Clinical trials: Genetic Mutation
Leukemia
Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia
The purpose of this study was to determine the efficacy and safety of twice daily (bid) oral midostaurin in patients with Aggressive Systemic Mastocytosis (ASM) or Mast Cell Leukemia (MCL) with or without an Associated…
- Trial name
- NCT00782067
- Testing
- Midostaurin (PKC412)
- Where
- 29 sites in United States, Australia and 10 more
- Volunteers
- 116 people
- Sponsor
- Novartis Pharmaceuticals
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
Testing GSK2636771 as a Potential Targeted Treatment in Cancers With PTEN Genetic Changes (MATCH-Subprotocol N)
This phase II MATCH treatment trial identifies the effects of GSK2636771 in patients whose cancer has a genetic change called PTEN mutation or deletion.
- Conditions
- Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Hematopoietic and Lymphoid Cell Neoplasm, Refractory Lymphoma
- Trial name
- NCT04439149
- Testing
- PI3K-beta Inhibitor GSK2636771
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Cancer Institute (NCI)
Leukemia, Acute Myeloid Leukemia
Phase I/II, Study of Selective Inhibitor of Nuclear Export (SINE) Selinexor (KPT-330) + Sorafenib in Acute Myeloid Leukemia
There are 2 parts to this study: Part 1 (dose escalation) and Part 2 (dose expansion). The goal of Part 1 of this clinical research study is to find the highest tolerated dose of the combination of selinexor (KPT-330) and…
- Trial name
- NCT02530476
- Testing
- Selinexor, Sorafenib
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- M.D. Anderson Cancer Center
Non-Squamous Non-Small Cell Lung Cancer
A Study to Assess the Incidence of EGFR Mutation in Patients With Newly Diagnosed Locally Advanced or Metastatic Non-Small Cell Lung Cancer in the UK, And of Tarceva (Erlotinib) as First-Line Therapy in EGFR Mutation Positive Patients.
This study will assess the prevalence of epidermal growth factor receptor (EGFR) mutations in newly diagnosed patients with locally advanced or metastatic non-small cell lung cancer (NSCLC).
- Trial name
- NCT01250119
- Testing
- erlotinib [Tarceva]
- Where
- 22 sites in United Kingdom
- Volunteers
- 688 people
- Sponsor
- Hoffmann-La Roche
Cystic Fibrosis
Lumacaftor/Ivacaftor Combination Therapy in Subjects With CF Who Have an A455E CFTR Mutation
This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter, crossover study that will evaluate the efficacy of LUM/IVA in subjects with CF 12 years of age and older who have at least one A455E mutation.
- Trial name
- NCT03061331
- Testing
- LUM/IVA
- Where
- 2 sites in Netherlands
- Volunteers
- 20 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
This is an open-label extension study for participants who completed a Phase 3, placebo-controlled study of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.
- Trial name
- NCT02456103
- Testing
- Ataluren
- Where
- 70 sites in United States, Argentina and 14 more
- Volunteers
- 246 people
- Sponsor
- PTC Therapeutics
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
Testing AZD9291 as Potentially Targeted Treatment in Cancers With EGFR Genetic Changes (MATCH-Subprotocol E)
This phase II MATCH treatment trial evaluates the effectiveness of osimertinib (AZD9291) in treating patients with cancer that has certain genetic changes called EGFR mutations.
- Conditions
- Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Hematopoietic and Lymphoid Cell Neoplasm, Refractory Lymphoma
- Trial name
- NCT06303167
- Testing
- Biopsy Procedure, Biospecimen Collection, Echocardiography Test
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- National Cancer Institute (NCI)
Carcinoma, Non-Small-Cell Lung
Afatinib in Patients With Non Small Cell Lung Cancer (NSCLC) With Epidermal Growth Factor Receptor (EGFR) Mutations
The main aim of this study is to evaluate the safety and tolerability of afatinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring Epidermal Growth Factor Receptor (EGFR) mutation(s)…
- Trial name
- NCT02044380
- Testing
- Afatinib
- Where
- 5 sites in Canada
- Volunteers
- 14 people
- Sponsor
- Boehringer Ingelheim
IFN-Gamma Therapy, CGD Gene Mutation
Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease
Background: Chronic granulomatous disease (CGD) is an immunodeficiency disease in which white blood cells are unable to kill certain bacteria and fungi.
