11 Years
Enrollment: 62 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 19
States / cities: Birmingham, Alabama • Tucson, Arizona • Long Beach, California + 16 more

Conditions: Netherton Syndrome
Interventions: QRX003-2% Lotion, QRX003-4% Lotion QAM, Vehicle, QRX003-4% Lotion BID; Drug
Lead sponsor: Quoin Pharmaceuticals; Industry
Eligibility: 14 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 5
States / cities: San Diego, California • Indianapolis, Indiana • Quincy, Massachusetts + 2 more

Conditions: Lactose Intolerance
Interventions: Milk containing A1 and A2 beta-casein, Milk containing only A2 beta-casein; Other
Lead sponsor: Purdue University; Other
Eligibility: 18 Years to 65 Years
Enrollment: 35 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2022 – 2025
U.S. locations: 1
States / cities: West Lafayette, Indiana

Conditions: Primary Ciliary Dyskinesia
Interventions: VX-371, Hypertonic Saline, Placebo (0.17% saline), VX-371 + HS, Ivacaftor; Drug
Lead sponsor: Parion Sciences; Industry
Eligibility: 12 Years and older
Enrollment: 123 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2018
U.S. locations: 21
States / cities: Birmingham, Alabama • Palo Alto, California • Aurora, Colorado + 18 more

Conditions: Hypothalamic Obesity
Interventions: Bivamelagon, Placebo; Drug
Lead sponsor: Rhythm Pharmaceuticals, Inc.; Industry
Eligibility: 12 Years and older
Enrollment: 28 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 7
States / cities: Aurora, Colorado • Iowa City, Iowa • Boston, Massachusetts + 4 more

Conditions: Hemophilia A
Interventions: Moroctocog alfa(AF-CC), Blood draw for laboratory testing; Biological · Procedure
Lead sponsor: Pfizer; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2011
U.S. locations: 5
States / cities: Los Angeles, California • Washington D.C., District of Columbia • Detroit, Michigan + 2 more

Conditions: Stargardt Disease
Interventions: Gene Therapy Product-MCO-010; Biological
Lead sponsor: Nanoscope Therapeutics Inc.; Industry
Eligibility: 16 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 2
States / cities: Miami, Florida • McAllen, Texas

Conditions: Tyrosinemia I
Interventions: NTBC; Drug
Lead sponsor: University of Washington; Other
Eligibility: Up to 17 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1998 – 2006
U.S. locations: 2
States / cities: Portland, Oregon • Seattle, Washington

Conditions: Factor X Deficiency
Interventions: FACTOR X; Biological
Lead sponsor: Bio Products Laboratory; Other
Eligibility: 12 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2014
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Williams Syndrome, Supravalvular Aortic Stenosis, Cardiovascular Disease
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 3 Years to 85 Years
Enrollment: 159 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2024
U.S. locations: 2
States / cities: Bethesda, Maryland • St Louis, Missouri

Conditions: Primary Immune Deficiency Disorder, Immune Deficiency Disease, Bone Marrow Failure, Short Telomere Length, Fanconi Anemia, Non Fanconi DNA-DSB Repair Disorder, Hoyeraal-Hreidarsson Syndrome, Dyskeratosis Congenita, Telomere Biology Disorder, Short Telomere Syndrome
Interventions: Alemtuzumab, Fludarabine, Melphalan, Low Dose Total Body Irradiation, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil; Drug · Radiation
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins; Other
Eligibility: 4 Months to 50 Years
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2028
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Retinopathy of Prematurity (ROP)
Interventions: rhIGF-I/rhIGFBP-3; Drug
Lead sponsor: Shire; Industry
Eligibility: Up to 1 Day
Enrollment: 121 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2016
U.S. locations: 8
States / cities: Mobile, Alabama • Irvine, California • Augusta, Georgia + 5 more

