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ClinicalTrials.gov public records Last synced Oct 29, 2026, 8:08 PM EDT

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Showing 1–24 of 1,469 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Mutation Clear all
Withdrawn Phase 1 Interventional

A Study of Fedratinib With IDH Inhibition in Advanced-Phase, IDH-Mutated Ph-Negative Myeloproliferative Neoplasms

NCT04955938
View directory record Official record
Conditions
IDH Mutation, IDH1 Mutation, IDH2 Gene Mutation, Blood Cancer, Myeloproliferative Neoplasm
Interventions
Ivosidenib, Enasidenib, Fedratinib
Drug
Lead sponsor
University of Chicago
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 31, 2024 · Synced Oct 29, 2026, 8:08 PM EDT
Terminated Phase 1 Interventional

Evaluate BL-M17D1 in Patients w/HER2-Expressing/Mutant Advanced or Metastatic Solid Tumors

NCT06714617
View directory record Official record
Conditions
Breast Cancer Stage III, HER2-positive Metastatic Breast Cancer, Unresectable Breast Carcinoma, Her2-Positive, HER2 Gene Mutation, Gastroesophageal-junction Cancer, Non Small Cell Lung Cancer, Endometrial Neoplasms, Peritoneal Cancer, Fallopian Tube Cancer, Ovarian Cancer, Urothelial Carcinoma Bladder, Solid Tumor, Adult, Gastric Cancer
Interventions
BL-M17D1
Drug
Lead sponsor
SystImmune Inc.
Industry
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
9
States / cities
Denver, Colorado • New Haven, Connecticut • Lake Mary, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 29, 2026, 8:08 PM EDT
Completed Phase 2 Interventional Results available

A Study of Sunitinib In Young Patients With Advanced Gastrointestinal Stromal Tumor

NCT01396148
View directory record Official record
Conditions
Gastrointestinal Stromal Tumors
Interventions
sunitinib malate dose escalation, sunitinib malate
Drug
Lead sponsor
Pfizer
Industry
Eligibility
6 Years to 20 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
3
States / cities
Los Angeles, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 27, 2019 · Synced Oct 29, 2026, 8:08 PM EDT
Terminated Phase 1 Interventional

A Study Investigating BG-60366 in Adults With Epidermal Growth Factor Receptor (EGFR)-Mutant Non-Small Cell Lung Cancer

NCT06685718
View directory record Official record
Conditions
Non-Small Cell Lung Cancer, Lung Cancer, NSCLC, NSCLC (Non-small Cell Lung Carcinoma), EGFR Activating Mutation, EGFR Mutation-Related Tumors
Interventions
BG-60366
Drug
Lead sponsor
BeOne Medicines
Industry
Eligibility
18 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
6
States / cities
Denver, Colorado • Boston, Massachusetts • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated May 22, 2026 · Synced Oct 29, 2026, 8:08 PM EDT
Completed Phase 2 Interventional Results available

Dabrafenib and Trametinib in People With BRAF V600E Mutation Positive Lesions in Erdheim Chester Disease

NCT02281760
View directory record Official record
Conditions
BRAF V600E Mutation
Interventions
Dabrafenib Mesylate, Trametinib Dimethyl Sulfoxide
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 80 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 31, 2021 · Synced Oct 29, 2026, 8:08 PM EDT
Completed No phase listed Observational

Awareness and Attitudes Regarding Prenatal and Preimplantation Genetic Diagnosis for Inherited Breast/Ovarian Cancer Risk

NCT00735150
View directory record Official record
Conditions
Breast Cancer, Ovarian Cancer
Interventions
questionnaire, interview
Behavioral
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years to 39 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 11, 2010 · Synced Oct 29, 2026, 8:08 PM EDT
Completed Phase 2 Interventional Results available

Tazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With EZH2, SMARCB1, or SMARCA4 Gene Mutations (A Pediatric MATCH Treatment Trial)

NCT03213665
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Hodgkin Lymphoma, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Hodgkin Lymphoma, Ann Arbor Stage IV Non-Hodgkin Lymphoma, Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Low Grade Glioma, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Hodgkin Lymphoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Refractory Hodgkin Lymphoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Soft Tissue Sarcoma, Rhabdoid Tumor, Stage III Soft Tissue Sarcoma AJCC v7, Stage IV Soft Tissue Sarcoma AJCC v7, Wilms Tumor
Interventions
Tazemetostat
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
115
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 91 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 29, 2026, 8:08 PM EDT
Recruiting Phase 2 Interventional

