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ClinicalTrials.gov public records Last synced Oct 14, 2026, 4:14 PM EDT

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Showing 361–384 of 9,407 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Disease Clear all
Completed Phase 1Phase 2 Interventional Results available

A Safety, Pharmacokinetic & Dose-Escalation Study of KD019 in Subjects With Autosomal Dominant Polycystic Kidney Disease

NCT01559363
View directory record Official record
Conditions
Polycystic Kidney, Autosomal Dominant
Interventions
Tesevatinib
Drug
Lead sponsor
Kadmon, a Sanofi Company
Industry
Eligibility
18 Years to 62 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
11
States / cities
Los Angeles, California • Denver, Colorado • Kansas City, Kansas + 8 more
Source: ClinicalTrials.gov public record
Updated Nov 8, 2022 · Synced Oct 14, 2026, 4:14 PM EDT
Recruiting Phase 2 Interventional

Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)

NCT07587021
View directory record Official record
Conditions
Primary Hyperoxaluria Type 1, PH1
Interventions
YOLT-203, Placebo
Genetic · Drug
Lead sponsor
YolTech Therapeutics Co., Ltd
Industry
Eligibility
6 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
4
States / cities
Birmingham, Alabama • Aurora, Colorado • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2026 · Synced Oct 14, 2026, 4:14 PM EDT
Completed No phase listed Observational

Resynchronization Therapy in Young Patients With and Without CHD

NCT00208806
View directory record Official record
Conditions
Dilated Cardiomyopathy, Pediatric
Interventions
congestive heart failure patients
Other
Lead sponsor
Emory University
Other
Eligibility
1 Day to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2006
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 10, 2013 · Synced Oct 14, 2026, 4:14 PM EDT
Completed No phase listed Observational

Studying Childhood-onset Behavioral, Psychiatric, and Developmental Disorders

NCT01778504
View directory record Official record
Conditions
Neuropsychiatric Disorder, Neurological Disorder, Neurodevelopmental Disorder, Sleep
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
30 Days to 99 Years
Enrollment
237 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 14, 2026, 4:14 PM EDT
Completed Phase 3 Interventional Results available

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

NCT00712348
View directory record Official record
Conditions
Gaucher Disease
Interventions
Taliglucerase alfa
Drug
Lead sponsor
Pfizer
Industry
Eligibility
2 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
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 Oct 14, 2026, 4:14 PM EDT
Recruiting Phase 3 Interventional

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

NCT05477563
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 35 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
3
States / cities
New York, New York • Charlotte, North Carolina • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced Oct 14, 2026, 4:14 PM EDT
Not listed Not applicable Interventional

HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia

NCT03135184
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia, HoFH
Interventions
HDL Therapeutics PDS-2 System
Device
Lead sponsor
HDL Therapeutics
Industry
Eligibility
12 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 23, 2019 · Synced Oct 14, 2026, 4:14 PM EDT
Not yet recruiting Not applicable Interventional Accepts healthy volunteers

Evaluate the Safety and Efficacy of a Topical Serum (AGE Serum) in Improving Visible Signs of Aging

NCT07216352
View directory record Official record
Conditions
Laxity; Skin
Interventions
AGE Serum
Other
Lead sponsor
Goldman, Butterwick, Fitzpatrick and Groff
Other
Eligibility
25 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Oct 28, 2025 · Synced Oct 14, 2026, 4:14 PM EDT
Active, not recruiting No phase listed Observational

Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome

NCT05344599
View directory record Official record
Conditions
Postural Orthostatic Tachycardia Syndrome, Acute Hepatic Porphyria
Interventions
Autonomic Function Testing, Genetic Testing, Urine Testing, Blood laboratory Testing
Diagnostic Test
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years to 65 Years
Enrollment
70 participants
Timeline
2022 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 14, 2026, 4:14 PM EDT
Recruiting Not applicable Interventional

How Simplified Language Affects Comprehension and Learning in Young Children With Down Syndrome

NCT06951516
View directory record Official record
Conditions
Down Syndrome
Interventions
Linguistic simplification
Behavioral
Lead sponsor
Michigan State University
Other
Eligibility
2 Years to 7 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
East Lansing, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 12, 2026 · Synced Oct 14, 2026, 4:14 PM EDT
Completed No phase listed Observational

Evaluation of Biomarkers of Atopic Dermatitis in Pediatric Patients (PEDISTAD BIOMARKER STUDY)

NCT03849716
View directory record Official record
Conditions
Dermatitis Atopic
Interventions
Blood sample, Cheek swab
Other
Lead sponsor
Sanofi
Industry
Eligibility
0 Years to 11 Years
Enrollment
266 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
22
States / cities
Birmingham, Alabama • Montgomery, Alabama • Los Angeles, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 14, 2026, 4:14 PM EDT
Completed Phase 2 Interventional

A Study Evaluating the Safety and Efficacy of ENV-101 (Taladegib) in Patients With Advanced Solid Tumors Harboring PTCH1 Loss of Function Mutations

