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ClinicalTrials.gov public records Last synced Oct 29, 2026, 11:57 PM EDT

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Showing 97–120 of 941 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Mutations Clear all
Recruiting Phase 2 Interventional

A Study of Selinexor in People With Wilms Tumors and Other Solid Tumors

NCT05985161
View directory record Official record
Conditions
Wilms Tumor, Rhabdoid Tumor, Malignant Peripheral Nerve Sheath Tumors, MPNST, Nephroblastoma, XPO1 Gene Mutation, Solid Tumor
Interventions
Selinexor
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
12 Months and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
16
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 13 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2026 · Synced Oct 29, 2026, 11:57 PM EDT
Active, not recruiting Phase 1 Interventional

Study of LY3410738 Administered to Patients With Advanced Solid Tumors With IDH1 or IDH2 Mutations

NCT04521686
View directory record Official record
Conditions
Cholangiocarcinoma, Chondrosarcoma, Glioma, Any Solid Tumor
Interventions
LY3410738, Gemcitabine, Cisplatin, Durvalumab
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
18 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
17
States / cities
Phoenix, Arizona • Tucson, Arizona • Los Angeles, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 29, 2026, 11:57 PM EDT
Completed Phase 3 Interventional Results available

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation

NCT00909727
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
6 Years to 11 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
17
States / cities
Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 13 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2012 · Synced Oct 29, 2026, 11:57 PM EDT
Completed Phase 1 Interventional

A Dose-escalation, Expansion Study of ARX788, in Advanced Solid Tumors Subjects With HER2 Expression (ACE-Pan Tumor 01)

NCT03255070
View directory record Official record
Conditions
Breast Neoplasms, Gastric Neoplasm, Solid Tumors
Interventions
ARX788
Drug
Lead sponsor
Ambrx, Inc.
Industry
Eligibility
18 Years and older
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
5
States / cities
Los Angeles, California • Santa Monica, California • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2024 · Synced Oct 29, 2026, 11:57 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Olaparib in Treating Patients With Metastatic Biliary Tract Cancer With Aberrant DNA Repair Gene Mutations

NCT04042831
View directory record Official record
Conditions
Bile Duct Adenocarcinoma, Fanconi Anemia Complementation Group Gene Mutation, Metastatic Bile Duct Carcinoma, PTEN Gene Deletion
Interventions
Biospecimen Collection, Computed Tomography, Magnetic Resonance Imaging, Olaparib
Procedure · Drug
Lead sponsor
Academic and Community Cancer Research United
Other
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
5
States / cities
Scottsdale, Arizona • Jacksonville, Florida • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 29, 2026, 11:57 PM EDT
Active, not recruiting Phase 3 Interventional

REZILIENT3 (REsearching ZIpaLertinib In Egfr Non-small Cell Lung Cancer Tumors)

NCT05973773
View directory record Official record
Conditions
Advanced or Metastatic NSCLS With Exon 20 Insertion Mutation
Interventions
TAS6417
Drug
Lead sponsor
Taiho Oncology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
285 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
9
States / cities
Henderson, Nevada • Las Vegas, Nevada • Canton, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2026 · Synced Oct 29, 2026, 11:57 PM EDT
Recruiting Phase 2 Interventional

Testing Osimertinib as a Treatment for Lung Cancers With an EGFR Exon 20 Change

NCT03191149
View directory record Official record
Conditions
Advanced Lung Non-Small Cell Carcinoma, Recurrent Lung Non-Small Cell Carcinoma, Stage IIIB Lung Non-Small Cell Cancer AJCC v7, Stage IV Lung Non-Small Cell Cancer AJCC v7
Interventions
Biospecimen Collection, Computed Tomography with Contrast, Echocardiography Test, Magnetic Resonance Imaging, Multigated Acquisition Scan, Osimertinib
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
347
States / cities
Anchorage, Alaska • Fairbanks, Alaska • Fort Smith, Arkansas + 238 more
Source: ClinicalTrials.gov public record
Updated Oct 28, 2026 · Synced Oct 29, 2026, 11:57 PM EDT
Completed No phase listed Observational

