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ClinicalTrials.gov public records Last synced Oct 15, 2026, 4:44 PM EDT

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Showing 529–552 of 9,409 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Disease Clear all
Completed Not applicable Interventional Accepts healthy volunteers

Improving Outcome in Schizophrenia Through Identification of Genetic Risk Factors

NCT00178932
View directory record Official record
Conditions
Schizophrenia, Schizoaffective Disorder, Bipolar Disorder, Major Depression
Interventions
genetic analysis, genetic assay
Procedure
Lead sponsor
Northwestern University
Other
Eligibility
18 Years to 65 Years
Enrollment
37 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1998 – 2024
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 18, 2025 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease

NCT02225132
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Hydroxyurea
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
15 Years to 99 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 6, 2019 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Phase 3 Interventional Results available

Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C

NCT03814187
View directory record Official record
Conditions
ASCVD, Elevated Cholesterol, Heterozygous Familial Hypercholesterolemia, Homozygous Familial Hypercholesterolemia
Interventions
Inclisiran Sodium
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
3,275 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
141
States / cities
Birmingham, Alabama • Foley, Alabama • Huntsville, Alabama + 109 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2024 · Synced Oct 15, 2026, 4:44 PM EDT
Completed No phase listed Observational

Clinical Studies of Progeria

NCT00094393
View directory record Official record
Conditions
Progeria
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
6 Months to 70 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 15, 2019 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Not applicable Interventional

A Pilot Study to Obtain Preliminary Information Regarding the Efficacy and Safety of the Combination of Immune Globulin and Ganciclovir as Compared to Ganciclovir Alone in the Treatment of Sight-Threatening CMV Retinitis in Patients With AIDS

NCT00001999
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Globulin, Immune, Ganciclovir
Drug
Lead sponsor
American National Red Cross
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Phase 4 Interventional Results available

The Impact of Dupilumab on Quality of Life in Moderate to Severe Atopic Dermatitis Patients

NCT03667014
View directory record Official record
Conditions
Atopic Dermatitis, Atopic Dermatitis Eczema
Interventions
Dupilumab
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jan 9, 2024 · Synced Oct 15, 2026, 4:44 PM EDT
Not listed Not applicable Interventional

Investigation of Supplemental L-alanine in the Management of Dietary Fructose Intolerance

NCT04022434
View directory record Official record
Conditions
Fructose Intolerance
Interventions
Placedo, Alanine
Dietary Supplement
Lead sponsor
Augusta University
Other
Eligibility
18 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 15, 2020 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Phase 3 Interventional Results available

Vincristine, Dactinomycin, and Doxorubicin With or Without Radiation Therapy or Observation Only in Treating Younger Patients Who Are Undergoing Surgery for Newly Diagnosed Stage I, Stage II, or Stage III Wilms' Tumor

NCT00352534
View directory record Official record
Conditions
Stage I Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor
Interventions
3-Dimensional Conformal Radiation Therapy, Biospecimen Collection, Chest Radiography, Computed Tomography, Dactinomycin, Doxorubicin Hydrochloride, Echocardiography Test, Magnetic Resonance Imaging, Therapeutic Conventional Surgery, Ultrasound Imaging, Vincristine Sulfate
Radiation · Procedure · Biological + 1 more
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 29 Years
Enrollment
808 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2026
U.S. locations
210
States / cities
Birmingham, Alabama • Mobile, Alabama • Phoenix, Arizona + 150 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 15, 2026, 4:44 PM EDT
Terminated Phase 2 Interventional Results available

A Study to Test the Long-term Safety of BI 655130 in Patients With Atopic Eczema Who Took Part in Study 1368-0032

NCT04086121
View directory record Official record
Conditions
Dermatitis, Atopic
Interventions
Spesolimab
Drug
Lead sponsor
Boehringer Ingelheim
Industry
Eligibility
18 Years to 75 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
7
States / cities
Scottsdale, Arizona • Fort Myers, Florida • Miami, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 24, 2025 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Phase 3 Interventional Results available

Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II

NCT06467084
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
KVD900 150 mg, KVD900 300 mg, KVD900 600 mg
Drug
Lead sponsor
KalVista Pharmaceuticals, Ltd.
Industry
Eligibility
2 Years to 11 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
10
States / cities
Birmingham, Alabama • Scottsdale, Arizona • San Diego, California + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Randomized Phase I Safety and Immunogenicity Trial of Live Recombinant Canarypox ALVAC-HIV vCP205 Delivered by Alternate Mucosal Routes in HIV-1 Uninfected Adult Volunteers

NCT00000884
View directory record Official record
Conditions
HIV Infections
Interventions
MN rgp120/HIV-1 and GNE8 rgp120/HIV-1, ALVAC-HIV MN120TMG (vCP205), ALVAC-RG Rabies Glycoprotein (vCP65)
Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 50 Years
Enrollment
84 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Ends 2000
U.S. locations
6
States / cities
Birmingham, Alabama • Baltimore, Maryland • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 29, 2021 · Synced Oct 15, 2026, 4:44 PM EDT
Recruiting Not applicable Interventional

