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ClinicalTrials.gov public records Last synced Oct 25, 2026, 10:32 PM EDT

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

Showing 1297–1320 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Recruiting Phase 3 Interventional

A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A

NCT07416526
View directory record Official record
Conditions
Hemophilia A
Interventions
NXT007, Human Coagulation Factor VIII
Combination Product · Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
12 Years and older
Enrollment
138 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Aurora, Colorado • Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 25, 2026, 10:32 PM EDT
Completed Not applicable Interventional

Telehealth Cognitive Behavioral Stress Management for Adults With Cystic Fibrosis

NCT03560726
View directory record Official record
Conditions
Anxiety, Depression, Stress, Coping Skills, Cystic Fibrosis
Interventions
Cognitive Behavioral Stress Management
Behavioral
Lead sponsor
National Jewish Health
Other
Eligibility
18 Years to 80 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Sep 9, 2020 · Synced Oct 25, 2026, 10:32 PM EDT
Completed Phase 1 Interventional

Busulfan, Melphalan, Topotecan Hydrochloride, and a Stem Cell Transplant in Treating Patients With Newly Diagnosed or Relapsed Solid Tumor

NCT00638898
View directory record Official record
Conditions
Solid Tumor, Adult Central Nervous System Germ Cell Tumor, Adult Rhabdomyosarcoma, Childhood Central Nervous System Germ Cell Tumor, Childhood Soft Tissue Sarcoma, Ewing Sarcoma, Metastatic Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Ovarian Mixed Germ Cell Tumor, Previously Untreated Childhood Rhabdomyosarcoma, Recurrent Adult Brain Tumor, Recurrent Adult Soft Tissue Sarcoma, Recurrent Childhood Brain Stem Glioma, Recurrent Childhood Cerebellar Astrocytoma, Recurrent Childhood Cerebral Astrocytoma, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Medulloblastoma, Recurrent Childhood Pineoblastoma, Recurrent Childhood Supratentorial Primitive Neuroectodermal Tumor, Recurrent Childhood Visual Pathway and Hypothalamic Glioma, Recurrent Childhood Visual Pathway Glioma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Extragonadal Germ Cell Tumor, Recurrent Extragonadal Non-seminomatous Germ Cell Tumor, Recurrent Malignant Testicular Germ Cell Tumor, Recurrent Neuroblastoma, Recurrent Ovarian Germ Cell Tumor, Recurrent Wilms Tumor and Other Childhood Kidney Tumors, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
busulfan, melphalan, topotecan hydrochloride, laboratory biomarker analysis, filgrastim, autologous hematopoietic stem cell transplantation, pharmacological study, autologous bone marrow transplantation
Drug · Other · Biological + 1 more
Lead sponsor
City of Hope Medical Center
Other
Eligibility
6 Months to 40 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2024
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Mar 20, 2025 · Synced Oct 25, 2026, 10:32 PM EDT
Recruiting No phase listed Observational

Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)

NCT06669949
View directory record Official record
Conditions
Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Interventions
no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 25, 2026, 10:32 PM EDT
Terminated Phase 2 Interventional Results available

A Study to Assess Subcutaneous Lirentelimab (AK002) in Atopic Dermatitis

NCT05155085
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
AK002, Placebo
Drug · Other
Lead sponsor
Allakos Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
131 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
40
States / cities
Birmingham, Alabama • Cullman, Alabama • Gilbert, Arizona + 33 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2024 · Synced Oct 25, 2026, 10:32 PM EDT
Recruiting No phase listed Observational

Study of BEST1 Vitelliform Macular Dystrophy

NCT05809635
View directory record Official record
Conditions
Best Vitelliform Macular Dystrophy, Retinitis Pigmentosa
Interventions
Natural History Study
Other
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 30, 2025 · Synced Oct 25, 2026, 10:32 PM EDT
Completed Phase 2 Interventional Results available

A Multicenter Study Assessing the Efficacy and Safety of STN1010904 Ophthalmic Suspension 0.03% and 0.1% Compared With Vehicle in Subjects With Fuchs Endothelial Corneal Dystrophy (FECD)

NCT05376176
View directory record Official record
Conditions
Fuchs Endothelial Corneal Dystrophy
Interventions
STN1010904 ophthalmic suspension 0.03% BID, STN1010904 ophthalmic suspension 0.1% BID, Placebo (Vehicle) BID
Drug
Lead sponsor
Santen Inc.
Industry
Eligibility
30 Years to 75 Years
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
11
States / cities
Los Angeles, California • Indianapolis, Indiana • Baltimore, Maryland + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2026 · Synced Oct 25, 2026, 10:32 PM EDT
Withdrawn Phase 2 Interventional

Liq-NOL Efficacy in Pediatric Patients With Down Syndrome

NCT00891917
View directory record Official record
Conditions
Down Syndrome
Interventions
Ubiquinol-10 Syrup, syrup (placebo)
Dietary Supplement
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
6 Years to 16 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 9, 2020 · Synced Oct 25, 2026, 10:32 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

AAV Gene Therapy Study for Subjects with PKU

NCT04480567
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
BMN 307
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
15 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
2
States / cities
Tampa, Florida • Morristown, New Jersey
Source: ClinicalTrials.gov public record
Updated Dec 12, 2024 · Synced Oct 25, 2026, 10:32 PM EDT
Terminated Phase 4 Interventional Results available

The Purpose of This Study is to Evaluate the Effects of Ruxolitinib Cream on Adults With Atopic Dermatitis Experiencing Sleep Disturbance.

