Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.

Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 15, 2026, 10:48 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 673–696 of 9,409 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Disease Clear all
Suspended Early Phase 1 Interventional

PBA Use for Treatment of ATF6-/- Patients

NCT04041232
View directory record Official record
Conditions
ACHROMATOPSIA 7, Achromatopsia
Interventions
PBA
Drug
Lead sponsor
Columbia University
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Oct 15, 2026, 10:48 PM EDT
Completed Not applicable Interventional

Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Myelodysplastic Syndrome and Acute Leukemia Related to Fanconi's Anemia

NCT00005892
View directory record Official record
Conditions
Fanconi's Anemia, Myelodysplastic Syndromes, Leukemia, Nonlymphocytic, Acute, Leukemia, Lymphocytic, Acute
Interventions
cyclophosphamide, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years to 54 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 15, 2026, 10:48 PM EDT
Completed Phase 3 Interventional

EMANATE: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway

NCT05093634
View directory record Official record
Conditions
Obesity, Genetic Obesity
Interventions
Setmelanotide, Placebo
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years to 65 Years
Enrollment
296 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
32
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Los Angeles, California + 26 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2026 · Synced Oct 15, 2026, 10:48 PM EDT
Terminated Phase 3 Interventional Results available

Study of Aztreonam for Inhalation in Children With Cystic Fibrosis and New Infection of the Airways by Pseudomonas Aeruginosa Bacteria

NCT03219164
View directory record Official record
Conditions
Pseudomonas Aeruginosa Respiratory Tract Infection/Colonization, Cystic Fibrosis
Interventions
AZLI, Placebo
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
3 Months to 18 Years
Enrollment
149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
12
States / cities
San Francisco, California • Washington D.C., District of Columbia • St. Petersburg, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2022 · Synced Oct 15, 2026, 10:48 PM EDT
Terminated Phase 2 Interventional Results available

Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome

NCT01348087
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
AFQ056
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
10
States / cities
Phoenix, Arizona • Sacramento, California • Decatur, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated May 25, 2016 · Synced Oct 15, 2026, 10:48 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Genetic Analysis of Inherited Urologic Malignant Disorders: Collection of Samples

NCT00001814
View directory record Official record
Conditions
Neoplastic Syndromes, Hereditary, Urologic Neoplasms
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1999 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 15, 2026, 10:48 PM EDT
Recruiting Not applicable Interventional

Empowering Adolescents and Young Adults With Sickle Cell Disease as Partners in Treatment Decision Making (EMPOWER-AYA)

NCT05383911
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
SCD Shared Decision-Making Toolkit for AYAs - including VR, SCD Shared Decision-Making Toolkit for AYAs - without VR
Behavioral
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
15 Years and older
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Oct 15, 2026, 10:48 PM EDT
Not listed No phase listed Observational

Genetic Basis of Mitral Valve Prolapse

NCT01719211
View directory record Official record
Conditions
Mitral Valve Prolapse
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 80 Years
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 8, 2023 · Synced Oct 15, 2026, 10:48 PM EDT
Completed Phase 3 Interventional

Standard Genetic Counseling With or Without a Decision Guide in Improving Communication Between Mothers Undergoing BRCA1/2 Testing and Their Minor-Age Children

NCT00685256
View directory record Official record
Conditions
Breast Cancer, Hereditary Breast/Ovarian Cancer (brca1, brca2)
Interventions
counseling intervention, educational intervention, survey administration, psychosocial assessment and care, supportive care
Other · Behavioral
Lead sponsor
Georgetown University
Other
Eligibility
8 Years to 120 Years · Female only
Enrollment
245 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 17, 2017 · Synced Oct 15, 2026, 10:48 PM EDT
Completed Phase 4 Interventional Results available

Infusion Duration Study To Assess Tolerability of Pegloticase Administered With a Shorter Infusion Duration in Subjects With Uncontrolled Gout Receiving Methotrexate

NCT04511702
View directory record Official record
Conditions
Chronic Uncontrolled Gout, Gout, Uncontrolled Gout
Interventions
Pegloticase with MTX
Biological
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
191 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
39
States / cities
Birmingham, Alabama • Anchorage, Alaska • Gilbert, Arizona + 33 more
Source: ClinicalTrials.gov public record
Updated Oct 20, 2025 · Synced Oct 15, 2026, 10:48 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers

Effects of Pimecrolimus Cream 1% on the Molecular and Cellular Profile of Adult Male Patients With Atopic Dermatitis

NCT00117377
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Pimecrolimus, Placebo
Drug
Lead sponsor
Novartis
Industry
Eligibility
20 Years and older · Male only
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2005
U.S. locations
3
States / cities
New York, New York • Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Dec 17, 2007 · Synced Oct 15, 2026, 10:48 PM EDT
Active, not recruiting Phase 2 Interventional

A Long-term Study of STAR-0215 in Participants With Hereditary Angioedema

NCT06007677
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
STAR-0215
Drug
Lead sponsor
Astria Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
12
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Little Rock, Arkansas + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 23, 2025 · Synced Oct 15, 2026, 10:48 PM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

