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ClinicalTrials.gov public records Last synced Oct 25, 2026, 1:43 PM EDT

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Showing 241–264 of 2,213 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genes Clear all
Completed Phase 1 Interventional

Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia

NCT00272857
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Retrovirus Construct
Genetic
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Year to 35 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 23, 2017 · Synced Oct 25, 2026, 1:43 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Phase 1/2 Study of KSQ-001EX, Autologous Tumor Infiltrating Lymphocytes Engineered to Inactivate the SOCS1 Gene, in Patients With Select Advanced Solid Tumors

NCT06237881
View directory record Official record
Conditions
Advanced Solid Tumor, Melanoma, Lung Neoplasms, Head and Neck Neoplasms
Interventions
KSQ-001EX, Interleukin-2, Cyclophosphamide, Fludarabine
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years to 70 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Oct 25, 2026, 1:43 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy

NCT02354781
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAV1.CMV.huFollistin344
Biological
Lead sponsor
Jerry R. Mendell
Other
Eligibility
7 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 11, 2023 · Synced Oct 25, 2026, 1:43 PM EDT
Completed Phase 1 Interventional

Durvalumab and Tremelimumab in Treating Patients With Microsatellite Stable Metastatic Colorectal Cancer to the Liver

NCT03005002
View directory record Official record
Conditions
Metastatic Carcinoma in the Liver, MLH1 Gene Mutation, MSH6 Gene Mutation, PMS2 Gene Mutation, Stage IV Colorectal Cancer, Stage IVA Colorectal Cancer, Stage IVB Colorectal Cancer
Interventions
Durvalumab, Laboratory Biomarker Analysis, Tremelimumab
Biological · Other
Lead sponsor
City of Hope Medical Center
Other
Eligibility
19 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Dec 26, 2019 · Synced Oct 25, 2026, 1:43 PM EDT
Completed Not applicable Interventional

Pilot Study of Curcumin for Women With Obesity and High Risk for Breast Cancer

NCT01975363
View directory record Official record
Conditions
Atypical Ductal Breast Hyperplasia, BRCA1 Gene Mutation, BRCA2 Gene Mutation, Ductal Breast Carcinoma in Situ, Lobular Breast Carcinoma in Situ
Interventions
curcumin, Biomarker analysis, Assessment of Dietary Intake, Daily Log
Dietary Supplement · Other
Lead sponsor
Ohio State University Comprehensive Cancer Center
Other
Eligibility
19 Years and older · Female only
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 30, 2019 · Synced Oct 25, 2026, 1:43 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders

NCT04416178
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
13 Years and older
Enrollment
352 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 1, 2026 · Synced Oct 25, 2026, 1:43 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Trial Evaluating Safety, Tolerability and Efficacy of CTX340 in Participants With Hypertension

NCT07758634
View directory record Official record
Conditions
Hypertension
Interventions
CTX340, Placebo
Drug
Lead sponsor
CRISPR Therapeutics AG
Industry
Eligibility
18 Years to 75 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Port Orange, Florida
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Oct 25, 2026, 1:43 PM EDT
Completed Phase 2 Interventional Results available

Olaparib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Defects in DNA Damage Repair Genes (A Pediatric MATCH Treatment Trial)

NCT03233204
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Low Grade Glioma, Malignant Glioma, Recurrent Childhood Central Nervous System Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Osteosarcoma, Refractory Childhood Malignant Germ Cell Tumor, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Glioma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Rhabdoid Tumor, Wilms Tumor
Interventions
Olaparib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
111
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 88 more
Source: ClinicalTrials.gov public record
Updated Dec 11, 2024 · Synced Oct 25, 2026, 1:43 PM EDT
Completed Phase 2 Interventional Results available

Azacitidine and Combination Chemotherapy in Treating Infants With Acute Lymphoblastic Leukemia and KMT2A Gene Rearrangement

