Clinical trials: Genetic Mutation
Neuroendocrine Tumors, Neuroendocrine Carcinoma
Study of Mutation-Targeted Therapy With Sunitinib or Everolimus in People With Advanced Low- or Intermediate-Grade Neuroendocrine Tumors of the Gastrointestinal Tract and Pancreas With or Without Cytoreductive Surgery
Background: Neuroendocrine tumors (NETs) come from cells of the hormonal and nervous systems. Some people have surgery to shrink the tumor. Sometimes the tumors come back.
- Conditions
- Neuroendocrine Tumors, Neuroendocrine Carcinoma, Neuroendocrine Neoplasms, Carcinoma, Neuroendocrine
- Trial name
- NCT02315625
- Testing
- Sunitinib, Everolimus
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- National Cancer Institute (NCI)
Carcinoma, Non-Small-Cell Lung
Study Evaluating Efficacy and Safety of Capmatinib in Combination With Osimertinib in Adult Subjects With Non-small Cell Lung Cancers as Second Line Therapy
This study aimed to evaluate the anticancer activity of capmatinib in combination with osimertinib compared to platinum-pemetrexed based doublet chemotherapy as second line treatment in patients with advanced or metastatic…
- Trial name
- GEOMETRY-E (NCT04816214)
- Testing
- Capmatinib, Osimertinib, Pemetrexed
- Where
- 4 sites in Japan, Singapore and 1 more
- Volunteers
- 6 people
- Sponsor
- Novartis Pharmaceuticals
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
Metastatic Malignant Solid Neoplasm, Refractory Malignant Solid Neoplasm
Trametinib and Navitoclax in Treating Patients With Advanced or Metastatic Solid Tumors
This phase Ib/II trial studies the side effects and best dose of trametinib and navitoclax and how well they work in treating patients with solid tumors that have spread to other places in the body (advanced or metastatic).
- Conditions
- Metastatic Malignant Solid Neoplasm, Refractory Malignant Solid Neoplasm, Unresectable Malignant Solid Neoplasm
- Trial name
- NCT02079740
- Testing
- Biopsy Procedure, Biospecimen Collection, Computed Tomography
- Where
- 2 sites in United States
- Volunteers
- 96 people
- Sponsor
- National Cancer Institute (NCI)
Activated PI3Kdelta Syndrome (APDS); PASLI Disease
Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI
This study is designed to provide long-term CDZ173 treatment, a selective PI3Kδ inhibitor, to the patients with genetically activated PI3Kδ, i.e., patients with APDS/PASLI who participated in the CCDZ173X2201 study or who were…
- Trial name
- NCT02859727
- Testing
- CDZ173
- Where
- 8 sites in United States, Belarus and 5 more
- Volunteers
- 37 people
- Sponsor
- Pharming Technologies B.V.
IDH1-mutant Cholangiocarcinoma
Ivosidenib, Nivolumab, and Ipilimumab Combination in Previously Treated Subjects With Nonresectable or Metastatic IDH1 Mutant Cholangiocarcinoma
This is a Phase 1/2 study evaluating the safety, tolerability, and activity of ivosidenib in combination with immunotherapy in participants with nonresectable or metastatic cholangiocarcinoma.
- Trial name
- NCT05921760
- Testing
- Ivosidenib, Nivolumab, Ipilimumab
- Where
- 5 sites in United States, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Servier Bio-Innovation LLC
Alzheimers Disease, Dementia
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.
The purpose of this study is to assess the safety, tolerability, biomarker and cognitive efficacy of investigational products in subjects who are known to have an Alzheimer's disease-causing mutation by determining if treatment…
- Conditions
- Alzheimers Disease, Dementia, Alzheimers Disease, Familial
- Trial name
- DIAN-TU (NCT04623242)
- Testing
- Gantenerumab, Solanezumab
- Where
- 25 sites in United States, Australia and 6 more
- Volunteers
- 194 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Cystic Fibrosis
A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
In some participants with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein.
- Trial name
- NCT00458341
- Testing
- Ataluren
- Where
- 3 sites in Belgium, France
- Volunteers
- 30 people
- Sponsor
- PTC Therapeutics
HRAS Gene Mutation, HNSCC
Safety and Efficacy of Tipifarnib in Head and Neck Cancer With HRAS Mutations and Impact of HRAS on Response to Therapy
An international, multicenter, open-label, 2 cohort, non-comparative, pivotal study evaluating the efficacy of tipifarnib in HRAS mutant HNSCC (AIM-HN).
- Trial name
- AIM-HN/SEQ-HN (NCT03719690)
- Testing
- Tipifarnib, HRAS Detection Assay
- Where
- 102 sites in United States, Australia and 14 more
- Volunteers
- 296 people
- Sponsor
- Kura Oncology, Inc.
Chronic Pancreatitis, Genetic Mutation
An Observational Study on the Natural Course of Chronic Pancreatitis
To explore the impact on genetic and environmental factors for clinical manifestation, and the progression of chronic pancreatitis, including development of pancreatic insufficiency and other complications.