TANDem-2: Closing the Gap to Interventions for TAND

NCT06879665
View directory record Official record
Conditions
Adult Caregivers of Individuals With TSC, Tuberous Sclerosis Complex (TSC)
Interventions
Well-Beans for Caregivers Program
Behavioral
Lead sponsor
Vrije Universiteit Brussel
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2027
U.S. locations
1
States / cities
Silver Spring, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Oct 15, 2026, 10:48 PM EDT
Completed Phase 2 Interventional

Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation

NCT02589236
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Cavosonstat, Placebo
Drug
Lead sponsor
Nivalis Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
138 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
46
States / cities
Birmingham, Alabama • Tucson, Arizona • Los Angeles, California + 42 more
Source: ClinicalTrials.gov public record
Updated Jan 10, 2017 · Synced Oct 15, 2026, 10:48 PM EDT
Recruiting Phase 1 Interventional

Drug-Drug Interaction Study of ABP-671 in Gout Patients

NCT07367971
View directory record Official record
Conditions
Gout Chronic, Gout, DDI (Drug-Drug Interaction)
Interventions
ABP-671 plus allopurinol
Drug
Lead sponsor
Atom Therapeutics Co., Ltd
Industry
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026
U.S. locations
1
States / cities
Cary, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 15, 2026, 10:48 PM EDT
Not listed No phase listed Observational

A Study to Prospectively Assess Disease Progression in Male Children With X-ALD

NCT03278899
View directory record Official record
Conditions
X-Linked Adrenoleukodystrophy
Interventions
Not listed
Lead sponsor
NeuroVia, Inc.
Industry
Eligibility
2 Years to 13 Years · Male only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 8, 2018 · Synced Oct 15, 2026, 10:48 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

NCT01907087
View directory record Official record
Conditions
Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease
Interventions
BMN 190
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
3 Years to 15 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 8, 2019 · Synced Oct 15, 2026, 10:48 PM EDT
Completed Phase 3 Interventional Results available

rVWF IN PROPHYLAXIS

NCT02973087
View directory record Official record
Conditions
Von Willebrand Disease
Interventions
von Willebrand factor (Recombinant), Antihemophilic Factor (Recombinant)
Biological
Lead sponsor
Takeda
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
8
States / cities
Loveland, Colorado • Gainesville, Florida • Jacksonville, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2021 · Synced Oct 15, 2026, 10:48 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers

The Role of 5-alpha Reductase in Mediating Testosterone Actions

NCT00493987
View directory record Official record
Conditions
Healthy
Interventions
Testosterone Enanthate, Dutasteride
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years to 50 Years · Male only
Enrollment
184 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2010
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 13, 2010 · Synced Oct 15, 2026, 10:48 PM EDT
Terminated Phase 2 Interventional Results available

Follicle Stimulating Hormone (FSH) to Improve Testicular Development in Men With Hypogonadism

NCT00064987
View directory record Official record
Conditions
Hypogonadism, Kallmann Syndrome
Interventions
Testicular biopsy, gonadotropin releasing hormone (GnRH), follicle stimulating hormone (FSH)
Procedure · Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years and older · Male only
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 7, 2017 · Synced Oct 15, 2026, 10:48 PM EDT
Completed Phase 4 Interventional Results available

A Study to Evaluate IGSC 20% Biweekly Dosing in Treatment-Experienced Participants and Loading/Maintenance Dosing in Treatment-Naïve Participants With Primary Immunodeficiency

NCT04566692
View directory record Official record
Conditions
Primary Immunodeficiency
Interventions
IGSC 20%
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
6 Years to 75 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
12
States / cities
Birmingham, Alabama • Litchfield Park, Arizona • North Palm Beach, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2023 · Synced Oct 15, 2026, 10:48 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluating Color Perception Under LED Red/Green and Green Dominant Light

NCT01927536
View directory record Official record
Conditions
Color Blindness, Acquired
Interventions
White LED Flashlight, Red/Green Green Dominant LED Flashlight
Device
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years to 66 Years
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2019
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated May 15, 2019 · Synced Oct 15, 2026, 10:48 PM EDT
Completed Phase 2 Interventional Results available

MEK Inhibitor PD-0325901 Trial in Adolescents and Adults With NF1

NCT02096471
View directory record Official record
Conditions
Neurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PN
Interventions
PD-0325901
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
16 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
11
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Jan 10, 2019 · Synced Oct 15, 2026, 10:48 PM EDT
Terminated No phase listed Observational

NGLY1 Deficiency: A Prospective Natural History Study

NCT03834987
View directory record Official record
Conditions
Genetic Syndrome
Interventions
Neurodevelopmental Assessment
Other
Lead sponsor
Stanford University
Other
Eligibility
Not listed
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Jun 8, 2022 · Synced Oct 15, 2026, 10:48 PM EDT