Sapanisertib and Serabelisib (PIKTOR) With Paclitaxel and a Substudy With an Insulin-Suppressing Diet in Patients With Advanced/Recurrent Endometrial Cancer

NCT06463028
View directory record Official record
Conditions
Endometrial Cancer
Interventions
Sapanisertib, Serabelisib, Paclitaxel
Drug
Lead sponsor
Faeth Therapeutics
Industry
Eligibility
18 Years and older · Female only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
18
States / cities
La Jolla, California • San Francisco, California • Miami Beach, Florida + 15 more
Source: ClinicalTrials.gov public record
Updated Dec 24, 2025 · Synced Oct 29, 2026, 8:08 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

An Intervention to Increase Genetic Testing in Families Who May Share a Gene Mutation Related to Cancer Risk and An Intervention to Help Patients and Their Primary Care Providers Stay Up-to-date About Uncertain Genetic Test Results

NCT05420064
View directory record Official record
Conditions
BRCA1 Mutation, POLD1 Gene Mutation, CDKN2A Mutation, BRCA2 Mutation, POLE Gene Mutation, APC Gene Mutation, ATM Gene Mutation, MLH1 Gene Mutation, BARD1 Gene Mutation, MSH2 Gene Mutation, BRIP1 Gene Mutation, MSH6 Gene Mutation, CHEK2 Gene Mutation, PMS2 Gene Mutation, PALB2 Gene Mutation, EPCAM Gene Mutation, RAD51C Gene Mutation, BMPR1A Gene Mutation, RAD51D Gene Mutation, SMAD4, PTEN Gene Mutation, GREM1
Interventions
Intervention Arm At-risk Relative/ARR Contacts, MyGene Portal, Standard of Care
Behavioral
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
25 Years and older
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
8
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Oct 29, 2026, 8:08 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Cause of Pigment Dispersion Syndrome

NCT00005919
View directory record Official record
Conditions
Glaucoma, Healthy, Pigment Dispersion Syndrome
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 29, 2026, 8:08 PM EDT
Completed Phase 1 Interventional

An Umbrella, Modular Study Based on Epidermal Growth Factor Receptor (EGFR) Mutation Status

NCT00903734
View directory record Official record
Conditions
Advanced Cancers
Interventions
Erlotinib Hydrochloride (Tarceva)
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 13, 2015 · Synced Oct 29, 2026, 8:08 PM EDT
Terminated Phase 2 Interventional

Poziotinib in EGFR Exon 20 Mutant Advanced NSCLC

NCT03066206
View directory record Official record
Conditions
EGFR Exon 20 Mutation, ERBB2 Gene Mutation, Recurrent Lung Non-Small Cell Carcinoma, Stage IV Non-Small Cell Lung Cancer AJCC v7
Interventions
Poziotinib
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
93 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 20, 2026 · Synced Oct 29, 2026, 8:08 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of BH-30643 in Subjects With Locally Advanced or Metastatic NSCLC Harboring EGFR and/or HER2 Mutations

NCT06706076
View directory record Official record
Conditions
NSCLC (Advanced Non-small Cell Lung Cancer)
Interventions
BH-30643, BH-30643 combo therapy with Carboplatin/Pemetrexed
Drug
Lead sponsor
BlossomHill Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
675 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
24
States / cities
Phoenix, Arizona • Irvine, California • La Jolla, California + 18 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Oct 29, 2026, 8:08 PM EDT
Completed Phase 2 Interventional Results available

Adavosertib Plus Chemotherapy in Platinum-Resistant Epithelial Ovarian, Fallopian Tube, or Primary Peritoneal Cancer

NCT02272790
View directory record Official record
Conditions
Ovarian, Fallopian Tube, Peritoneal Cancer, P53 Mutation
Interventions
Adavosertib, Paclitaxel, Carboplatin, Gemcitabine, PLD
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years · Female only
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
18
States / cities
Gilbert, Arizona • Tucson, Arizona • La Jolla, California + 14 more
Source: ClinicalTrials.gov public record
Updated Oct 3, 2023 · Synced Oct 29, 2026, 8:08 PM EDT
Completed Phase 2 Interventional Results available

A Study Osimertinib in Patients With Stage 4 Non-small Cell Lung Cancer With Uncommon EGFR Mutations

NCT03434418
View directory record Official record
Conditions
Non Small Cell Lung Cancer
Interventions
osimertinib
Drug
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
3
States / cities
Durham, North Carolina • Columbus, Ohio • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 17, 2023 · Synced Oct 29, 2026, 8:08 PM EDT
Completed No phase listed Observational

