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ClinicalTrials.gov public records Last synced Oct 19, 2026, 5:07 PM EDT

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Showing 25–48 of 10,329 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital, Hereditary, and Neonatal Diseases and Abnormalities Clear all
Terminated Phase 1 Interventional Accepts healthy volunteers

Evaluation of Oral Lipid Ingestion in Relation to Ovarian Androgen Secretion in Polycystic Ovary Syndrome (PCOS)

NCT01489319
View directory record Official record
Conditions
Polycystic Ovary Syndrome
Interventions
Salsalate
Drug
Lead sponsor
Indiana University
Other
Eligibility
18 Years to 40 Years · Female only
Enrollment
47 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2015
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 10, 2017 · Synced Oct 19, 2026, 5:07 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

NCT01966367
View directory record Official record
Conditions
Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndromes, Acquired Immunodeficiency Syndromes, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia
Interventions
CD34 Stem Cell Selection Therapy
Biological
Lead sponsor
Diane George
Other
Eligibility
Up to 40 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 14, 2025 · Synced Oct 19, 2026, 5:07 PM EDT
Recruiting No phase listed Observational

An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene

NCT06435000
View directory record Official record
Conditions
Stargardt, Stargardt's Disease, Stargardt Disease, STGD1
Interventions
Not listed
Lead sponsor
Splice Bio
Industry
Eligibility
12 Years to 65 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
14
States / cities
San Diego, California • Aurora, Colorado • Gainesville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2025 · Synced Oct 19, 2026, 5:07 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

PET Scans in Normal Volunteers and Patients With Fabry Disease

NCT00005111
View directory record Official record
Conditions
Fabry Disease, Healthy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 19, 2026, 5:07 PM EDT
Terminated Phase 3 Interventional

Rofecoxib Efficacy and Safety Evaluation Trial in Hemophilic Arthropathy

NCT04684511
View directory record Official record
Conditions
Hemophilic Arthropathy
Interventions
TRM-201 (Rofecoxib), Placebo
Drug
Lead sponsor
Tremeau Pharmceuticals, Inc.
Industry
Eligibility
12 Years to 75 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
19
States / cities
Los Angeles, California • Orange, California • Sacramento, California + 15 more
Source: ClinicalTrials.gov public record
Updated Oct 27, 2022 · Synced Oct 19, 2026, 5:07 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Study Of PF-06817024 In Healthy Subjects, In Patients With Chronic Rhinosinusitis With Nasal Polyps And in Patients With Atopic Dermatitis

NCT02743871
View directory record Official record
Conditions
Healthy, Chronic Rhinosinusitis With Nasal Polyps, Atopic Dermatitis
Interventions
PF-06817024, Placebo for PF-06817024
Biological · Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
97 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2021
U.S. locations
20
States / cities
Sacramento, California • New Haven, Connecticut • Shelton, Connecticut + 12 more
Source: ClinicalTrials.gov public record
Updated May 19, 2022 · Synced Oct 19, 2026, 5:07 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)

NCT07161544
View directory record Official record
Conditions
Stargardt Disease
Interventions
AAVB-039
Biological
Lead sponsor
AAVantgarde Bio Srl
Industry
Eligibility
8 Years to 55 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
8
States / cities
Phoenix, Arizona • Los Angeles, California • Jacksonville, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 19, 2026, 5:07 PM EDT
Withdrawn Not applicable Interventional

In Infants With Laryngomalacia, Does Acid-Blocking Medication Improve Respiratory Symptoms?

NCT02700087
View directory record Official record
Conditions
Laryngomalacia, Acid Reflux, Stridor
Interventions
ranitidine or famotidine, Placebo
Drug
Lead sponsor
Stanford University
Other
Eligibility
Up to 4 Months
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Nov 17, 2017 · Synced Oct 19, 2026, 5:07 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Prophylactic Continuous Positive Airway Pressure (CPAP) in Delivery Room, in the Prevention of Transient Tachypnea of Newborn (TTN): A Randomized Controlled Trial

NCT01082900
View directory record Official record
Conditions
Transient Tachypnea
Interventions
Prophylactic CPAP Administration
Other
Lead sponsor
New York Hospital Queens
Other
Eligibility
2 Minutes to 30 Minutes
Enrollment
138 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2013
U.S. locations
1
States / cities
Flushing, New York
Source: ClinicalTrials.gov public record
Updated Oct 6, 2015 · Synced Oct 19, 2026, 5:07 PM EDT
Completed No phase listed Observational

Computer Models of Airways in Children and Young Adults With Sleep Apnea and Down Syndrome

NCT01902407
View directory record Official record
Conditions
Down Syndrome, Obstructive Sleep Apnea
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
1 Year to 90 Years
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2026
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 19, 2026, 5:07 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease

