Clinical trials: Gene Mutations
Anemia, ASXL1 Gene Mutation
Allogeneic Stem Cell Transplantation for Multiple Myeloma and Myelofibrosis
This phase II trial studies how well busulfan, fludarabine, donor stem cell transplant, and cyclophosphamide in treating participants with multiple myeloma or myelofibrosis.
- Conditions
- Anemia, ASXL1 Gene Mutation, EZH2 Gene Mutation, IDH1 Gene Mutation
- Trial name
- NCT03303950
- Testing
- Busulfan, Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Utah
Recurrent Lung Squamous Cell Carcinoma, Stage IV Lung Squamous Cell Carcinoma AJCC v7
Sapanisertib in Treating Patients With Stage IV or Recurrent Lung Cancer
This phase II trial studies how well sapanisertib works in treating patients with lung cancer that is stage IV or has come back (recurrent) and has a mutation in the NFE2L2, KEAP-1, or KRAS gene.
- Trial name
- NCT02417701
- Testing
- Laboratory Biomarker Analysis, Pharmacological Study, Sapanisertib
- Where
- 7 sites in United States
- Volunteers
- 34 people
- Sponsor
- National Cancer Institute (NCI)
KRAS Gene Mutation, Metastatic Non-Squamous Non-Small Cell Lung Carcinoma
Pembrolizumab and Trametinib in Treating Patients With Stage IV Non-Small Cell Lung Cancer and KRAS Gene Mutations
This phase Ib trial studies the side effects of pembrolizumab and trametinib in treating patients with non-small cell lung cancer and KRAS gene mutations that has spread to other places in the body.
- Conditions
- KRAS Gene Mutation, Metastatic Non-Squamous Non-Small Cell Lung Carcinoma, Recurrent Non-Squamous Non-Small Cell Lung Carcinoma, Stage IV Non-Small Cell Lung Cancer AJCC v7
- Trial name
- NCT03299088
- Testing
- Pembrolizumab, Trametinib
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Jonathan Riess
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- RESCUE (NCT02652767)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 39 people
- Sponsor
- GenSight Biologics
Waldenstrom Macroglobulinemia, MYD88 Gene Mutation
Ibrutinib + Venetoclax in Untreated WM
This study evaluates the safety and efficacy of Ibrutinib combined with Venetoclax (IVEN) in the treatment of adults diagnosed with Waldenstrom's macroglobulinemia (WM) cancer with a specific MYD88 gene mutation.
- Trial name
- NCT04273139
- Testing
- IBRUTINIB, Venetoclax
- Where
- 2 sites in United States
- Volunteers
- 45 people
- Sponsor
- Dana-Farber Cancer Institute
Prostate Cancer
Prostate Cancer Screening in Men With Germline BRCA2 Mutations
The purpose of this study is to determine the incidence of prostate cancer in men with the BRCa2 gene mutation as an independent indicator for prostate cancer screening.
- Trial name
- NCT02154672
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Yale University
Relapsed Ovarian Cancer, BRCA Mutation, Platinum Sensitivity
Olaparib Treatment in Relapsed Germline Breast Cancer Susceptibility Gene (BRCA) Mutated Ovarian Cancer Patients Who Have Progressed at Least 6 Months After Last Platinum Treatment and Have Received at Least 2 Prior Platinum Treatments
Comparison of olaparib vs. physician's choice of single agent standard of care non-platinum based chemotherapy in patients with germline Breast Cancer susceptibility gene (gBRCA) mutated ovarian cancer who have progressed at…
- Trial name
- SOLO3 (NCT02282020)
- Testing
- OLAPARIB, Single agent chemotherapy
- Where
- 94 sites in United States, Argentina and 11 more
- Volunteers
- 266 people
- Sponsor
- AstraZeneca
Neoplasms, Breast, Carcinoma of Breast
A Phase III Trial of Niraparib Versus Physician's Choice in HER2 Negative, Germline BRCA Mutation-positive Breast Cancer Patients
The purpose of this study is to compare progression-free survival (PFS) in patients with advanced/metastatic breast cancer who have a BRCA mutation when treated with niraparib as compared to those treated with physician's choice
- Conditions
- Neoplasms, Breast, Carcinoma of Breast, Human Epidermal Growth Factor 2 Negative Carcinoma of Breast, BRCA1 Gene Mutation
- Trial name
- BRAVO (NCT01905592)
- Testing
- niraparib, Physician's choice
- Where
- 104 sites in United States, Belgium and 12 more
- Volunteers
- 216 people
- Sponsor
- Tesaro, Inc.
Carcinoma, Non-Small-Cell Lung
Study of Capmatinib and Spartalizumab/Placebo in Advanced NSCLC Patients With MET Exon 14 Skipping Mutations
A double-blind, placebo controlled, randomized, phase II study evaluating the efficacy and safety of capmatinib (INC280) and spartalizumab (PDR001) combination therapy versus capmatinib and placebo as first line treatment for…
- Trial name
- NCT04323436
- Testing
- Spartalizumab, Capmatinib
- Where
- 15 sites in United States, Belgium and 7 more
- Volunteers
- 31 people
- Sponsor
- Novartis Pharmaceuticals
Advanced Cholangiocarcinoma, FGFR2 Gene Mutation
Phase 3 Study of BGJ398 (Oral Infigratinib) in First Line Cholangiocarcinoma With FGFR2 Gene Fusions/Translocations
Infigratinib is an oral drug which selectively binds to fibroblast growth factor receptor (FGFR) 2 and is being developed to treat participants with FGFR2 mutated cholangiocarcinoma.