NCT02828358
View directory record Official record
Conditions
Acute Leukemia of Ambiguous Lineage, B Acute Lymphoblastic Leukemia, Mixed Phenotype Acute Leukemia
Interventions
Azacitidine, Biospecimen Collection, Computed Tomography, Cyclophosphamide, Cytarabine, Daunorubicin, Daunorubicin Hydrochloride, Dexamethasone, Echocardiography, Hydrocortisone Sodium Succinate, Leucovorin, Leucovorin Calcium, Magnetic Resonance Imaging, Mercaptopurine, Methotrexate, Multigated Acquisition Scan, Pegaspargase, Prednisolone, Thioguanine, Vincristine, Vincristine Sulfate
Drug · Procedure
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 364 Days
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
157
States / cities
Birmingham, Alabama • Mobile, Alabama • Anchorage, Alaska + 128 more
Source: ClinicalTrials.gov public record
Updated Feb 27, 2025 · Synced Oct 25, 2026, 1:43 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency

NCT02991144
View directory record Official record
Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Interventions
scAAV8OTC, Reactive Corticosteroid Taper Regimen, Prophylactic Corticosteroid Taper Regimen
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
4
States / cities
Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 26, 2023 · Synced Oct 25, 2026, 1:43 PM EDT
Active, not recruiting Phase 1 Interventional

Nintedanib and Azacitidine in Treating Participants With HOX Gene Overexpression Relapsed or Refractory Acute Myeloid Leukemia

NCT03513484
View directory record Official record
Conditions
Acute Myeloid Leukemia With t(9;11)(p22.3;q23.3); MLLT3-KMT2A, Fibroblast Growth Factor Basic Form Measurement, FLT3 Internal Tandem Duplication, Recurrent Adult Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Interventions
Azacitidine, Laboratory Biomarker Analysis, Nintedanib
Drug · Other
Lead sponsor
Northwestern University
Other
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced Oct 25, 2026, 1:43 PM EDT
Terminated Phase 2 Interventional

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

NCT06462547
View directory record Official record
Conditions
Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets Type 2
Interventions
INZ-701
Drug
Lead sponsor
Inozyme Pharma
Industry
Eligibility
1 Year and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
2
States / cities
Rochester, Minnesota • Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Oct 19, 2026 · Synced Oct 25, 2026, 1:43 PM EDT
Recruiting Phase 1Phase 2 Interventional

ASC618 Gene Therapy in Hemophilia A Patients

NCT04676048
View directory record Official record
Conditions
Hemophilia A
Interventions
ASC618
Biological
Lead sponsor
ASC Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated Feb 1, 2023 · Synced Oct 25, 2026, 1:43 PM EDT
Completed Phase 3 Interventional Results available

Study of Olaparib (Lynparza™) Versus Enzalutamide or Abiraterone Acetate in Men With Metastatic Castration-Resistant Prostate Cancer (PROfound Study)

NCT02987543
View directory record Official record
Conditions
Metastatic Castration-resistant Prostate Cancer
Interventions
olaparib, enzalutamide, abiraterone acetate
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years · Male only
Enrollment
387 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
35
States / cities
Anchorage, Alaska • Tucson, Arizona • Duarte, California + 30 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2023 · Synced Oct 25, 2026, 1:43 PM EDT
Completed Phase 2 Interventional Results available

Cilengitide, Temozolomide, and Radiation Therapy in Treating Patients With Newly Diagnosed Glioblastoma and Unmethylated Gene Promoter Status

NCT00813943
View directory record Official record
Conditions
Glioblastoma
Interventions
Cilengitide (2-times weekly), cilengitide (5-times weekly), Temozolomide, Radiotherapy
Drug · Radiation
Lead sponsor
EMD Serono
Industry
Eligibility
18 Years and older
Enrollment
265 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Rockland, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 30, 2017 · Synced Oct 25, 2026, 1:43 PM EDT
Not listed No phase listed Observational

Gene-STEPS: Shortening Time of Evaluation in Paediatric Epilepsy Services

NCT06082999
View directory record Official record
Conditions
Epilepsy
Interventions
Not listed
Lead sponsor
Great Ormond Street Hospital for Children NHS Foundation Trust
Other
Eligibility
Up to 12 Months
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 13, 2023 · Synced Oct 25, 2026, 1:43 PM EDT
Not yet recruiting Phase 3 Interventional

A Long-Term Extension (LTE) Study Of Enpatoran In Cutaneous Manifestations Of Lupus With Or Without Systemic Disease [Elowen-LTE]