NCT05696392
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
ruxolitinib cream
Drug
Lead sponsor
Incyte Corporation
Industry
Eligibility
18 Years and older
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
30
States / cities
Fountain Valley, California • Long Beach, California • Washington D.C., District of Columbia + 26 more
Source: ClinicalTrials.gov public record
Updated Dec 19, 2025 · Synced Oct 25, 2026, 10:32 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 25, 2026, 10:32 PM EDT
Completed Not applicable Interventional

Impact of Telerehabilitation Training on Pediatric Cystic Fibrosis Patients: An Exploratory Study

NCT02715921
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Tele-exercise
Other
Lead sponsor
MemorialCare Health System
Other
Eligibility
8 Years to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Jul 30, 2026 · Synced Oct 25, 2026, 10:32 PM EDT
Active, not recruiting No phase listed Observational

An Observational Study in Children and Adults With Stargardt Disease

NCT06591806
View directory record Official record
Conditions
Stargardt Disease
Interventions
Not listed
Lead sponsor
AAVantgarde Bio Srl
Industry
Eligibility
8 Years to 50 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Oct 25, 2026, 10:32 PM EDT
Completed Early Phase 1 Interventional

Angelman Syndrome (AS) Biomarker Study

NCT04103333
View directory record Official record
Conditions
Angelman Syndrome, Chromosome 15q Duplication (dup15q) Syndrome
Interventions
Lumbar Puncture, Blood Collection
Procedure
Lead sponsor
Biogen
Industry
Eligibility
Up to 50 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
5
States / cities
San Diego, California • Chicago, Illinois • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2022 · Synced Oct 25, 2026, 10:32 PM EDT
Completed No phase listed Observational

Estimation of the Carrier Frequency and Incidence of Smith-Lemli-Opitz Syndrome in African Americans

NCT00017732
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 25, 2026, 10:32 PM EDT
Terminated Not applicable Interventional Results available

Gene Expression Following Ultherapy® Treatment

NCT02441036
View directory record Official record
Conditions
Skin Laxity
Interventions
Ultherapy Treatment
Device
Lead sponsor
Ulthera, Inc
Industry
Eligibility
45 Years to 70 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Phoenix, Arizona • Mequon, Wisconsin
Source: ClinicalTrials.gov public record
Updated Feb 20, 2019 · Synced Oct 25, 2026, 10:32 PM EDT
Completed Phase 2 Interventional Results available

Topical NanoDox® for Atopic Dermatitis

NCT02910011
View directory record Official record
Conditions
Dermatitis, Atopic
Interventions
Nanodox 1% (doxycycline monohydrate hydrogel)
Drug
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
2
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Mar 26, 2020 · Synced Oct 25, 2026, 10:32 PM EDT
Withdrawn Phase 1 Interventional

Nivolumab for High-Risk MDS/AML Patients After Allogeneic Stem Cell Transplant With Post-Transplant Cyclophosphamide

NCT04361058
View directory record Official record
Conditions
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Myelodysplastic Syndrome Acute Myeloid Leukemia
Interventions
Nivolumab
Drug
Lead sponsor
SCRI Development Innovations, LLC
Other
Eligibility
18 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Dec 5, 2023 · Synced Oct 25, 2026, 10:32 PM EDT
Completed No phase listed Observational

Genetic Analysis of the Chiari I Malformation

NCT00004738
View directory record Official record
Conditions
Syringomyelia, Type I Arnold Chiari Malformation
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
1 Year and older
Enrollment
152 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 25, 2026, 10:32 PM EDT
Completed Not applicable Interventional

An Interactive Program to Improve Care for Children With CF

NCT00185549
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
CF. DOC
Behavioral
Lead sponsor
Stanford University
Other
Eligibility
1 Month to 21 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Oct 5, 2006 · Synced Oct 25, 2026, 10:32 PM EDT
Terminated Phase 1Phase 2 Interventional

Oral Hydroxychloroquine (HCQ) for Retinitis Pigmentosa Caused by P23H- Rhodopsin (RHO)

NCT04120883
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Hydroxychloroquine lower dose, Hydroxychloroquine higher dose
Drug
Lead sponsor
University of Michigan
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Oct 26, 2024 · Synced Oct 25, 2026, 10:32 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants

NCT07349394
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VNZ/TEZ/D-IVA, Rosuvastatin
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 55 Years
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Apr 2, 2026 · Synced Oct 25, 2026, 10:32 PM EDT
Not yet recruiting No phase listed Observational

Clinical Validation of AI-Based Quantitative Eye Movement Analysis From Smartphone 9-Gaze Videos (Glandy EOM)

NCT07623837
View directory record Official record
Conditions
Ocular Motility Disorders, Strabismus, Extraocular Muscle Dysfunction, Thyroid Eye Disease
Interventions
Glandy EOM (AI-based quantitative eye movement analysis software)
Device
Lead sponsor
THYROSCOPE INC.
Industry
Eligibility
22 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
San Jose, California
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 25, 2026, 10:32 PM EDT
Recruiting No phase listed Observational

Brain Development Research Program

NCT00305305
View directory record Official record
Conditions
Brain Disorders, Aicardi Syndrome
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2027
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 25, 2026, 10:32 PM EDT