Using the EHR to Advance Genomic Medicine Across a Diverse Health System

NCT06377033
View directory record Official record
Conditions
Genetic Predisposition, Paraganglioma, Pheochromocytoma, ALS, Parkinson Disease, Polyneuropathies, Frontotemporal Dementia, Alzheimer Disease, Cardiomyopathy Non-ischemic, Thoracic Aortic Aneurysm
Interventions
Behavioral nudge
Behavioral
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 14, 2026 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Phase 2 Interventional

Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants

NCT01624311
View directory record Official record
Conditions
Urea Cycle Disorders, Organic Acidemias
Interventions
Therapeutic Hypothermia, Standard of Care Therapy
Other
Lead sponsor
Uta Lichter-Konecki
Other
Eligibility
1 Day to 30 Days
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2015
U.S. locations
3
States / cities
Washington D.C., District of Columbia • New York, New York • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jun 1, 2015 · Synced Oct 15, 2026, 4:44 PM EDT
Recruiting Not applicable Interventional

Deep Brain Stimulation Therapy in Movement Disorders

NCT02119611
View directory record Official record
Conditions
Parkinson's Disease
Interventions
Deep Brain Stimulation management
Device
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 100 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Phase 2 Interventional

Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

NCT01728064
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Placebo, EPI-743 400 mg, EPI-743 200 mg
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
3
States / cities
Los Angeles, California • Tampa, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 31, 2020 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Sildenafil Exercise: Role of PDE5 Inhibition

NCT04039087
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Sildenafil 40mg oral capsule, Placebo Oral capsule
Drug
Lead sponsor
National Jewish Health
Other
Eligibility
9 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
2
States / cities
Denver, Colorado • Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Aug 28, 2026 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Phase 2 Interventional

Lymphocyte Infusion in Treating Patients With Relapsed Cancer After Bone Marrow or Peripheral Stem Cell Transplantation

NCT00003887
View directory record Official record
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Kidney Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Neuroblastoma, Ovarian Cancer, Sarcoma, Testicular Germ Cell Tumor
Interventions
peripheral blood lymphocyte therapy
Biological
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
1 Year to 70 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2003
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Nov 30, 2011 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Not applicable Interventional

Impact of an Orthotic Intervention in Children With Peripheral Neuropathy

NCT03655587
View directory record Official record
Conditions
Gait, Drop Foot, Chemotherapy-induced Peripheral Neuropathy, Acute Lymphocytic Leukemia, Pediatric, Rhabdomyosarcoma, Wilms Tumor
Interventions
Solid ankle foot orthotic, Resting night splint
Device
Lead sponsor
Children's Hospitals and Clinics of Minnesota
Other
Eligibility
5 Years to 14 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
2
States / cities
Minneapolis, Minnesota • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 10, 2023 · Synced Oct 15, 2026, 4:44 PM EDT
Terminated No phase listed Observational

Kidney Information Network for Disease Research and Education

NCT03321604
View directory record Official record
Conditions
ESRD, Fabry Disease, Rare Diseases, Kidney Diseases, End Stage Renal Disease, Genetic Diseases, X-Linked
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
338 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 24, 2021 · Synced Oct 15, 2026, 4:44 PM EDT
Terminated Phase 2 Interventional Results available

Celecoxib for Thyroid Eye Disease

NCT02845336
View directory record Official record
Conditions
Thyroid Eye Disease
Interventions
Celecoxib, artificial tears
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 31, 2020 · Synced Oct 15, 2026, 4:44 PM EDT
Completed No phase listed Observational Results available

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

NCT00882921
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Idursulfase
Biological
Lead sponsor
Shire
Industry
Eligibility
5 Years and older · Male only
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Oct 15, 2026, 4:44 PM EDT
Completed No phase listed Observational

Family Disclosure in Cascade Testing

NCT04168736
View directory record Official record
Conditions
Genetic Predisposition
Interventions
Feasibility and Acceptability of Genetic Counseling Materials
Other
Lead sponsor
University of Virginia
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Charlottesville, Virginia
Source: ClinicalTrials.gov public record
Updated Nov 19, 2019 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Phase 2 Interventional Results available

A Gene Therapy Study for Hemophilia B

NCT02484092
View directory record Official record
Conditions
Hemophilia B
Interventions
SPK-9001
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
9
States / cities
Sacramento, California • Jackson, Mississippi • Madison, Mississippi + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2020 · Synced Oct 15, 2026, 4:44 PM EDT
Completed Phase 4 Interventional

TESTO: Testosterone Effects on Short-Term Outcomes in Infants With XXY

NCT03325647
View directory record Official record
Conditions
Klinefelter Syndrome
Interventions
Testosterone Cypionate 200 Milligram/Milliliter Injectable Solution, Placebo injectable saline
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
31 Days to 90 Days · Male only
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated May 6, 2022 · Synced Oct 15, 2026, 4:44 PM EDT