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ClinicalTrials.gov public records Last synced Oct 16, 2026, 12:58 AM EDT

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

Showing 769–792 of 10,531 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
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 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM EDT
Completed Phase 3 Interventional

A Randomized Trial of Unruptured Brain AVMs

NCT00389181
View directory record Official record
Conditions
Arteriovenous Malformations, Cerebral
Interventions
Interventional therapy, Medical management
Procedure · Other
Lead sponsor
Columbia University
Other
Eligibility
18 Years and older
Enrollment
226 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
25
States / cities
Phoenix, Arizona • Los Angeles, California • Redwood City, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2015 · Synced Oct 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM 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 16, 2026, 12:58 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers

Electrogastrography (EGC) in Premature Infants With Feeding Intolerance

NCT00008736
View directory record Official record
Conditions
Infant, Newborn, Diseases
Interventions
metoclopramide
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
1 Minute to 1 Month
Healthy volunteers
Accepts healthy volunteers
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 16, 2026, 12:58 AM EDT
Completed No phase listed Observational

Registry to Monitor the Susceptibility to Aztreonam of Pseudomonas Aeruginosa Isolates From Cystic Fibrosis Patients

NCT01375036
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
Gilead Sciences
Industry
Eligibility
6 Years and older
Enrollment
510 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
31
States / cities
Anchorage, Alaska • Tucson, Arizona • Little Rock, Arkansas + 20 more
Source: ClinicalTrials.gov public record
Updated Jan 11, 2017 · Synced Oct 16, 2026, 12:58 AM EDT
Completed Not applicable Interventional

Assessment of Bone Density and Bone Turnover Markers in Patients With Down Syndrome and Comparison to the Ts65Dn Model

NCT01148121
View directory record Official record
Conditions
Down Syndrome
Interventions
Skeletal Status Assessment of a Down Syndrome Population
Other
Lead sponsor
University of Arkansas
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated Dec 7, 2016 · Synced Oct 16, 2026, 12:58 AM EDT
Completed Phase 2 Interventional Results available

Rilonacept for Treatment of Familial Mediterranean Fever (FMF)

NCT00582907
View directory record Official record
Conditions
Familial Mediterranean Fever
Interventions
Rilonacept, Placebo
Drug
Lead sponsor
The Cleveland Clinic
Other
Eligibility
4 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
5
States / cities
Los Angeles, California • Madera, California • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 11, 2013 · Synced Oct 16, 2026, 12:58 AM EDT
Completed Phase 3 Interventional

Study Evaluating rFIX; BeneFIX® in Hemophilia B

NCT00093171
View directory record Official record
Conditions
Hemophilia B
Interventions
rFIX
Drug
Lead sponsor
Wyeth is now a wholly owned subsidiary of Pfizer
Industry
Eligibility
12 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 2005
U.S. locations
7
States / cities
Los Angeles, California • Aurora, Colorado • Detroit, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2009 · Synced Oct 16, 2026, 12:58 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Improving Quality by Maintaining Accurate Problems in the EHR

NCT02596087
View directory record Official record
Conditions
Asthma, Atrial Fibrillation, Chronic Obstructive Pulmonary Disease, Coronary Artery Disease, Congestive Heart Failure, Hyperlipidemia, Hypertension, Myocardial Infarction, Sickle Cell Disease, Sleep Apnea, Smoking, Stroke, Tuberculosis
Interventions
Problem List Suggestion
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
2,386 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2016
U.S. locations
4
States / cities
Boston, Massachusetts • Portland, Oregon • Camp Hill, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 8, 2023 · Synced Oct 16, 2026, 12:58 AM EDT
Completed Phase 3 Interventional Results available

A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B

NCT02053792
View directory record Official record
Conditions
Hemophilia B
Interventions
rIX-FP
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
Up to 70 Years · Male only
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
4
States / cities
Aurora, Colorado • Indianapolis, Indiana • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2022 · Synced Oct 16, 2026, 12:58 AM EDT
Not listed Phase 2 Interventional

Efficacy and Safety of Pirfenidone Treatment in HPS-ILD

NCT04193592
View directory record Official record
Conditions
Hermansky Pudlak Syndrome, Interstitial Lung Disease
Interventions
Pirfenidone
Drug
Lead sponsor
Jesse Roman
Other
Eligibility
18 Years to 75 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 10, 2019 · Synced Oct 16, 2026, 12:58 AM EDT