- Trial name
- NCT03773302
- Testing
- BGJ398, Gemcitabine, Cisplatin
- Where
- 116 sites in United States, Australia and 13 more
- Volunteers
- 48 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Cystic Fibrosis
Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation
The purpose of this study is to provide information regarding the long-term safety and pharmacodynamics of ivacaftor treatment in the pediatric population younger than 6 years of age with Cystic Fibrosis (CF) who have a CFTR…
- Trial name
- NCT01946412
- Testing
- Ivacaftor
- Where
- 16 sites in United States, Canada and 1 more
- Volunteers
- 33 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Malignant Melanoma
Lapatinib in Stage IV Melanoma With ERBB4 Mutations
Background: Studies of melanoma tumor samples have shown that tumor cells from approximately 20 percent of melanoma patients contain a specific mutation of a gene involved in making a protein called ERBB4, and that changes in…
- Trial name
- NCT01264081
- Testing
- Lapatinib
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- National Cancer Institute (NCI)
Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Inv(16)(p13.1q22) or t(16;16)(p13.1;q22); CBFB-MYH11
Sorafenib Tosylate and Chemotherapy in Treating Older Patients With Acute Myeloid Leukemia
This phase II trial studies how well sorafenib tosylate and chemotherapy work in treating older patients with acute myeloid leukemia (AML).
- Conditions
- Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Inv(16)(p13.1q22) or t(16;16)(p13.1;q22); CBFB-MYH11, Acute Myeloid Leukemia With t(8;21); (q22; q22.1); RUNX1-RUNX1T1, Acute Promyelocytic Leukemia With PML-RARA
- Trial name
- NCT01253070
- Testing
- Biopsy, Bone Marrow Aspiration, Cytarabine
- Where
- 43 sites in United States
- Volunteers
- 54 people
- Sponsor
- National Cancer Institute (NCI)
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma
Selumetinib Sulfate in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well selumetinib sulfate works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with MAPK pathway activation mutations that have spread to…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Recurrent Childhood Central Nervous System Neoplasm
- Trial name
- NCT03213691
- Testing
- Laboratory Biomarker Analysis, Selumetinib, Selumetinib Sulfate
- Where
- 110 sites in United States, Puerto Rico
- Volunteers
- 21 people
- Sponsor
- National Cancer Institute (NCI)
Hypertrophic Cardiomyopathy
Hypertrophic Regression With N-Acetylcysteine in HCM
The purpose of the sudy is to conduct a small study to gather the preliminary data for future lage scale clinical studies that will be designed test the potential beneficial effect of over-the counter study anti-oxidant drug…
- Trial name
- HALT (NCT01537926)
- Testing
- N-acetylcysteine
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- The University of Texas Health Science Center, Houston
EGFR Exon 19 Deletion Mutation, EGFR NP_005219.2:p.L858R
Erlotinib Hydrochloride With or Without Bevacizumab in Treating Patients With Stage IV Non-small Cell Lung Cancer With Epidermal Growth Factor Receptor Mutations
This randomized phase II trial studies how well erlotinib hydrochloride (Tarceva) with or without bevacizumab (Avastin) works in treating patients with stage IV non-small cell lung cancer (NSCLC) with epidermal growth factor…
- Conditions
- EGFR Exon 19 Deletion Mutation, EGFR NP_005219.2:p.L858R, Lung Non-Squamous Non-Small Cell Carcinoma, Stage IV Lung Non-Small Cell Cancer AJCC v7
- Trial name
- NCT01532089
- Testing
- Bevacizumab, Erlotinib, Erlotinib Hydrochloride
- Where
- 19 sites in United States
- Volunteers
- 88 people
- Sponsor
- Academic and Community Cancer Research United
Non-Small Cell Lung Cancer
Study For Patients With NSCLC EGFR Mutations (Del 19 or L858R +/- T790M)
This is a Phase 1/2 study of PF-06747775 as a single agent and in combination with other cancer treatments in patients with advanced EGFRm NSCLC.
- Trial name
- NCT02349633
- Testing
- PF-06747775, Palbociclib, Avelumab
- Where
- 15 sites in United States, Australia and 2 more
- Volunteers
- 65 people
- Sponsor
- Pfizer
Carcinoma，Non-Small-Cell Lung
A Study to Assess Biomarkers Impact on Participants Response to Erlotinib Treatment for First-line Non-Small Cell Lung Cancer With Endothelial Growth Factor Receptor (EGFR) Activating Mutations
This open-label, single-arm, multi-center study will evaluate the progression-free survival in participants with histologically documented, advanced and/or metastatic chemotherapy naive, non-small cell lung cancer (NSCLC) with…
- Trial name
- BIOTEC (NCT01153984)
- Testing
- Erlotinib
- Where
- 9 sites in Romania
- Volunteers
- 23 people
- Sponsor
- Hoffmann-La Roche
Solid Malignancy, Solid Tumor
Everolimus in Patients With Advanced Solid Malignancies With TSC1, TSC2, NF1, NF2, or STK11 Mutations
The purpose of this research study is to look at participants with solid tumor malignancies and specific mutations respond to treatment with everolimus.
- Trial name
- NCT02352844
- Testing
- Everolimus
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Washington University School of Medicine
Ovarian Cancer
BMN 673 (Talazoparib), an Oral PARP Inhibitor, in People With Deleterious BRCA1/2 Mutation-Associated Ovarian Cancer Who Have Had Prior PARP Inhibitor Treatment
Background: The new drug BMN 673 (talazoparib) has been shown to fight tumor cells in animals and some people. It is a poly (ADP-ribose) polymerase (PARP) inhibitor.
- Trial name
- NCT02326844
- Testing
- BMN 673 (talazoparib)
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- National Cancer Institute (NCI)