NCT04536792
View directory record Official record
Conditions
Healthy Volunteers, Anemia, Sickle Cell
Interventions
AG-946, AG-946 Matched Placebo
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
122 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
8
States / cities
La Jolla, California • Boston, Massachusetts • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2024 · Synced Oct 19, 2026, 5:07 PM EDT
Terminated Not applicable Interventional

Customized Orthosis for Children With Clubfoot

NCT03853811
View directory record Official record
Conditions
Congenital Talipes Equinovarus
Interventions
Ankle Foot Orthosis (AFO)
Device
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
Up to 12 Months
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 11, 2022 · Synced Oct 19, 2026, 5:07 PM EDT
Recruiting Phase 3 Interventional

A Trial to Assess the Efficacy and Safety of Engasertib in Participants With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)

NCT07743671
View directory record Official record
Conditions
Hereditary Hemorrhagic Telangiectasia
Interventions
Engasertib, Placebo
Drug
Lead sponsor
Vaderis Therapeutics AG
Industry
Eligibility
18 Years and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
4
States / cities
Chicago, Illinois • Indianapolis, Indiana • Rochester, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 19, 2026, 5:07 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Pure Impact Treatment to Strengthen and Tone Abdomen, Quadriceps and Glutes Muscles

NCT07094971
View directory record Official record
Conditions
Muscle Tone, Muscle Strength
Interventions
Pure Impact
Device
Lead sponsor
Sofwave Medical LTD
Industry
Eligibility
25 Years to 60 Years
Enrollment
44 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Eugene, Oregon
Source: ClinicalTrials.gov public record
Updated Aug 3, 2025 · Synced Oct 19, 2026, 5:07 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Neurobiology and Treatment of Reading Disability in NF-1

NCT00624234
View directory record Official record
Conditions
Neurofibromatosis Type 1, Reading Disabilities
Interventions
Tutoring Program I, Tutoring Program II
Behavioral
Lead sponsor
Vanderbilt University
Other
Eligibility
8 Years to 17 Years
Enrollment
184 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2017
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 8, 2025 · Synced Oct 19, 2026, 5:07 PM EDT
Completed Phase 2 Interventional

Assessing Physical Function in Sickle Cell Patients Taking Voxelotor

NCT06023199
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Voxelotor
Drug
Lead sponsor
Inova Health Care Services
Other
Eligibility
18 Years to 65 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Falls Church, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 19, 2026, 5:07 PM EDT
Recruiting No phase listed Observational

Fontan Associated Liver Disease and the Evaluation of Biomarkers for Disease Severity Assessment

NCT05213598
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
Not listed
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
18 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2051
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 19, 2026, 5:07 PM EDT
Completed No phase listed Observational

C-Type Natriuretic Peptide and Achondroplasia

NCT01541306
View directory record Official record
Conditions
Achondroplasia, Hypochondroplasia
Interventions
Not listed
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
3 Months and older
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated Apr 14, 2015 · Synced Oct 19, 2026, 5:07 PM EDT
Terminated Phase 2 Interventional Results available

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation

NCT00953706
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
34
States / cities
Birmingham, Alabama • Anchorage, Alaska • Oakland, California + 30 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2015 · Synced Oct 19, 2026, 5:07 PM EDT
Completed Not applicable Interventional

Sleep Intervention in Young Boys with Duchenne Muscular Dystrophy

NCT04529707
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Transdiagnostic Behavioral Sleep Intervention
Behavioral
Lead sponsor
University of Pittsburgh
Other
Eligibility
6 Years to 18 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 7, 2025 · Synced Oct 19, 2026, 5:07 PM EDT
Completed Phase 1 Interventional

Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)

NCT00028236
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
Gene-Transduced Autologous CD34+ Stem Cells
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Months to 20 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 19, 2026, 5:07 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

NCT02536911
View directory record Official record
Conditions
Gaucher Disease
Interventions
eliglustat
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 79 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
2
States / cities
Miami, Florida • Knoxville, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 10, 2017 · Synced Oct 19, 2026, 5:07 PM EDT
Active, not recruiting No phase listed Observational

Colorectal Cancer Screening in Cystic Fibrosis

NCT05362344
View directory record Official record
Conditions
Cystic Fibrosis, Colorectal Cancer, Adenoma
Interventions
Stool tests
Procedure
Lead sponsor
University of Washington, the Collaborative Health Studies Coordinating Center
Other
Eligibility
18 Years to 75 Years
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
26
States / cities
Los Angeles, California • Palo Alto, California • Denver, Colorado + 21 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Oct 19, 2026, 5:07 PM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

The Electronic Medical Records and GEnomics (eMERGE) Network Genomic Risk Assessment

NCT05277116
View directory record Official record
Conditions
Genetic Disease
Interventions
Genome Informed Risk Assessment (GIRA) report
Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
3 Years to 75 Years
Enrollment
26,877 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2030
U.S. locations
12
States / cities
Birmingham, Alabama • Tempe, Arizona • Chicago, Illinois + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 19, 2026, 5:07 PM EDT