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ClinicalTrials.gov public records Last synced Sep 9, 2026, 7:24 AM EDT

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Showing 1–24 of 10,426 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Congenital Deformity Clear all
Recruiting No phase listed Observational

Characteristics of Patients With Recessive Dystrophic Epidermolysis Bullosa

NCT01019148
View directory record Official record
Conditions
Epidermolysis Bullosa Dystrophica
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2029
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 3 Interventional

A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita

NCT05180708
View directory record Official record
Conditions
Pachyonychia Congenita
Interventions
QTORIN 3.9% rapamycin anhydrous gel, Vehicle
Drug
Lead sponsor
Palvella Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
7
States / cities
Palo Alto, California • New Haven, Connecticut • Orange Park, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2024 · Synced Sep 9, 2026, 7:24 AM EDT
Recruiting No phase listed Observational

Molecular and Cellular Characterization of Cardiac Tissue in Postnatal Development

NCT00243776
View directory record Official record
Conditions
Congenital Heart Disease, Tetralogy of Fallot
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
Up to 20 Years
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2027
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Sep 9, 2026, 7:24 AM EDT
Recruiting No phase listed Observational

Study of Chediak-Higashi Syndrome

NCT00005917
View directory record Official record
Conditions
Chediak-Higashi Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 70 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 2Phase 3 Interventional Results available

A Long Term Safety Study of BCX7353 in Hereditary Angioedema

NCT03472040
View directory record Official record
Conditions
Hereditary Angioedema, HAE, Prophylaxis
Interventions
BCX7353
Drug
Lead sponsor
BioCryst Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
387 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
42
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Bentonville, Arkansas + 39 more
Source: ClinicalTrials.gov public record
Updated Jun 17, 2023 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 2 Interventional Results available

Stem Cell Transplant w/Laronidase for Hurler

NCT00176891
View directory record Official record
Conditions
Mucopolysaccharidosis I, Hurler Syndrome
Interventions
Stem Cell Transplant, Laronidase ERT
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 7 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2012
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 10, 2019 · Synced Sep 9, 2026, 7:24 AM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Ovarian Contribution to Androgen Production in Adolescent Girls

NCT01421810
View directory record Official record
Conditions
Polycystic Ovary Syndrome, Obesity, Hyperandrogenism
Interventions
Dexamethasone, rhCG
Drug
Lead sponsor
University of Virginia
Other
Eligibility
7 Years to 18 Years · Female only
Enrollment
80 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2025
U.S. locations
1
States / cities
Charlottesville, Virginia
Source: ClinicalTrials.gov public record
Updated Oct 26, 2023 · Synced Sep 9, 2026, 7:24 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Pathogenesis of Primary Ciliary Dyskinesia (PCD) Lung Disease

NCT00807482
View directory record Official record
Conditions
Kartagener Syndrome
Interventions
Not listed
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
Not listed
Enrollment
1,800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2027
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 28, 2026 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 2 Interventional

Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia

NCT00029380
View directory record Official record
Conditions
Hematologic Diseases, Anemia, Sickle Cell, Beta-Thalassemia, Hematopoietic Stem Cell Transplantation
Interventions
Sangstat, Cyclophosphamide, Busulfan, Mycophenolate Mofetil, Cyclosporine, Cord Blood Transplantation
Drug · Procedure
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
3 Years to 14 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2006
U.S. locations
14
States / cities
Oakland, California • Washington D.C., District of Columbia • Jacksonville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2016 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 2 Interventional Results available

Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia

NCT03214588
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
TAK-831, TAK-831 Placebo
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
18 Years to 55 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
6
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 13, 2021 · Synced Sep 9, 2026, 7:24 AM EDT
Completed No phase listed Observational

Clinical Biomarkers in Alpha-mannosidosis

NCT02141503
View directory record Official record
Conditions
Alpha-Mannosidosis
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
5 Years to 60 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 2, 2019 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 2 Interventional Results available

A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease

NCT00445978
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Sapropterin Dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
15 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
12
States / cities
Washington D.C., District of Columbia • Augusta, Georgia • Savannah, Georgia + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 24, 2021 · Synced Sep 9, 2026, 7:24 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Characterization of the Pathogenesis of Primary and Secondary Lymphatic Disorders

NCT02156115
View directory record Official record
Conditions
Lymphangiomatosis, Lymphedema, Lymphangiectasia, Pulmonary Lymphangiectasia
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 90 Years
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2024
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 10, 2024 · Synced Sep 9, 2026, 7:24 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia

NCT03914625
View directory record Official record
Conditions
B Acute Lymphoblastic Leukemia, B Lymphoblastic Lymphoma, Down Syndrome
Interventions
Asparaginase Erwinia chrysanthemi, Blinatumomab, Cyclophosphamide, Cytarabine, Dexamethasone, Doxorubicin Hydrochloride, Leucovorin Calcium, Mercaptopurine, Mercaptopurine Oral Suspension, Methotrexate, Pegaspargase, Prednisolone, Prednisone, Radiation Therapy, Thioguanine, Vincristine Sulfate
Drug · Biological · Radiation
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
365 Days to 31 Years
Enrollment
6,720 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
201
States / cities
Birmingham, Alabama • Mobile, Alabama • Anchorage, Alaska + 153 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Sep 9, 2026, 7:24 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

TCA Cycle in the Dentate in Friedreich's Ataxia

NCT03122925
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
MR
Device
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2023
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 26, 2023 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 3 Interventional Results available

A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia

NCT01179217
View directory record Official record
Conditions
Sickle Cell Anemia, Sickle ß0-Thalassemia
Interventions
L-glutamine, Placebo
Drug
Lead sponsor
Emmaus Medical, Inc.
Industry
Eligibility
5 Years and older
Enrollment
230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
31
States / cities
Mobile, Alabama • Phoenix, Arizona • Inglewood, California + 23 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2020 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 3 Interventional Results available

Cyclosporine Eye Drops in Preventing Graft-Versus-Host Disease of the Eye in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer or Bone Marrow Failure Disorder

NCT00755040
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
cyclosporine ophthalmic emulsion, placebo
Drug · Other
Lead sponsor
Vanderbilt-Ingram Cancer Center
Other
Eligibility
18 Years and older
Enrollment
164 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2016
U.S. locations
5
States / cities
Palo Alto, California • Chicago, Illinois • Nashville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2017 · Synced Sep 9, 2026, 7:24 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

The Influence of Different Mood States and Emotions on the Physiologic, Metabolic, and Perceptual Responses to Feeding Before Exercise

NCT05217589
View directory record Official record
Conditions
Other Gastrointestinal Disorders, Cramp, Abdominal
Interventions
Funny/Amusing Video Clips, Horror/Scary Video Clips, Thrilling/Suspenseful Video Clips
Other
Lead sponsor
Old Dominion University
Other
Eligibility
18 Years to 55 Years
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Mar 19, 2025 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 2 Interventional Results available

Preliminary Efficacy, Safety and Pharmacokinetics Study of Nepadutant in Infant With Feeding Intolerance

NCT01532518
View directory record Official record
Conditions
Colic
Interventions
Nepadutant
Drug
Lead sponsor
Menarini Group
Industry
Eligibility
Up to 6 Months
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
5
States / cities
Little Rock, Arkansas • Atlanta, Georgia • Louisville, Kentucky + 2 more
Source: ClinicalTrials.gov public record
Updated May 5, 2023 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Not applicable Interventional

Ganciclovir: Compassionate Use in Patients With Serious or Life-Threatening Cytomegalovirus Infections

NCT00002024
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 9, 2026, 7:24 AM EDT
Recruiting Not applicable Interventional

Balloon Inflation Time for Esophageal Strictures (BITES): A Randomized Multi-Center Study

NCT07100379
View directory record Official record
Conditions
Esophageal Atresia With Tracheo-esophageal Fistula, Esophageal Atresia, Esophageal Strictures
Interventions
Endoscopic Balloon Dilation
Procedure
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Up to 18 Years
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
2
States / cities
Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 29, 2026 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Not applicable Interventional

Effects of a Partially Supervised Conditioning Program in CF

NCT01744561
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Exercise Intervention
Behavioral
Lead sponsor
Wuerzburg University Hospital
Other
Eligibility
12 Years and older
Enrollment
155 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
2
States / cities
Birmingham, Alabama • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 21, 2018 · Synced Sep 9, 2026, 7:24 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Generating Advancements Through Longitudinal Analysis in X and Y Variations (GALAXY)

NCT06373861
View directory record Official record
Conditions
Sex Chromosome Aneuploidy
Interventions
no intervention
Other
Lead sponsor
University of Colorado, Denver
Other
Eligibility
0 Days to 100 Years
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2037
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Apr 17, 2024 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 4 Interventional Results available

A Study Evaluating the Efficacy of Fluocinonide Cream 0.1% (Vanos) in Subjects With Atopic Dermatitis

NCT01049243
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Fluocinonide Cream 0.1%
Drug
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
12 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 31, 2018 · Synced Sep 9, 2026, 7:24 AM EDT