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ClinicalTrials.gov public records Last synced Nov 3, 2026, 5:08 AM EST

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Showing 1033–1056 of 10,349 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital, Hereditary, and Neonatal Diseases and Abnormalities Clear all
Completed No phase listed Observational

Down Syndrome Biomarker Initiative (DSBI)

NCT02141971
View directory record Official record
Conditions
Down Syndrome, Alzheimer's Disease
Interventions
Not listed
Lead sponsor
Michael Rafii
Other
Eligibility
30 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated May 2, 2017 · Synced Nov 3, 2026, 5:08 AM EST
Recruiting Phase 2Phase 3 Interventional

Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease

NCT07378644
View directory record Official record
Conditions
Huntington Disease
Interventions
SKY-0515, SKY-0515 Placebo
Drug
Lead sponsor
Skyhawk Therapeutics, Inc.
Industry
Eligibility
25 Years and older
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
4
States / cities
Englewood, Colorado • Boston, Massachusetts • Charlestown, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 29, 2026 · Synced Nov 3, 2026, 5:08 AM EST
Recruiting Phase 1 Interventional

Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

NCT05302271
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
Interventions
AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
Biological · Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
12 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Nov 3, 2026, 5:08 AM EST
Completed No phase listed Observational

Biliary Atresia Research Network Northeast

NCT06184971
View directory record Official record
Conditions
Biliary Atresia
Interventions
Not listed
Lead sponsor
Yale University
Other
Eligibility
Up to 12 Years
Enrollment
320 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
14
States / cities
Hartford, Connecticut • New Haven, Connecticut • Bangor, Maine + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2026 · Synced Nov 3, 2026, 5:08 AM EST
Completed Phase 1Phase 2 Interventional

RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM

NCT00457964
View directory record Official record
Conditions
Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions
RAD001
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 25, 2013 · Synced Nov 3, 2026, 5:08 AM EST
Completed Phase 2 Interventional Results available

Efficacy and Safety of Extended Release and Immediate Release Febuxostat in Participants With Gout and Moderate Renal Impairment

NCT02128490
View directory record Official record
Conditions
Gout, Moderate Renal Impairment
Interventions
Febuxostat IR, Febuxostat XR, Febuxostat placebo, Colchicine, Naproxen, Lansoprazole
Drug
Lead sponsor
Takeda
Industry
Eligibility
18 Years and older
Enrollment
189 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
89
States / cities
Birmingham, Alabama • Muscle Shoals, Alabama • Phoenix, Arizona + 75 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2016 · Synced Nov 3, 2026, 5:08 AM EST
Completed Phase 1 Interventional

Motexafin Gadolinium and Doxorubicin in Treating Patients With Advanced Cancer

NCT00036790
View directory record Official record
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Colorectal Cancer, Head and Neck Cancer, Leukemia, Lung Cancer, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic/Myeloproliferative Diseases, Prostate Cancer, Small Intestine Cancer, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
doxorubicin hydrochloride, motexafin gadolinium
Drug
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years to 120 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
2
States / cities
Pittsburgh, Pennsylvania • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Nov 3, 2026, 5:08 AM EST
Terminated Phase 1 Interventional

Monoclonal Antibody Therapy in Treating Patients With Ovarian Epithelial Cancer, Melanoma, Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Non-Small Cell Lung Cancer

NCT00039091
View directory record Official record
Conditions
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Recurrent Melanoma, Recurrent Non-small Cell Lung Cancer, Recurrent Ovarian Epithelial Cancer, Stage IV Melanoma, Stage IV Non-small Cell Lung Cancer
Interventions
ipilimumab
Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
19 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 5, 2018 · Synced Nov 3, 2026, 5:08 AM EST
Completed No phase listed Observational

A Chart Review of Newborns With the Presence of Confirmed or Suspected Maternal Chorioamnionitis and Utilization of the Early Onset Sepsis Calculator

NCT04513691
View directory record Official record
Conditions
Maternal; Chorioamnionitis, Affecting Fetus, Early-Onset Sepses, Neonatal
Interventions
EOS Calculator
Diagnostic Test
Lead sponsor
Pediatrix
Other
Eligibility
35 Weeks and older
Enrollment
236 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 22, 2022 · Synced Nov 3, 2026, 5:08 AM EST
Not listed Not applicable Interventional

A Clinical Study to Evaluate the Safety and Efficacy of T92 in Pediatric Patients With Tourette Syndrome

NCT05188274
View directory record Official record
Conditions
Tourette Syndrome in Children, Tourette Syndrome in Adolescence
Interventions
T92, Placebo
Dietary Supplement
Lead sponsor
Tasly Pharmaceuticals, Inc.
Industry
Eligibility
6 Years to 17 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jan 12, 2022 · Synced Nov 3, 2026, 5:08 AM EST
Recruiting No phase listed Observational Accepts healthy volunteers