NCT05199584
View directory record Official record
Conditions
Solid Tumors With PTCH1 Loss-of-function Mutations
Interventions
ENV-101 (taladegib)
Drug
Lead sponsor
Endeavor Biomedicines, Inc.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
16
States / cities
Los Angeles, California • Santa Rosa, California • Tampa, Florida + 13 more
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Oct 14, 2026, 4:14 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Does Daily Supplementation of Lactobacillus Acidophilus MPH734, for One Week, Affect Acute (Immediate), Subacute (7 Days), and Post-treatment Discontinuation Lactose Metabolism, Gastrointestinal Symptoms, and Clinical Markers of Inflammation and Safety Compared to a Placebo

NCT04531033
View directory record Official record
Conditions
Lactose Intolerance
Interventions
Lactobacillus acidophilus MPH734, Placebo
Dietary Supplement
Lead sponsor
Lemuel W. Taylor IV
Other
Eligibility
18 Years to 55 Years
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Belton, Texas
Source: ClinicalTrials.gov public record
Updated May 16, 2024 · Synced Oct 14, 2026, 4:14 PM EDT
Completed No phase listed Observational

Adaptation and Quality of Life Among Adults With Neurofibromatosis Type I

NCT00684398
View directory record Official record
Conditions
Neurofibromatosis Type 1, Von Recklinghausen Disease, NF1
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
645 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced Oct 14, 2026, 4:14 PM EDT
Completed Phase 3 Interventional Results available

Open-Label Extension Study of Upadacitinib in Adult Participants With Moderate to Severe Atopic Dermatitis

NCT04195698
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Upadacitinib
Drug
Lead sponsor
AbbVie
Industry
Eligibility
18 Years to 75 Years
Enrollment
475 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
33
States / cities
Little Rock, Arkansas • Fountain Valley, California • Fresno, California + 27 more
Source: ClinicalTrials.gov public record
Updated Jan 9, 2025 · Synced Oct 14, 2026, 4:14 PM EDT
Terminated No phase listed Observational

A Study to Identify and Characterize LAL-D Patients in High-risk Populations

NCT02345421
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years and older
Enrollment
640 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 12 more
Source: ClinicalTrials.gov public record
Updated May 24, 2016 · Synced Oct 14, 2026, 4:14 PM EDT
Completed No phase listed Observational

PAD Registry and Fitbit Sub-Study Program

NCT03446014
View directory record Official record
Conditions
Peripheral Artery Disease
Interventions
Fitbit Sub-Study
Device
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years to 90 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 4, 2019 · Synced Oct 14, 2026, 4:14 PM EDT
Completed Early Phase 1 Interventional Results available

RHD Genotype Matched Red Cells for Anti-D

NCT04156906
View directory record Official record
Conditions
Sickle Cell Disease, Anti-D Antibodies
Interventions
D+ RH genotype matched red cell units for transfusion
Biological
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
8 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 2, 2025 · Synced Oct 14, 2026, 4:14 PM EDT
Recruiting Phase 3 Interventional

Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)

NCT07428499
View directory record Official record
Conditions
Hereditary Angioedema (HAE), Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2, HAE
Interventions
ADX-324 Dose Level 1, ADX-324 Dose Level 2
Drug
Lead sponsor
ADARx Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
15
States / cities
Litchfield Park, Arizona • Little Rock, Arkansas • San Diego, California + 12 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2026 · Synced Oct 14, 2026, 4:14 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Extinction Learning in Youth With Tourette Syndrome

NCT03765463
View directory record Official record
Conditions
Tourette Syndrome in Children, Tourette Syndrome in Adolescence, Habit Reversal Training, Tic
Interventions
Habit Reversal Training
Behavioral
Lead sponsor
Johns Hopkins University
Other
Eligibility
8 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2023
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 8, 2023 · Synced Oct 14, 2026, 4:14 PM EDT
Completed No phase listed Observational Results available

Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09

NCT02235493
View directory record Official record
Conditions
Hypophosphatasia, Bone Disease
Interventions
Retrospective Case Only
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
5 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 13, 2019 · Synced Oct 14, 2026, 4:14 PM EDT
Not listed Not applicable Interventional

A Pilot and Feasibility Study to Evaluate High vs Low Glycemic Index Mixed Meal Tolerance Test in Adolescents and Young Adults With Cystic Fibrosis

NCT06350149
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Sugar Sweetened Beverages (SSB), Hi GI, Lo GI
Other
Lead sponsor
Emory University
Other
Eligibility
12 Years to 21 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 5, 2024 · Synced Oct 14, 2026, 4:14 PM EDT
Completed No phase listed Observational

Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation

NCT01316549
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Fludarabine
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 4, 2021 · Synced Oct 14, 2026, 4:14 PM EDT
Recruiting Phase 3 Interventional

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

NCT06302348
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sepiapterin
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Up to 9 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Aug 28, 2026 · Synced Oct 14, 2026, 4:14 PM EDT