Molecular Mechanisms and Diagnosis of Mastocytosis

NCT00336076
View directory record Official record
Conditions
Mastocytosis
Interventions
Collection of blood and bone marrow
Other
Lead sponsor
University of Michigan
Other
Eligibility
Not listed
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2009
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 7, 2016 · Synced Oct 29, 2026, 11:57 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)

NCT03210714
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Medulloblastoma, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Childhood Osteosarcoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Neuroblastoma, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Refractory Childhood Malignant Germ Cell Tumor, Refractory Childhood Osteosarcoma, Refractory Childhood Rhabdomyosarcoma, Refractory Childhood Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Wilms Tumor
Interventions
Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan, Computed Tomography, Erdafitinib, Laboratory Biomarker Analysis, Magnetic Resonance Imaging, Pharmacological Study, Positron Emission Tomography, Radionuclide Imaging, X-Ray Imaging
Procedure · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
114
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 91 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 29, 2026, 11:57 PM EDT
Approved For Marketing No phase listed Expanded access

Expanded Access Study of Gilteritinib (ASP2215) in Patients With FMS-like Tyrosine Kinase 3 (FLT3) Mutated Relapsed or Refractory Acute Myeloid Leukemia (AML) or FLT3-Mutated AML in Complete Remission (CR) With Minimal Residual Disease (MRD)

NCT03070093
View directory record Official record
Conditions
Acute Myeloid Leukemia (AML), FMS-like Tyrosine Kinase-3 (FLT3) Mutations
Interventions
gilteritinib
Drug
Lead sponsor
Astellas Pharma Global Development, Inc.
Industry
Eligibility
18 Years and older
U.S. locations
32
States / cities
Los Angeles, California • Aurora, Colorado • Pembroke Pines, Florida + 23 more
Source: ClinicalTrials.gov public record
Updated Apr 20, 2021 · Synced Oct 29, 2026, 11:57 PM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate Multiple Doses of GLPG2222 in Adult Subjects With Cystic Fibrosis

NCT03119649
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
GLPG2222 50 mg, GLPG2222 100 mg, Placebo, GLPG2222 200 mg, GLPG2222 400 mg
Drug
Lead sponsor
Lakefront Biotherapeutics NV
Industry
Eligibility
18 Years to 99 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
7
States / cities
Chatom, Alabama • Little Rock, Arkansas • Orlando, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 16, 2018 · Synced Oct 29, 2026, 11:57 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene

NCT04123626
View directory record Official record
Conditions
Autosomal Dominant Retinitis Pigmentosa, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Disease, Retinitis, Vision Tunnel, Vision Disorders
Interventions
QR-1123, Sham procedure
Drug · Other
Lead sponsor
ProQR Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
5
States / cities
Aurora, Colorado • Gainesville, Florida • Lexington, Kentucky + 2 more
Source: ClinicalTrials.gov public record
Updated May 6, 2022 · Synced Oct 29, 2026, 11:57 PM EDT
Completed No phase listed Observational

Detecting EGFR T790M Mutations From Circulating Tumor Cells

NCT01734915
View directory record Official record
Conditions
Non Small Cell Lung Cancer
Interventions
Circulating tumor cell chip
Device
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
4
States / cities
Boston, Massachusetts • New York, New York • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 26, 2016 · Synced Oct 29, 2026, 11:57 PM EDT
Completed Phase 1 Interventional

Pacritinib and Chemotherapy in Treating Patients With Acute Myeloid Leukemia and FLT3 Mutations

NCT02323607
View directory record Official record
Conditions
Recurrent Adult Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, Therapy-Related Acute Myeloid Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions
Pacritinib, Cytarabine, Daunorubicin Hydrochloride, Decitabine, Laboratory Biomarker Analysis, Pharmacological Study
Drug · Other
Lead sponsor
Bhavana Bhatnagar
Other
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 25, 2019 · Synced Oct 29, 2026, 11:57 PM EDT
Enrolling by invitation No phase listed Observational

Autosomal Dominant Polycystic Kidney Disease Somatic Mutation Biorepository

NCT03901521
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney Disease
Interventions
Not listed
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Oct 29, 2026, 11:57 PM EDT
Completed Phase 2 Interventional

