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ClinicalTrials.gov public records Last synced Oct 20, 2026, 11:14 AM EDT

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Showing 217–240 of 8,005 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed Phase 1Phase 2 Interventional

Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)

NCT00792766
View directory record Official record
Conditions
Tuberous Sclerosis, Angiolipoma
Interventions
everolimus (RAD001)
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 18, 2013 · Synced Oct 20, 2026, 11:14 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Cancer Preventive Vaccine Nous-209 for Lynch Syndrome Patients

NCT05078866
View directory record Official record
Conditions
Colorectal Carcinoma, Lynch Syndrome
Interventions
Adenoviral Tumor-specific Neoantigen Priming Vaccine GAd-209-FSP, Biospecimen Collection, Endoscopic Biopsy, MVA Tumor-specific Neoantigen Boosting Vaccine MVA-209-FSP, Questionnaire Administration
Biological · Procedure · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
3
States / cities
Duarte, California • Philadelphia, Pennsylvania • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 3, 2026 · Synced Oct 20, 2026, 11:14 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

NCT06152237
View directory record Official record
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
5 Years to 8 Years · Female only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
3
States / cities
Chicago, Illinois • Saint Paul, Minnesota • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 15, 2025 · Synced Oct 20, 2026, 11:14 AM EDT
Recruiting No phase listed Observational

Congenital Uterine Anomalies: Identifying Cancer Associations and Genetic and Environmental Factors to Improve Clinical Care

NCT04661072
View directory record Official record
Conditions
Congenital Uterine Anomaly
Interventions
Not listed
Lead sponsor
Yale University
Other
Eligibility
13 Years and older · Female only
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Oct 20, 2026, 11:14 AM EDT
Completed Not applicable Interventional

Randomized Study of Folic Acid Therapy for Hyperhomocysteinemia in Patients With End Stage Renal Disease Receiving Hemodialysis

NCT00004495
View directory record Official record
Conditions
End Stage Renal Disease, Hyperhomocysteinemia
Interventions
cyanocobalamin, folic acid, pyridoxine
Drug
Lead sponsor
Georgetown University
Other
Eligibility
21 Years to 89 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2000
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 20, 2026, 11:14 AM EDT
Completed No phase listed Observational

Positional Cloning of the Gene(s) Responsible for Alagille Syndrome

NCT00001642
View directory record Official record
Conditions
Alagille Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 20, 2026, 11:14 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Mayo AVC Registry and Biobank

NCT03049254
View directory record Official record
Conditions
Arrhythmogenic Right Ventricular Cardiomyopathy, Cardiomyopathies, Heart Diseases, Cardiovascular Diseases, Sudden Cardiac Arrest, Sudden Cardiac Death, Arrhythmogenic Right Ventricular Dysplasia, Arrhythmogenic Ventricular Cardiomyopathy, Familial Dilated Cardiomyopathy, Cardiovascular Abnormalities, Sarcoidosis, Cardiac Arrhythmia, Cardiac Sarcoidosis, Myocarditis, Inflammatory Cardiomyopathy, Ventricular Tachycardia, Right Ventricular Outflow Tract Ventricular Tachycardia
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2027
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced Oct 20, 2026, 11:14 AM EDT
Completed Phase 2 Interventional Results available

ERG/5-HTP in Fragile X Syndrome (FXS)

NCT05030129
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
5-Hydroxytryptophan, Ergoloid Mesylates, Matching placebo for Ergoloid mesylates, Matching placebo for 5-Hydroxytryptophan
Drug
Lead sponsor
Elizabeth Berry-Kravis
Other
Eligibility
18 Years to 45 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 20, 2026, 11:14 AM EDT
Completed Phase 3 Interventional Results available

A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

NCT03125395
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
LUM/IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years and older
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
17
States / cities
Palo Alto, California • Aurora, Colorado • Chicago, Illinois + 14 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2020 · Synced Oct 20, 2026, 11:14 AM EDT
Completed Phase 1 Interventional

Continuous Hyperthermic Peritoneal Perfusion (CHPP) With Cisplatin for Children With Peritoneal Cancer

NCT00436657
View directory record Official record
Conditions
Peritoneal Neoplasms, Retroperitoneal Neoplasms, Gastrointestinal Neoplasms, Adenocarcinoma, Neuroblastoma, Ovarian Neoplasms, Sarcoma, Adrenocortical Carcinoma, Wilms Tumor, Rhabdomyosarcoma, Desmoplastic Small Round Cell Tumor
Interventions
CHPP of Cisplatin, Abdominal Surgery
Drug · Procedure
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
3 Years to 18 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 2, 2012 · Synced Oct 20, 2026, 11:14 AM EDT
Terminated Phase 2 Interventional Results available

Efficacy and Safety Study of the Use of Benralizumab for Patients With Moderate to Severe Atopic Dermatitis