NCT07840287
View directory record Official record
Conditions
Systemic Lupus Erythematosus (SLE), Cutaneous Lupus Erythematosus (CLE)
Interventions
Enpatoran
Drug
Lead sponsor
EMD Serono Research & Development Institute, Inc.
Industry
Eligibility
18 Years to 76 Years
Enrollment
404 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Billerica, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 25, 2026 · Synced Oct 25, 2026, 1:43 PM EDT
Enrolling by invitation Phase 1 Interventional

Long-Term Follow-Up Study of HIV-1 Infected Adults Who Received EBT-101

NCT05143307
View directory record Official record
Conditions
HIV-1-infection
Interventions
EBT-101
Biological
Lead sponsor
Excision BioTherapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2037
U.S. locations
3
States / cities
San Francisco, California • St Louis, Missouri • Camden, New Jersey
Source: ClinicalTrials.gov public record
Updated May 14, 2024 · Synced Oct 25, 2026, 1:43 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

MAGE-A3/12 Metastatic Cancer Treatment With Anti-MAGE-A3/12 TCR-Gene Engineered Lymphocytes

NCT01273181
View directory record Official record
Conditions
Metastatic Cancer, Metastatic Renal Cancer, Metastatic Melanoma
Interventions
PG13-MAGE-A3 TCR9W11 (anti-MAGE-A3/12 TCR) Transduced Autologous Peripheral Blood Lymphocytes, Aldesleukin, Cyclophosphamide, Fludarabine
Biological · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 28, 2015 · Synced Oct 25, 2026, 1:43 PM EDT
Completed Phase 1 Interventional

Mirvetuximab Soravtansine and Rucaparib Camsylate in Treating Participants With Recurrent Endometrial, Ovarian, Fallopian Tube or Primary Peritoneal Cancer

NCT03552471
View directory record Official record
Conditions
BRCA1 Gene Mutation, BRCA2 Gene Mutation, Folate Receptor Alpha Positive, Recurrent Fallopian Tube Carcinoma, Recurrent Ovarian Carcinoma, Recurrent Primary Peritoneal Carcinoma, Recurrent Uterine Corpus Carcinoma, Recurrent Uterine Serous Carcinoma, Recurrent Uterine Carcinosarcoma, Platinum Resistant Ovarian Cancer
Interventions
Laboratory Biomarker Analysis, Mirvetuximab Soravtansine, Pharmacokinetic Study, Rucaparib Camsylate
Other · Biological · Drug
Lead sponsor
Ohio State University Comprehensive Cancer Center
Other
Eligibility
18 Years and older · Female only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 13, 2025 · Synced Oct 25, 2026, 1:43 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

NCT05419492
View directory record Official record
Conditions
Dravet Syndrome
Interventions
ETX101
Drug
Lead sponsor
Encoded Therapeutics
Industry
Eligibility
6 Months to 17 Years
Enrollment
113 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2034
U.S. locations
11
States / cities
San Francisco, California • Aurora, Colorado • Miami, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2026 · Synced Oct 25, 2026, 1:43 PM EDT
Completed Phase 1Phase 2 Interventional

Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (The EXACT Trial)

NCT04125732
View directory record Official record
Conditions
Coronary Artery Disease, Ischemia, Angina Refractory, Cardiovascular Diseases, Heart Diseases
Interventions
AdVEGFXC1
Biological
Lead sponsor
XyloCor Therapeutics, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
16
States / cities
Stanford, California • Washington D.C., District of Columbia • Daytona Beach, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Jan 30, 2024 · Synced Oct 25, 2026, 1:43 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Metabolic Screening in Patients With Donnai-Barrow Syndrome

NCT01509287
View directory record Official record
Conditions
Donnai-Barrow Syndrome
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
Not listed
Enrollment
78 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 22, 2022 · Synced Oct 25, 2026, 1:43 PM EDT
Withdrawn Not applicable Interventional

Assessment of EGFR Genomic Alterations as Prognostic Markers in Cervical Cancer

NCT00298064
View directory record Official record
Conditions
Cervical Cancer, Cervix
Interventions
Tissue Repository
Genetic
Lead sponsor
University of New Mexico
Other
Eligibility
Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005
U.S. locations
1
States / cities
Albuquerque, New Mexico
Source: ClinicalTrials.gov public record
Updated Jul 5, 2023 · Synced Oct 25, 2026, 1:43 PM EDT