A Study Evaluating the Safety and Efficacy of ENV-101 (Taladegib) in Patients With Advanced Solid Tumors Harboring PTCH1 Loss of Function Mutations

NCT05199584
View directory record Official record
Conditions
Solid Tumors With PTCH1 Loss-of-function Mutations
Interventions
ENV-101 (taladegib)
Drug
Lead sponsor
Endeavor Biomedicines, Inc.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
16
States / cities
Los Angeles, California • Santa Rosa, California • Tampa, Florida + 13 more
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Oct 29, 2026, 11:57 PM EDT
Recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)

NCT07215416
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
Antisense oligonucleotide targeting the ATM gene
Drug
Lead sponsor
Timothy Yu
Other
Eligibility
0 Years to 17 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2036
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 29, 2026, 11:57 PM EDT
Completed Phase 2 Interventional Results available

Neurobiology of a Mutation in Glycine Metabolism in Psychotic Disorders

NCT01720316
View directory record Official record
Conditions
Schizo-affective Disorder, Bipolar Disorder
Interventions
Glycine, placebo
Drug
Lead sponsor
Mclean Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Belmont, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 19, 2017 · Synced Oct 29, 2026, 11:57 PM EDT
Withdrawn No phase listed Observational

A Natural History Study in Pediatric Participants With Hearing Loss Due to OTOF, GJB2, or GJB2/GJB6 Mutations

NCT06019481
View directory record Official record
Conditions
Congenital Hearing Loss Secondary to Biallelic Mutations in the Otoferlin Gene (OTOF), Biallelic Mutations in the Gap Junction Beta 2 (GJB2) Gene, Digenic Mutations in GJB2/Gap Junction Beta 6 (GJB6) Genes
Interventions
Not listed
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
Up to 7 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
2
States / cities
Phoenix, Arizona • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated May 15, 2025 · Synced Oct 29, 2026, 11:57 PM EDT
Completed Phase 3 Interventional Results available

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation

NCT00909532
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
167 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
39
States / cities
Birmingham, Alabama • Oakland, California • Palo Alto, California + 34 more
Source: ClinicalTrials.gov public record
Updated Jan 18, 2013 · Synced Oct 29, 2026, 11:57 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

LRRK2, GBA and Other Genetic Biomarkers in Eastern European (Ashkenazi) Jews With and Without Parkinson's Disease

NCT04668898
View directory record Official record
Conditions
Parkinson Disease, LRRK2, GBA Gene Mutation
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
30 Years and older
Enrollment
203 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 16, 2025 · Synced Oct 29, 2026, 11:57 PM EDT
Completed Phase 1Phase 2 Interventional

Study of WNT974 in Combination With LGX818 and Cetuximab in Patients With BRAF-mutant Metastatic Colorectal Cancer (mCRC) and Wnt Pathway Mutations

NCT02278133
View directory record Official record
Conditions
Metastatic Colorectal Cancer
Interventions
WNT974, LGX818, Cetuximab
Drug · Biological
Lead sponsor
Array BioPharma
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
4
States / cities
New York, New York • Charleston, South Carolina • Houston, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2017 · Synced Oct 29, 2026, 11:57 PM EDT
Completed Phase 2 Interventional

Gefitinib in Treating Patients With Previously Untreated Stage IIIB or Stage IV Non-Small Cell Lung Cancer

NCT00411047
View directory record Official record
Conditions
Lung Cancer
Interventions
gefitinib
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 14, 2013 · Synced Oct 29, 2026, 11:57 PM EDT
Terminated Phase 2 Interventional Results available

Targeted Therapy in Treating Patients With Incurable Non-Small Cell Lung Cancer With Genetic Mutations

NCT02949843
View directory record Official record
Conditions
EGFR Activating Mutation, Recurrent Non-Small Cell Lung Carcinoma, Stage IV Non-Small Cell Lung Cancer
Interventions
Chemotherapy, Immunotherapy, Laboratory Biomarker Analysis, Nivolumab, Pembrolizumab, Targeted Molecular Therapy, Tyrosine Kinase Inhibitor
Drug · Biological · Other
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
Not listed
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 28, 2024 · Synced Oct 29, 2026, 11:57 PM EDT