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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Genetic Mutations Clear all
Completed Phase 2 Interventional Results available

Selumetinib in Cancers With BRAF Mutations

NCT00888134
View directory record Official record
Conditions
Adult Solid Neoplasm
Interventions
Selumetinib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
5
States / cities
Boston, Massachusetts • Charlestown, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 14, 2016 · Synced Jul 21, 2026, 9:22 PM EDT
Completed Phase 1 Interventional

A Study of ZN-e4 in Subjects With Epidermal Growth Factor Receptor Mutated Non-Small Cell Lung Cancer

NCT03446417
View directory record Official record
Conditions
Carcinoma, Non-Small-Cell Lung
Interventions
ZN-e4
Drug
Lead sponsor
Zeno Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
5
States / cities
Duarte, California • Detroit, Michigan • East Setauket, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 24, 2024 · Synced Jul 21, 2026, 9:22 PM EDT
Completed Phase 1 Interventional

1336GCC: Study of Erwinaze for Treatment of Acute Myeloid Leukemia (AML)

NCT02283190
View directory record Official record
Conditions
Acute Myeloid Leukemia
Interventions
Erwinase
Drug
Lead sponsor
Ashkan Emadi
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 11, 2018 · Synced Jul 21, 2026, 9:22 PM EDT
Suspended Phase 2 Interventional

Study of Chemotherapy Plus Ipatasertib for People With Solid Tumors With PTEN/AKT Mutations, A ComboMATCH Treatment Trial

NCT05554380
View directory record Official record
Conditions
Locally Advanced Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm, Unresectable Malignant Solid Neoplasm
Interventions
Biopsy Procedure, Biospecimen Collection, Computed Tomography, Ipatasertib, Magnetic Resonance Imaging, Paclitaxel
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
207
States / cities
Birmingham, Alabama • Mobile, Alabama • Anchorage, Alaska + 167 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Jul 21, 2026, 9:22 PM EDT
Terminated Phase 3 Interventional Results available

Study of Ataluren (PTC124) in Cystic Fibrosis

NCT02107859
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
7
States / cities
Birmingham, Alabama • Long Beach, California • Aurora, Colorado + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2020 · Synced Jul 21, 2026, 9:22 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 8, 2017 · Synced Jul 21, 2026, 9:22 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional Accepts healthy volunteers

Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia. A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation. Master Protocol DIAN-TU-001

NCT01760005
View directory record Official record
Conditions
Alzheimers Disease, Dementia, Alzheimers Disease, Familial
Interventions
Gantenerumab, Solanezumab, Matching Placebo (Gantenerumab), Matching Placebo (Solanezumab), E2814, Lecanemab, Matching Placebo (E2814)
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 80 Years
Enrollment
490 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2028
U.S. locations
12
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 12, 2026 · Synced Jul 21, 2026, 9:22 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

RASopathy Biorepository

NCT04395495
View directory record Official record
Conditions
RAS Mutation, Neurofibromatosis 1, Noonan Syndrome, Noonan Syndrome With Multiple Lentigines, Noonan Neurofibromatosis Syndrome, Cardiofaciocutaneous Syndrome, Costello Syndrome, Legius Syndrome, Smith-Kingsmore Syndrome, MTOR Gene Mutation, GATOR-1 Gene Mutation, SYNGAP1-Related Intellectual Disability, DLG4, MAPK1 Gene Mutation
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2065
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Jul 21, 2026, 9:22 PM EDT
Completed Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia. A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation.

NCT04623242
View directory record Official record
Conditions
Alzheimers Disease, Dementia, Alzheimers Disease, Familial
Interventions
Gantenerumab, Solanezumab, Matching Placebo (Gantenerumab), Matching Placebo (Solanezumab)
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 80 Years
Enrollment
194 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2020
U.S. locations
10
States / cities
Birmingham, Alabama • La Jolla, California • New Haven, Connecticut + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2022 · Synced Jul 21, 2026, 9:22 PM EDT
Not yet recruiting Phase 2 Interventional

Zipalertinib With Carboplatin and Pemetrexed for the Treatment of Resectable, Stage II-IIIB, Non-Small Cell Lung Cancer

NCT07229339
View directory record Official record
Conditions
Lung Non-Squamous Non-Small Cell Carcinoma
Interventions
Biospecimen Collection, Carboplatin, Computed Tomography, Magnetic Resonance Imaging, Pemetrexed, Positron Emission Tomography, Zipalertinib
Procedure · Drug
Lead sponsor
Jonsson Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2030
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Jul 21, 2026, 9:22 PM EDT
Terminated No phase listed Observational

Genetic Study of Brain Tumors in Young Children

NCT00010101
View directory record Official record
Conditions
Central Nervous System Tumor, Pediatric
Interventions
Not listed
Lead sponsor
Pediatric Brain Tumor Consortium
Network
Eligibility
Up to 3 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
4
States / cities
Washington D.C., District of Columbia • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2011 · Synced Jul 21, 2026, 9:22 PM EDT
Recruiting Phase 2 Interventional

Tofersen in Non-SOD1 ALS

NCT07294144
View directory record Official record
Conditions
ALS (Amyotrophic Lateral Sclerosis)
Interventions
Tofersen
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
3
States / cities
Atlanta, Georgia • Boston, Massachusetts • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Jul 21, 2026, 9:22 PM EDT
Completed Phase 3 Interventional Results available

