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ClinicalTrials.gov public records Last synced Oct 23, 2026, 4:12 PM EDT

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Showing 385–408 of 10,802 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Developmental Defect Clear all
Enrolling by invitation No phase listed Observational

North American Fetal Therapy Network Prospective Registry for Long-term Outcome Following Fetoscopic Endoluminal Tracheal Occlusion in Severe Left and Right Congenital Diaphragmatic Hernia

NCT07564297
View directory record Official record
Conditions
Congenital Diaphragmatic Hernia
Interventions
Fetoscopic Endoluminal Tracheal Occlusion, Standard of Care
Device · Other
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2038
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 23, 2026, 4:12 PM EDT
Completed Phase 1 Interventional

Pharmacokinetics of C1 Esterase Inhibitor in Hereditary Angioedema Subjects

NCT00432510
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
C1 esterase inhibitor [human] (C1INH-nf)
Drug
Lead sponsor
Shire
Industry
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
6
States / cities
San Diego, California • Walnut Creek, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2021 · Synced Oct 23, 2026, 4:12 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

PKU Skin Stripping

NCT04368624
View directory record Official record
Conditions
Phenylketonurias
Interventions
Skin Tape, Blood Draw
Other · Diagnostic Test
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
86 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2021
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 28, 2021 · Synced Oct 23, 2026, 4:12 PM EDT
Completed Not applicable Interventional

Building Adherence to Live With And Navigate my Cystic Fibrosis (CF) Experience

NCT01013896
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Motivational Interviewing, Cystic Fibrosis Education
Behavioral · Other
Lead sponsor
Johns Hopkins University
Other
Eligibility
16 Years and older
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 31, 2015 · Synced Oct 23, 2026, 4:12 PM EDT
Recruiting Phase 2 Interventional

Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)

NCT07209462
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Low dose of MRM-3379, Middle Dose of MRM-3379, High dose of MRM-3379, Placebo, Low dose of MRM-3379 Open-Label
Drug
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
13 Years to 45 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
15
States / cities
Phoenix, Arizona • Irvine, California • Orange, California + 12 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 23, 2026, 4:12 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Study On Bioavailability And Pharmacokinetics Of Various Doses Of Testosterone Administered With And Without Dutasteride

NCT00400335
View directory record Official record
Conditions
Hypogonadism, Hypogonadism, Male
Interventions
Nanomilled testosterone, commercially available dutasteride, Nanomilled dutasteride
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years to 50 Years · Male only
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2006
U.S. locations
1
States / cities
Tacoma, Washington
Source: ClinicalTrials.gov public record
Updated Jun 4, 2012 · Synced Oct 23, 2026, 4:12 PM EDT
Completed Phase 1Phase 2 Interventional

Scotoma Reduction in AMD Patients Treated With Ranibizumab

NCT00467935
View directory record Official record
Conditions
Macular Degeneration
Interventions
intravitreal injection Lucentis (ranibizumab)
Drug
Lead sponsor
Retina Research Foundation
Other
Eligibility
50 Years to 90 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007
U.S. locations
1
States / cities
Slingerlands, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2011 · Synced Oct 23, 2026, 4:12 PM EDT
Completed Phase 2 Interventional Results available

A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

NCT03583697
View directory record Official record
Conditions
Achondroplasia
Interventions
BMN 111, Placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 59 Months
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
9
States / cities
Oakland, California • Torrance, California • Wilmington, Delaware + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 13, 2024 · Synced Oct 23, 2026, 4:12 PM EDT
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 23, 2026, 4:12 PM EDT
Terminated No phase listed Observational

A Scan Ultrasonography in the Evaluation of Retinopathy of Prematurity

NCT01335113
View directory record Official record
Conditions
Retinopathy of Prematurity
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Jul 9, 2020 · Synced Oct 23, 2026, 4:12 PM EDT
Active, not recruiting Not applicable Interventional

Early Weight-Bearing After the Lapiplasty Procedure

NCT03740282
View directory record Official record
Conditions
Hallux Valgus, Bunion
Interventions
Lapiplasty
Device
Lead sponsor
Treace Medical Concepts, Inc.
Industry
Eligibility
14 Years to 58 Years
Enrollment
183 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
7
States / cities
Greeley, Colorado • Ankeny, Iowa • Jefferson City, Missouri + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced Oct 23, 2026, 4:12 PM EDT
Completed Phase 3 Interventional Results available

Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year

NCT03293524
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy
Interventions
GS010, Placebo
Genetic · Drug
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
7
States / cities
Pasadena, California • Aurora, Colorado • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 23, 2026, 4:12 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Studies of Disorders in Antibody Production and Related Primary Immunodeficiency States

NCT00266513
View directory record Official record
Conditions
Hyper-IgM Syndrome, Ectodermal Dysplasia
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
119 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 6, 2017 · Synced Oct 23, 2026, 4:12 PM EDT
No Longer Available No phase listed Expanded access

Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

NCT00962260
View directory record Official record
Conditions
Gaucher Disease
Interventions
Plant cell expressed recombinant glucocerebrosidase (prGCD)
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
U.S. locations
14
States / cities
La Jolla, California • Aurora, Colorado • Coral Springs, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 7, 2018 · Synced Oct 23, 2026, 4:12 PM EDT
Terminated No phase listed Observational

Descemet Membrane Endothelial Keratoplasty vs. Descemet's Stripping With Endothelial Keratoplasty vs. Descemet Stripping Only

NCT04527523
View directory record Official record
Conditions
Cystoid Macular Edema, Fuchs Dystrophy
Interventions
Optical Coherence Tomography
Diagnostic Test
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 13, 2023 · Synced Oct 23, 2026, 4:12 PM EDT
Completed Not applicable Interventional Results available

Trial of Doxycycline to Reduce Sputum MMP-9 Activity in Adult Cystic Fibrosis (CF) Patients

NCT01112059
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Doxycycline, placebo
Drug · Other
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
19 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Mar 6, 2017 · Synced Oct 23, 2026, 4:12 PM EDT
Completed Not applicable Interventional

The Safety and Effectiveness of Ganciclovir Used Alone or in Combination With Granulocyte-Macrophage Colony Stimulating Factor in the Treatment of Cytomegalovirus (CMV) of the Eye in Patients With AIDS

NCT00000989
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Zidovudine, Sargramostim, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years to 65 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1992
U.S. locations
4
States / cities
Los Angeles, California • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2021 · Synced Oct 23, 2026, 4:12 PM EDT
Completed No phase listed Observational

The Use of Maternal Serum Unconjugated Estriol for Monitoring the Prevention of Neonatal Respiratory Distress Syndrome

NCT02112513
View directory record Official record
Conditions
Neonatal Respiratory Distress Syndrome
Interventions
Not listed
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older · Female only
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
2
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 28, 2019 · Synced Oct 23, 2026, 4:12 PM EDT
Completed Phase 2 Interventional Results available

A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia

NCT02255435
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg, Placebo, Omaveloxolone Capsules, 20 mg, Omaveloxolone Capsules, 40 mg, Omaveloxolone Capsules, 80 mg, Omaveloxolone Capsules, 160 mg, Omaveloxolone Capsules, 300 mg, Omaveloxolone Capsules, 150 mg
Drug
Lead sponsor
Biogen
Industry
Eligibility
16 Years to 40 Years
Enrollment
172 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2025
U.S. locations
7
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 23, 2026, 4:12 PM EDT
Completed Not applicable Interventional

Resiliency Training for Adults With Neurofibromatosis Via Live Videoconferencing

NCT03406208
View directory record Official record
Conditions
Neurofibromatoses, Neurofibromatosis 1, Neurofibromatosis 2, Schwannomatosis
Interventions
Stress and Symptom Management Program 1, Stress and Symptom Management Program 2
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
228 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 24, 2025 · Synced Oct 23, 2026, 4:12 PM EDT
Active, not recruiting Phase 3 Interventional

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

NCT05881408
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
Male only
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
14
States / cities
Little Rock, Arkansas • Palo Alto, California • Sacramento, California + 11 more
Source: ClinicalTrials.gov public record
Updated May 22, 2026 · Synced Oct 23, 2026, 4:12 PM EDT
Completed Not applicable Interventional Results available

Clinical Study on a New Flowable Composite as a Restorative in Adult Teeth

NCT01369108
View directory record Official record
Conditions
Dental Caries
Interventions
Flowable composite, Conventional composite restorative
Device
Lead sponsor
Solventum US LLC
Industry
Eligibility
19 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Oct 2, 2024 · Synced Oct 23, 2026, 4:12 PM EDT
Completed No phase listed Observational

Neurocognitive and Psychosocial Outcomes in Survivors of Childhood Leukemia With Down Syndrome

NCT02859389
View directory record Official record
Conditions
Leukemia, Down Syndrome
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 18, 2019 · Synced Oct 23, 2026, 4:12 PM EDT
Terminated Phase 2 Interventional

Hospital-Based Program for Treatment of Severe Cardiopulmonary Disease With Inhaled Nitric Oxide

NCT00041574
View directory record Official record
Conditions
Pulmonary Hypertension, Lung Disease, Sickle Cell Disease, Cardiac Transplant, Lung Transplant
Interventions
Inhaled Nitric Oxide
Drug
Lead sponsor
Mallinckrodt
Industry
Eligibility
5 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2005
U.S. locations
3
States / cities
Denver, Colorado • Providence, Rhode Island
Source: ClinicalTrials.gov public record
Updated Sep 9, 2016 · Synced Oct 23, 2026, 4:12 PM EDT