NCT04605094
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Benralizumab, Placebo / Benralizumab
Biological
Lead sponsor
AstraZeneca
Industry
Eligibility
12 Years to 130 Years
Enrollment
194 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
13
States / cities
Los Angeles, California • Newport Beach, California • Bridgeport, Connecticut + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2023 · Synced Oct 20, 2026, 11:14 AM EDT
Completed No phase listed Observational

Familial Prostate Cancer

NCT00001377
View directory record Official record
Conditions
Neoplastic Syndromes, Hereditary, Prostatic Neoplasms
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 20, 2026, 11:14 AM EDT
Completed Not applicable Interventional

PACE CF, a 3-session Mind-body Pain Management Program for Adults With Cystic Fibrosis

NCT06606457
View directory record Official record
Conditions
Cystic Fibrosis (CF), Pain Management
Interventions
Pain Acknowledgment Coping and Empowerment in CF (PACE CF)
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 12, 2025 · Synced Oct 20, 2026, 11:14 AM EDT
Recruiting Phase 1 Interventional

PrP-targeting siRNA Safety & Mechanism Study

NCT07444580
View directory record Official record
Conditions
Prion Disease
Interventions
PrP-siRNA
Drug
Lead sponsor
Broad Institute of MIT and Harvard
Other
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
5
States / cities
Boston, Massachusetts • Rochester, Minnesota • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 20, 2026, 11:14 AM EDT
Recruiting Phase 2 Interventional

Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients

NCT07766980
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy in Children, DMD, Muscular Dystrophy
Interventions
VM100 Nutritional Supplement
Drug
Lead sponsor
University of Florida
Other
Eligibility
6 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 20, 2026, 11:14 AM EDT
Completed Phase 1Phase 2 Interventional

Safety and Efficacy of an Antioxidant-rich Multivitamin Supplement in Cystic Fibrosis

NCT01018303
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
AquADEK
Dietary Supplement
Lead sponsor
University of Colorado, Denver
Other
Eligibility
10 Years to 40 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Oct 15, 2012 · Synced Oct 20, 2026, 11:14 AM EDT
Recruiting Phase 4 Interventional

Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency

NCT07135427
View directory record Official record
Conditions
Alpha 1-Antitrypsin, COPD, Antibody Deficiency
Interventions
20-valent pneumococcal conjugate vaccine
Biological
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
19 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Nov 10, 2025 · Synced Oct 20, 2026, 11:14 AM EDT
Completed No phase listed Observational

Abnormalities of the Eye's Anterior Chamber, Iris, Cornea and Lens

NCT00001161
View directory record Official record
Conditions
Aniridia, Eye Abnormality, Ocular Hypertension, Open Angle Glaucoma
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1977 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 20, 2026, 11:14 AM EDT
Completed Not applicable Interventional

Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

NCT02428673
View directory record Official record
Conditions
Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Rett Syndrome
Interventions
Assisted Standing Treatment Program
Other
Lead sponsor
Gillette Children's Specialty Healthcare
Other
Eligibility
3 Years to 14 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Oct 20, 2026, 11:14 AM EDT
Recruiting Phase 2 Interventional

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old

NCT07536269
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
Crinecerfont
Drug
Lead sponsor
Neurocrine Switzerland GmbH
Industry
Eligibility
3 Months to 47 Months
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
11
States / cities
San Francisco, California • Aurora, Colorado • Hartford, Connecticut + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 20, 2026, 11:14 AM EDT
Recruiting Not applicable Interventional

Trial of Combined Obstetric Carrier Screening and Hereditary Cancer Screening

NCT07052266
View directory record Official record
Conditions
Hereditary Cancer Syndromes
Interventions
MyRisk Hereditary Cancer Test
Genetic
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years to 55 Years · Female only
Enrollment
550 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Brooklyn, New York • New York, New York • Queens, New York
Source: ClinicalTrials.gov public record
Updated Dec 26, 2025 · Synced Oct 20, 2026, 11:14 AM EDT
Recruiting Phase 2 Interventional

A Phase 2b Dose-Ranging Study to Evaluate the Efficacy and Safety of ENV-294 in Adults With Moderate-to-Severe Atopic Dermatitis

NCT07643766
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
ENV-294, Placebo
Drug · Other
Lead sponsor
Enveda Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
9
States / cities
Encinitas, California • Los Angeles, California • Northridge, California + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2026 · Synced Oct 20, 2026, 11:14 AM EDT
Completed Phase 3 Interventional Results available

The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency

NCT00301366
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
alpha-1 proteinase inhibitor (human)
Drug
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years and older
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
8
States / cities
Denver, Colorado • Gainesville, Florida • Miami, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2014 · Synced Oct 20, 2026, 11:14 AM EDT
Completed Not applicable Interventional Results available

Trial of Minocycline to Treat Children With Fragile X Syndrome

NCT01053156
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
minocycline hydrochloride, Placebo
Drug
Lead sponsor
University of California, Davis
Other
Eligibility
42 Months to 16 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated May 30, 2017 · Synced Oct 20, 2026, 11:14 AM EDT