Phase III Trial of Coenzyme Q10 in Mitochondrial Disease

NCT00432744
View directory record Official record
Conditions
Mitochondrial Diseases
Interventions
CoenzymeQ10, Placebo
Drug
Lead sponsor
University of Florida
Other
Eligibility
12 Months to 17 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
2
States / cities
Cincinnati, Ohio • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 10, 2017 · Synced Jul 21, 2026, 9:22 PM EDT
Recruiting Phase 2 Interventional

Zanubrutinib in Patients With DLBCL and MYD88 or NOTCH1 Mutation or CD5+

NCT06846463
View directory record Official record
Conditions
Diffuse Large B-cell Lymphoma, DLBCL - Diffuse Large B Cell Lymphoma
Interventions
Zanubrutinib
Drug
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Jul 21, 2026, 9:22 PM EDT
Not listed No phase listed Observational

Noninvasive Prenatal Diagnosis: Using Fetal Cells From Maternal Blood

NCT00064597
View directory record Official record
Conditions
Chromosome Disorders
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
16 Years to 45 Years · Female only
Enrollment
3,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1987 – 2003
U.S. locations
5
States / cities
Chicago, Illinois • Boston, Massachusetts • Detroit, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 21, 2026, 9:22 PM EDT
Recruiting Phase 1 Interventional

Ulixertinib/Palbociclib in Patients With Advanced Pancreatic and Other Solid Tumors

NCT03454035
View directory record Official record
Conditions
Tumor, Solid, Pancreatic Cancer, Melanoma
Interventions
Ulixertinib, Palbociclib
Drug
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Years to 99 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced Jul 21, 2026, 9:22 PM EDT
Terminated Phase 4 Interventional Results available

Boceprevir Drug Combination for Hepatitis C Treatment in People With and Without HIV

NCT01443923
View directory record Official record
Conditions
Hepatitis, HIV/AIDS
Interventions
Boceprevir, Peg-Interferon-alfa 2B, Ribavirin
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 12, 2015 · Synced Jul 21, 2026, 9:22 PM EDT
Completed No phase listed Observational

Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT

NCT02575430
View directory record Official record
Conditions
Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP)
Interventions
No treatment: retrospective chart review
Other
Lead sponsor
QLT Inc.
Industry
Eligibility
8 Years and older
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Baltimore, Maryland • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Apr 28, 2016 · Synced Jul 21, 2026, 9:22 PM EDT
Terminated Phase 1 Interventional

A Study of ASP8273 in Subjects With Non-small Cell Lung Cancer (NSCLC) Harboring Epidermal Growth Factor Receptor (EGFR) Mutations

NCT03082300
View directory record Official record
Conditions
Non-small Cell Lung Cancer (NSCLC), Epidermal Growth Factor Receptor (EGFR) Mutations
Interventions
naquotinib
Drug
Lead sponsor
Astellas Pharma Global Development, Inc.
Industry
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
3
States / cities
Beverly Hills, California • The Woodlands, Texas • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Nov 19, 2024 · Synced Jul 21, 2026, 9:22 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Adaptation to Living With a BRCA1/2 Mutation

NCT01866865
View directory record Official record
Conditions
Cancer Genetics
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
142 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 4, 2018 · Synced Jul 21, 2026, 9:22 PM EDT
Withdrawn No phase listed Observational

Anonymous Testing of Pathology Specimens for BRCA Mutations in Ashkenazi Jewish Individuals Who Have Cancer

NCT00588263
View directory record Official record
Conditions
Extrahepatic Bile Duct Cancer, Gallbladder Cancer, Gastric Cancer, Lung Cancer, Melanoma, Non-Hodgkin's Lymphoma, Uterine Cancer, CORPUS UTERI,ENDOMETRIUM, LUNG, OVARY
Interventions
Not listed
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2018
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 18, 2018 · Synced Jul 21, 2026, 9:22 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Natural Killer Cells Before and After Donor Stem Cell Transplant in Treating Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelogenous Leukemia

NCT01904136
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia With Gene Mutations, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, de Novo Myelodysplastic Syndrome, Myelodysplastic Syndrome, Recurrent Acute Myeloid Leukemia, Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Therapy-Related Acute Myeloid Leukemia
Interventions
Allogeneic Hematopoietic Stem Cell Transplantation, Bone Marrow Transplantation, Cyclophosphamide, Fludarabine, Laboratory Biomarker Analysis, Melphalan, Mycophenolate Mofetil, Natural Killer Cell Therapy, Peripheral Blood Stem Cell Transplantation, Tacrolimus, Total-Body Irradiation
Procedure · Drug · Other + 2 more
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
2 Years to 65 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 23, 2024 · Synced Jul 21, 2026, 9:22 PM EDT
Completed Phase 1 Interventional Results available

(NAVIGATOR) Study of BLU-285 in Patients With Gastrointestinal Stromal Tumors (GIST) and Other Relapsed and Refractory Solid Tumors

NCT02508532
View directory record Official record
Conditions
Gastrointestinal Stromal Tumors (GIST), Other Relapsed or Refractory Solid Tumors
Interventions
Avapritinib
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
9
States / cities
Scottsdale, Arizona • Santa Monica, California • Miami, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 20, 2022 · Synced Jul 21, 2026, 9:22 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Hereditary Gastric Cancer Syndromes: An Integrated Genomic and Clinicopathologic Study of the Predisposition to Gastric Cancer

NCT03030404
View directory record Official record
Conditions
Stomach Neoplasms, Stomach Cancer, Gastric Adenocarcinoma and Proximal Polyposis of the Stomach (GAPPS), Hereditary Diffuse Gastric Cancer (HDGC), Familial Diffuse Gastric Cancer
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years and older
Enrollment
733 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Jul 21, 2026, 9:22 PM EDT