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ClinicalTrials.gov public records Last synced Oct 17, 2026, 6:42 AM EDT

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Showing 961–984 of 10,533 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Completed No phase listed Observational Accepts healthy volunteers

Multi-Center Study of Iron Overload: Pilot Study

NCT01114776
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
Interventions
Not listed
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
16 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2013
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Sep 24, 2020 · Synced Oct 17, 2026, 6:42 AM EDT
Active, not recruiting Phase 3 Interventional

A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

NCT05898620
View directory record Official record
Conditions
Rett Syndrome
Interventions
NGN-401
Genetic
Lead sponsor
Neurogene Inc.
Industry
Eligibility
3 Years and older · Female only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
13
States / cities
Birmingham, Alabama • Oakland, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Oct 17, 2026, 6:42 AM EDT
Completed Phase 2 Interventional Results available

Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes

NCT00004143
View directory record Official record
Conditions
Sickle Cell Anemia, Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria (PNH), Pure Red Cell Aplasia
Interventions
Campath, Chemo and/or TBI Allo SCT
Drug
Lead sponsor
David Rizzieri, MD
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
2
States / cities
Orlando, Florida • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 5, 2014 · Synced Oct 17, 2026, 6:42 AM EDT
Completed Phase 2 Interventional Results available

Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)

NCT02750618
View directory record Official record
Conditions
X-Linked Hypophosphatemia
Interventions
Burosumab
Biological
Lead sponsor
Kyowa Kirin, Inc.
Industry
Eligibility
1 Year to 4 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
3
States / cities
New Haven, Connecticut • Indianapolis, Indiana • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated May 6, 2024 · Synced Oct 17, 2026, 6:42 AM EDT
Completed Phase 1 Interventional

XK469R in Treating Patients With Refractory Hematologic Cancer

NCT00095797
View directory record Official record
Conditions
Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Myelomonocytic Leukemia (M4), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Blastic Phase Chronic Myelogenous Leukemia, Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation, Refractory Chronic Lymphocytic Leukemia, Relapsing Chronic Myelogenous Leukemia, Secondary Myelodysplastic Syndromes, Stage III Chronic Lymphocytic Leukemia, Stage IV Chronic Lymphocytic Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions
R(+)XK469, pharmacological study, laboratory biomarker analysis
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 8, 2013 · Synced Oct 17, 2026, 6:42 AM EDT
Completed No phase listed Observational

Podocan and Wnt Pathway in the Development of Aortopathy in Bicuspid Valve Disease

NCT02806310
View directory record Official record
Conditions
Aortic Valve Disease
Interventions
Blood draw
Other
Lead sponsor
Bassett Healthcare
Other
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Cooperstown, New York
Source: ClinicalTrials.gov public record
Updated Mar 2, 2022 · Synced Oct 17, 2026, 6:42 AM EDT
Terminated Phase 2 Interventional Results available

Effect of Ranitidine on Hyper-IgE Recurrent Infection (Job's) Syndrome

NCT00527878
View directory record Official record
Conditions
JOB's Syndrome, Hyper-IgE Recurrent Infection Syndrome, Immune Deficiency
Interventions
Ranitidine, Placebo
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
2 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 4, 2013 · Synced Oct 17, 2026, 6:42 AM EDT
Completed No phase listed Observational

Outcomes of Children With Congenital Single Ventricle Heart

NCT00266968
View directory record Official record
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Up to 21 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2013
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 2, 2014 · Synced Oct 17, 2026, 6:42 AM EDT
Completed Phase 2 Interventional

Evaluate the Efficacy and Safety of Arhalofenate for Preventing Flares and Reducing Serum Uric Acid in Gout Patients

NCT02063997
View directory record Official record
Conditions
Gout
Interventions
Arhalofenate 600 mg, Allopurinol 300 mg, Colchicine 0.6 mg, Placebo, Arhalofenate 800 mg
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
18 Years to 75 Years
Enrollment
248 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
52
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Tucson, Arizona + 49 more
Source: ClinicalTrials.gov public record
Updated Jan 29, 2018 · Synced Oct 17, 2026, 6:42 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluation of Home Based Assessments on Participants With DMD

NCT05657938
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Wearable Device, Duchenne Video Assessment (DVA)
Device · Other
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
4 Years to 12 Years · Male only
Enrollment
17 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
1
States / cities
Charlestown, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 14, 2023 · Synced Oct 17, 2026, 6:42 AM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

A Trial to Examine if Repinatrabit is Processed Differently in Adults With Reduced Liver or Kidney Function Compared to Adults With Normal Liver and Kidney Function

NCT07713758
View directory record Official record
Conditions
Phenylketonuria
Interventions
Repinatrabit
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2027
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Oct 17, 2026, 6:42 AM EDT
Completed Phase 4 Interventional Results available

Study to Evaluate the Safety and Efficacy of Alefacept (Amevive) in Subjects With Moderate to Severe Atopic Dermatitis

NCT00832585
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Alefacept
Drug
Lead sponsor
Rush University Medical Center
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Mar 10, 2011 · Synced Oct 17, 2026, 6:42 AM EDT
Terminated Phase 1 Interventional Results available

Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B

NCT02695160
View directory record Official record
Conditions
Hemophilia B
Interventions
SB-FIX
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jul 19, 2024 · Synced Oct 17, 2026, 6:42 AM EDT
Completed Phase 4 Interventional Results available

NOrth American Study for the Treatment of Recurrent epIstaxis With DoxycycLine: The NOSTRIL Trial

NCT04167085
View directory record Official record
Conditions
Epistaxis
Interventions
Doxycycline, Placebo
Drug · Other
Lead sponsor
University of California, Los Angeles
Other
Eligibility
18 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 20, 2022 · Synced Oct 17, 2026, 6:42 AM EDT
Completed Phase 2 Interventional Results available

Intranasal Oxytocin in Hypothalamic Obesity

NCT02849743
View directory record Official record
Conditions
Craniopharyngioma, Hypothalamic Obesity
Interventions
Syntocinon, Placebo (for Syntocinon)
Drug
Lead sponsor
Shana McCormack, MD
Other
Eligibility
10 Years to 35 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 5, 2022 · Synced Oct 17, 2026, 6:42 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

THC Crossover Study

NCT04429568
View directory record Official record
Conditions
THC, Cannabis, Cannabis Smoking, Cannabis Use, Unspecified, Cigarette Smoking, Tobacco Use, Nicotine Dependence, Nicotine Withdrawal, Cardiovascular Risk Factor
Interventions
Smoked Cannabis, Vaped Cannabis, Tobacco Cigarette
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
21 Years and older
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2024
U.S. locations
2
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 1, 2024 · Synced Oct 17, 2026, 6:42 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Actis Total Hip System 2 Year Follow-up

NCT02783274
View directory record Official record
Conditions
Osteoarthritis, Traumatic Arthritis, Rheumatoid Arthritis, Congenital Hip Dysplasia, Avascular Necrosis of the Femoral Head, Acute Traumatic Fracture of the Femoral Head or Neck, Certain Cases of Ankylosis, Non-union of Femoral Neck Fractures, Certain High Sub-Capital & Femoral Neck Fractures in Elderly
Interventions
Actis Total Hip System
Device
Lead sponsor
DePuy Orthopaedics
Industry
Eligibility
21 Years and older
Enrollment
266 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2024
U.S. locations
9
States / cities
Louisville, Kentucky • Boston, Massachusetts • Rochester, Minnesota + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 15, 2025 · Synced Oct 17, 2026, 6:42 AM EDT
Enrolling by invitation Not applicable Interventional

Applying Population Management Best Practices to Preventative Genomic Medicine Trial

NCT07053813
View directory record Official record
Conditions
Neoplastic Syndromes, Hereditary
Interventions
Web resources, Personalized outreach
Behavioral
Lead sponsor
University of Washington
Other
Eligibility
18 Years to 75 Years
Enrollment
900 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
2
States / cities
Denver, Colorado • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Feb 27, 2026 · Synced Oct 17, 2026, 6:42 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Dose Ranging Escalation Study of Tramadol Hydrochloride in Healthy Volunteers

NCT01947920
View directory record Official record
Conditions
Healthy
Interventions
Tramadol HCl, 50 mg, Placebo
Drug
Lead sponsor
Janssen Scientific Affairs, LLC
Industry
Eligibility
18 Years to 45 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Dec 16, 2014 · Synced Oct 17, 2026, 6:42 AM EDT
Completed No phase listed Observational

Owlet Smart Sock 3 Accuracy on Infants

NCT05635513
View directory record Official record
Conditions
Hypoxia Neonatal
Interventions
Owlet OSS V3
Device
Lead sponsor
Owlet Baby Care, Inc.
Industry
Eligibility
Not listed
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Feb 22, 2023 · Synced Oct 17, 2026, 6:42 AM EDT
Active, not recruiting Phase 3 Interventional

Oral N-acetylcysteine for Retinitis Pigmentosa

NCT05537220
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
N-acetylcysteine, Placebo
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 65 Years
Enrollment
485 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
24
States / cities
Davis, California • Los Angeles, California • San Francisco, California + 21 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 17, 2026, 6:42 AM EDT
Completed Phase 2Phase 3 Interventional Results available

A Long Term Safety Study of BCX7353 in Hereditary Angioedema

NCT03472040
View directory record Official record
Conditions
Hereditary Angioedema, HAE, Prophylaxis
Interventions
BCX7353
Drug
Lead sponsor
BioCryst Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
387 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
42
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Bentonville, Arkansas + 39 more
Source: ClinicalTrials.gov public record
Updated Jun 18, 2023 · Synced Oct 17, 2026, 6:42 AM EDT
Withdrawn Not applicable Interventional

Effects of Growth Hormone in Chronically Ill Children

NCT00286689
View directory record Official record
Conditions
Hurler Syndrome, Cerebral Palsy, Juvenile Rheumatoid Arthritis, Crohn Disease, HIV Infections
Interventions
Growth Hormone, Whole body Protein turnover, DEXA scan
Drug · Procedure
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
3 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 14, 2018 · Synced Oct 17, 2026, 6:42 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Clinical and Genetic Studies of Li-Fraumeni Syndrome

NCT01443468
View directory record Official record
Conditions
Li-Fraumeni Syndrome, Neoplasms, Tp53 Mutations
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2012
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 17, 2026, 6:42 AM EDT