Genetic Mutations in Blood and Tissue Samples in Predicting Response to Treatment in Patients With Locally Advanced Rectal Cancer Undergoing Chemoradiation

NCT02132858
View directory record Official record
Conditions
Mucinous Adenocarcinoma of the Rectum, Recurrent Rectal Cancer, Signet Ring Adenocarcinoma of the Rectum, Stage IIA Rectal Cancer, Stage IIB Rectal Cancer, Stage IIC Rectal Cancer, Stage IIIA Rectal Cancer, Stage IIIB Rectal Cancer, Stage IIIC Rectal Cancer
Interventions
cytology specimen collection procedure, laboratory biomarker analysis
Other
Lead sponsor
Fox Chase Cancer Center
Other
Eligibility
18 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 2, 2024 · Synced Oct 29, 2026, 8:08 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation

NCT02516410
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-661 plus ivacaftor combination, Ivacaftor, Placebo (matched to VX-661 plus ivacaftor combination), Placebo (matched to ivacaftor)
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
168 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
22
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 19 more
Source: ClinicalTrials.gov public record
Updated Jun 12, 2018 · Synced Oct 29, 2026, 8:08 PM EDT
Active, not recruiting Phase 2 Interventional

(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis

NCT03731260
View directory record Official record
Conditions
Indolent Systemic Mastocytosis
Interventions
Avapritinib, Placebo
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
251 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
19
States / cities
Birmingham, Alabama • Phoenix, Arizona • Stanford, California + 15 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 29, 2026, 8:08 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS

NCT07743268
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis (ALS)
Interventions
nL-TARDB-006
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
Not listed
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
2
States / cities
St Louis, Missouri • New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Oct 29, 2026, 8:08 PM EDT
Active, not recruiting Phase 2 Interventional

Talazoparib in Treating Patients With Recurrent, Refractory, Advanced, or Metastatic Cancers and Alterations in the BRCA Genes

NCT02286687
View directory record Official record
Conditions
Advanced Malignant Neoplasm, ATM Gene Mutation, BRCA1 Gene Mutation, BRCA2 Gene Mutation, Metastatic Malignant Neoplasm, PALB2 Gene Mutation, Recurrent Malignant Neoplasm, Refractory Malignant Neoplasm
Interventions
Laboratory Biomarker Analysis, Pharmacological Study, Talazoparib
Other · Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2026
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 12, 2026 · Synced Oct 29, 2026, 8:08 PM EDT
Recruiting No phase listed Observational

Longitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum Disorder

NCT06190626
View directory record Official record
Conditions
Zellweger Spectrum Disorder
Interventions
Not listed
Lead sponsor
McGill University Health Centre/Research Institute of the McGill University Health Centre
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 29, 2026, 8:08 PM EDT
Completed Phase 1 Interventional

A Phase I/IB Trial of MEK162 in Combination With Erlotinib in NSCLC Harboring KRAS or EGFR Mutation

NCT01859026
View directory record Official record
Conditions
Lung Cancer, Non-Small Cell Lung Cancer
Interventions
MEK162, Erlotinib
Drug
Lead sponsor
H. Lee Moffitt Cancer Center and Research Institute
Other
Eligibility
18 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jan 19, 2023 · Synced Oct 29, 2026, 8:08 PM EDT
Completed Phase 3 Interventional Results available

A Study of ASP2215 (Gilteritinib) by Itself, ASP2215 Combined With Azacitidine or Azacitidine by Itself to Treat Adult Patients Who Have Recently Been Diagnosed With Acute Myeloid Leukemia With a FLT3 Gene Mutation and Who Cannot Receive Standard Chemotherapy

NCT02752035
View directory record Official record
Conditions
Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation
Interventions
gilteritinib, azacitidine
Drug
Lead sponsor
Astellas Pharma Global Development, Inc.
Industry
Eligibility
18 Years and older
Enrollment
183 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
12
States / cities
Los Angeles, California • Orange, California • Chicago, Illinois + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Oct 29, 2026, 8:08 PM EDT
Recruiting No phase listed Observational

Study of Individuals and Families With Aberrations in DDX41 or Similar Cancer Predisposition Variants

NCT07019155
View directory record Official record
Conditions
Germline Mutation, Myelodysplastic Syndromes, Acute Myeloid Leukemia
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month to 120 Years
Enrollment
510 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 29, 2026, 8:08 PM EDT