Conditions: Pancreatic Cancer, ATM, BRCA, Hereditary Cancer
Interventions: Not listed
Lead sponsor: Johns Hopkins University; Other
Eligibility: 18 Years and older
Enrollment: 12,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 1994 – 2032
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Neurofibromatosis Type 1, Plexiform Neurofibromas
Interventions: Not listed
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 2 Years to 18 Years
Enrollment: 37 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2024
U.S. locations: 10
States / cities: Phoenix, Arizona • Los Angeles, California • Miami, Florida + 7 more

Conditions: Mucopolysaccharidosis I
Interventions: JR-171; Drug
Lead sponsor: JCR Pharmaceuticals Co., Ltd.; Industry
Eligibility: Not listed
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 1
States / cities: Oakland, California

Conditions: Cryopyrin-Associated Periodic Syndrome
Interventions: ATI-450; Drug
Lead sponsor: Aclaris Therapeutics, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 2
States / cities: San Diego, California • San Francisco, California

Conditions: Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Interventions: 14C-VX-407; Drug
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 19 Years to 55 Years · Male only
Enrollment: 8 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026
U.S. locations: 1
States / cities: Lincoln, Nebraska

Conditions: Neurofibromatosis, Neurofibromatosis I, Neurofibromatosis 2, Schwannomatosis
Interventions: The Relaxation Response Resiliency Program (3RP) via Skype; Behavioral
Lead sponsor: Massachusetts General Hospital; Other
Eligibility: 18 Years and older
Enrollment: 65 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Cardiovascular Diseases, Metabolic Disease, Peripheral Artery Disease, Carotid Disease, Aneurysmal Disease, Venous Disease, Thrombosis, Lymphedema, Lipedema, Non-Atherosclerotic Chronic Arterial Occlusive Disease, Vasculitis, Fibromuscular Dysplasia, Arterial Dissection, May-Thurner Syndrome, Thoracic Outlet Syndrome, Vasospasm
Interventions: Not listed
Lead sponsor: The Cleveland Clinic; Other
Eligibility: 18 Years and older
Enrollment: 500 participants
Timeline: 2019 – 2031
U.S. locations: 1
States / cities: Cleveland, Ohio

Conditions: Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
Interventions: Not listed
Lead sponsor: UCSF Benioff Children's Hospital Oakland; Other
Eligibility: 16 Years and older
Enrollment: 423 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 6
States / cities: Oakland, California • Augusta, Georgia • Chicago, Illinois + 3 more

Conditions: COPD, Asthma, Cystic Fibrosis, Emphysema, Small Airways Disease
Interventions: Medical Grade Oxygen; Drug
Lead sponsor: Hal C Charles; Other
Eligibility: 18 Years to 90 Years
Enrollment: 15 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2012
U.S. locations: 1
States / cities: Durham, North Carolina

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PV Loop & Coarctation Study

Pulmonary Resistance in Fontan Who Have Undergone Heart Transplantation

Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency

Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome

Comparing the Inflammation, Maldigestion and Symptoms Due to Commercial Milk and A2 Milk

Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia

A Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic Obesity

Study Evaluating The Safety Of Xyntha In Usual Care Settings

Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study

Study of NTBC for Tyrosinemia I

Safety & Efficacy of BPL's High Purity FACTOR X in Treatment of Factor X Deficient Subjects Undergoing Surgery

Impact of Elastin Mediated Vascular Stiffness on End Organs

Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

IGF-1/IGFBP3 Prevention of Retinopathy of Prematurity

Pancreatic Cancer Registry: For Any Person With a Personal or Family History

US Selumetinib Registry

An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)

Study of ATI-450 in Patients With Cryopyrin-Associated Periodic Syndrome (CAPS)

Absorption, Distribution, Metabolism, and Excretion of 14C-VX-407 After a Single Oral Dose

Resiliency Training for Patients With Neurofibromatosis Via Videoconferencing With Skype

Vascular Lab Resource (VLR) Biorepository

Multi-Center Study of Iron Overload: Survey Study (MCSIO)

Imaging Lung Function Using Oxygen Enhanced MRI