- Conditions
- Chronic Pancreatitis, Genetic Mutation, Smoking, Tobacco, Drinking, Alcohol
- Trial name
- NCT04574297
- Testing
- smoking and alcohol assumption, genetic sequencing
- Volunteers
- 798 people
- Sponsor
- Changhai Hospital
Advanced Solid Tumor, BRAF Gene Mutation
Study of Ulixertinib for Patients With Advanced Malignancies Harboring MEK or Atypical BRAF Alterations
This BVD-523-ABC study builds on the safety and clinical activity experience of previous studies that have evaluated ulixertinib as a novel targeted cancer treatment in cohorts of patients with specific genetic alterations and…
- Conditions
- Advanced Solid Tumor, BRAF Gene Mutation, BRAF Gene Alteration, MEK Mutation
- Trial name
- NCT04488003
- Testing
- Ulixertinib, Physician's Choice
- Where
- 22 sites in United States
- Volunteers
- 104 people
- Sponsor
- BioMed Valley Discoveries, Inc
Pulmonary Cancer
First-line Treatment for Adenocarcinoma Patients With Epidermal Growth Factor Receptor (EGFR) Mutation
The purpose of this study is to evaluate the overall objective tumor response rate (ORR) of Gefitinib.
- Trial name
- NCT00344773
- Testing
- Gefitinib
- Where
- 2 sites in South Korea
- Volunteers
- 46 people
- Sponsor
- AstraZeneca
Essential Thrombocythemia (ET)
Exploratory Multi-centre Trial In Patients With ET Treated With XAGRID®
This study is hypothesis-generating to explore the impact of JAK2 (V617F) mutation status on the treatment response to anagrelide hydrochloride
- Trial name
- EMIX (NCT01352585)
- Testing
- Anagrelide hydrochloride
- Where
- 10 sites in Italy
- Volunteers
- 47 people
- Sponsor
- Shire
Acute Myeloid Leukemia
Gilteritinib vs Midostaurin in FLT3 Mutated Acute Myeloid Leukemia
Eligible untreated patients with FLT3 acute myeloid leukemia (AML) between the ages of 18 and 70 will be randomized to receive gilteritinib or midostaurin during induction and consolidation.
- Trial name
- NCT03836209
- Testing
- Gilteritinib, Midostaurin, Daunorubicin
- Where
- 44 sites in United States
- Volunteers
- 181 people
- Sponsor
- PrECOG, LLC.
Glioblastoma, Brain Tumor, Recurrent
Safety and Efficacy of PF-299804 (Dacomitinib), a Pan-HER Irreversible Inhibitor, in Patients With Recurrent Glioblastoma With EGFR Amplification or Presence of EGFRvIII Mutation. A Phase II CT.
This multicenter, 2-stage, open-label, phase II trial aims to assess the efficacy and safety of dacomitinib in adult patients with recurrent Glioblastoma (GBM) with EGFR gene amplification and/or EGFRvIII mutation.
- Trial name
- NCT01520870
- Testing
- PF-299804 (Dacomitinib)
- Where
- 12 sites in Spain
- Volunteers
- 49 people
- Sponsor
- Grupo Español de Investigación en Neurooncología
Colorectal Cancer Metastatic
Regorafenib+FOLFIRI Versus Placebo+FOLFIRI as 2nd Line Tx in Metastatic Colorectal Cancer
This randomized (2:1), multi-center, placebo-controlled, phase II efficacy study is designed to compare PFS between regorafenib + FOLFIRI chemotherapy (ARM A) versus placebo + FOLFIRI (ARM B) in patients with mCRC previously…
- Trial name
- NCT01298570
- Testing
- Regorafenib (BAY 73-4506), FOLFIRI
- Where
- 24 sites in United States, Ireland
- Volunteers
- 181 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
EGFR Gene Mutation, Nonsmall Cell Lung Cancer
Dasatinib and Osimertinib (AZD9291) in Advanced Non-Small Cell Lung Cancer With EGFR Mutations
This is a study for patients with advanced non-small cell lung cancer with changes to their cancer cells called EGFR mutations. Mutated EGFR is important in the growth of cancer cells.
- Trial name
- NCT02954523
- Testing
- Dasatinib, Osimertinib
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Chul Kim
Parkinson's Disease
A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of Venglustat (GZ/SAR402671) in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation
Primary Objectives: Part 1: To determine the safety and tolerability of 4, 8, and 15 milligrams of GZ/SAR402671 (venglustat) administered orally for 4 weeks, as compared to placebo in participants with early-stage Parkinson's…
- Trial name
- MOVES-PD (NCT02906020)
- Testing
- venglustat GZ/SAR402671
- Where
- 52 sites in United States, Austria and 14 more
- Volunteers
- 273 people
- Sponsor
- Genzyme, a Sanofi Company
Melanoma and Metastatic Colorectal Cancer
A Phase I Study of Oral LGX818 in Adult Patients With Advanced or Metastatic BRAF Mutant Melanoma
CLGX818X2101 is a first-time in-human, phase I study to establish the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of daily administered LGX818 (daily, twice daily and/or every-other-day), a RAF kinase…
- Trial name
- NCT01436656
- Testing
- LGX818
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 107 people
- Sponsor
- Pfizer
Cystic Fibrosis
A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis
This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for…
- Trial name
- NCT03559062
- Testing
- TEZ/IVA, IVA
- Where
- 27 sites in Australia, Belgium and 7 more
- Volunteers
- 67 people
- Sponsor
- Vertex Pharmaceuticals Incorporated