Genetic Analysis of Pheochromocytomas, Paragangliomas and Associated Conditions

NCT03160274
View directory record Official record
Conditions
Pheochromocytoma, Paraganglioma, Inherited Cancer Syndrome, Associated Conditions, Kidney Neoplasms, Bone Cancer, Thyroid Neoplasms, Other Cancer
Interventions
Genetic screening
Genetic
Lead sponsor
The University of Texas Health Science Center at San Antonio
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2030
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Oct 15, 2025 · Synced Nov 3, 2026, 5:08 AM EST
Terminated Phase 1 Interventional Accepts healthy volunteers

Response to Phenylketonuria to Tetrahydrobiopterin (BH4)

NCT00244218
View directory record Official record
Conditions
Phenylketonuria
Interventions
tetrahydrobiopterin (BH4)
Drug
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
10 Years and older
Enrollment
57 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2015
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 21, 2021 · Synced Nov 3, 2026, 5:08 AM EST
Recruiting Phase 4 Interventional

Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

NCT07477691
View directory record Official record
Conditions
Phenylketonuria
Interventions
Pegvaliase, Methotrexate
Biological · Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
12
States / cities
Los Angeles, California • Aurora, Colorado • Tampa, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Nov 3, 2026, 5:08 AM EST
Terminated No phase listed Observational

A Full-Arch Implant- Supported Prosthesis in the Upper and/or Lower Jaw-USA

NCT03655990
View directory record Official record
Conditions
Missing Teeth
Interventions
Treatment with the JUVORA™ Dental Disc
Device
Lead sponsor
Juvora Ltd.
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated May 11, 2022 · Synced Nov 3, 2026, 5:08 AM EST
Completed Not applicable Interventional Accepts healthy volunteers Results available

Absorptive Clearance in the Cystic Fibrosis Airway

NCT00541190
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Technetium [Tc-99m] sulfur colloid and Indium [In-111] DTPA
Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2008
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 24, 2017 · Synced Nov 3, 2026, 5:08 AM EST
Completed Phase 1 Interventional

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

NCT01917708
View directory record Official record
Conditions
Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions
Abatacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 26, 2019 · Synced Nov 3, 2026, 5:08 AM EST
Not listed No phase listed Observational Accepts healthy volunteers

Role of the Protein Osteoprotegerin in the Bone Health of Women With Congenital Adrenal Hyperplasia

NCT00694525
View directory record Official record
Conditions
Adrenal Hyperplasia, Congenital
Interventions
Not listed
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
20 Years to 35 Years · Female only
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 2, 2009 · Synced Nov 3, 2026, 5:08 AM EST
Completed Phase 3 Interventional Results available

Comparison of Chronocort® With Standard Glucocorticoid Therapy in Patients With Congenital Adrenal Hyperplasia

NCT02716818
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
Chronocort®, standard glucocorticoid therapy
Drug
Lead sponsor
Neurocrine UK Limited
Industry
Eligibility
18 Years and older
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Nov 3, 2026, 5:08 AM EST
Completed No phase listed Observational

Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

NCT01509768
View directory record Official record
Conditions
Sanfilippo Syndrome Type B
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
1 Year to 10 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 17, 2021 · Synced Nov 3, 2026, 5:08 AM EST
Completed Phase 3 Interventional Results available

A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

NCT00376168
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
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2018 · Synced Nov 3, 2026, 5:08 AM EST
Recruiting No phase listed Observational

Monitoring Pregnancy and Infant Outcomes Following Tralokinumab Exposure During Pregnancy in the US and Canada - PROTECT

NCT05938478
View directory record Official record
Conditions
Atopic Dermatitis, Eczema
Interventions
Tralokinumab
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
Female only
Enrollment
900 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2035
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Jan 28, 2025 · Synced Nov 3, 2026, 5:08 AM EST
Terminated Phase 1 Interventional

Nonmyeloablative Conditioning for Mismatched Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Disease

NCT02678143
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Alemtuzumab, Cyclophosphamide, Mycophenolate mofetil, Sirolimus, Fludarabine, Total body irradiation, Hematopoietic stem cell transplant
Drug · Radiation · Procedure
Lead sponsor
Washington University School of Medicine
Other
Eligibility
19 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Feb 14, 2022 · Synced Nov 3, 2026, 5:08 AM EST
Recruiting Phase 1 Interventional

Patients With Congenital Myasthenic Syndrome Will be Treated With Umbilical or Placental Derived Mesenchymal Stem Cell Exosome Solution

NCT07226726
View directory record Official record
Conditions
Congenital Myasthenic Syndrome
Interventions
AlloEx exosomes
Biological
Lead sponsor
The Foundation for Orthopaedics and Regenerative Medicine
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Naples, Florida
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Nov 3, 2026, 5:08 AM EST
Terminated Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Infliximab to Treat Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease

NCT00325078
View directory record Official record
Conditions
Chronic Granulomatous Disease, Crohn'S-like IBD, Inflammatory Bowel Disease (IBD)
Interventions
Infliximab
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
10 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 29, 2015 · Synced Nov 3, 2026, 5:08 AM EST