- Conditions
- IFN-Gamma Therapy, CGD Gene Mutation, CGD Response to IFNg, CGD - Chronic Granulomatous Disease
- Trial name
- NCT01147042
- Testing
- IFN-gamma
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Colorectal Cancer (CRC)
Sotorasib and Panitumumab Versus Investigator's Choice for Participants With Kirsten Rat Sarcoma (KRAS) p.G12C Mutation
The aim of the study is to compare progression-free survival (PFS) in previously treated participants with Kirsten rat sarcoma (KRAS) p.G12C mutated colorectal cancer (CRC) receiving sotorasib 240 mg once daily (QD) and…
- Trial name
- CodeBreak300 (NCT05198934)
- Testing
- Sotorasib, Panitumumab, Trifluridine and Tipiracil
- Where
- 105 sites in United States, Australia and 10 more
- Volunteers
- 160 people
- Sponsor
- Amgen
Carcinoma, Non-Small-Cell Lung
A Study of Atezolizumab in Combination With Bevacizumab in Patients With EGFR Mutation Positive Stage IIIB-IV Non-Squamous Non-Small Cell Lung Cancer
This is an open-label, single-arm, phase II, multicenter study designed to evaluated the efficacy and safety of atezolizumab in combination with bevacizumab in PD-L1-selected patients with Stage IIIB-IV Non-Squamous NSCLC…
- Trial name
- NCT04426825
- Testing
- Atezolizumab, Bevacizumab
- Where
- 10 sites in China
- Volunteers
- 23 people
- Sponsor
- Hoffmann-La Roche
Breast Cancer
Study of the Molecular Features of Postmenopausal Women With HR+ HER2-negative aBC on First-line Treatment With Ribociclib and Letrozole and, in Patients With a PIK3CA Mutation, on Second-line Treatment With Alpelisib Plus Fulvestrant
The purpose of this clinical trial is to study of the molecular features of postmenopausal women with hormone receptor-positive (HR+) HER2-negative advanced breast cancer on first-line treatment with ribociclib and letrozole and…
- Trial name
- BioItaLEE (NCT03439046)
- Testing
- Ribociclib, Letrozole, Alpelisib
- Where
- 41 sites in Italy
- Volunteers
- 287 people
- Sponsor
- Novartis Pharmaceuticals
Adult Burkitt Lymphoma, B-Cell Lymphoma, Unclassifiable, With Features Intermediate Between Diffuse Large B-Cell Lymphoma and Burkitt Lymphoma
Combination Chemotherapy, Rituximab, and Ixazomib Citrate in Treating Patients With Non-Hodgkin Lymphoma
The purpose of this study is to evaluate the effects, good and bad of a new drug called ixazomib (also called MLN9708), when it is given along with a common treatment combination, called Dose-Adjusted EPOCH-R (DA-EPOCH-R, for…
- Conditions
- Adult Burkitt Lymphoma, B-Cell Lymphoma, Unclassifiable, With Features Intermediate Between Diffuse Large B-Cell Lymphoma and Burkitt Lymphoma, Diffuse Large B-Cell Lymphoma, MYC Gene Mutation
- Trial name
- NCT02481310
- Testing
- Cyclophosphamide, Cytarabine, Doxorubicin Hydrochloride
- Where
- 5 sites in United States
- Volunteers
- 38 people
- Sponsor
- Northwestern University
Metastatic Urothelial Carcinoma, Unresectable Urothelial Carcinoma
Pemigatinib + Pembrolizumab vs Pemigatinib Alone vs Standard of Care for Urothelial Carcinoma (FIGHT-205)
The purpose of this study is to evaluate the safety and efficacy of pemigatinib plus pembrolizumab or pemigatinib alone versus the standard of care for participants with metastatic or unresectable urothelial carcinoma who are not…
- Trial name
- NCT04003610
- Testing
- Pemigatinib, Pembrolizumab, Gemcitabine
- Where
- 79 sites in United States, Austria and 14 more
- Volunteers
- 7 people
- Sponsor
- Incyte Corporation
Aniridia
Study of Ataluren in Participants With Nonsense Mutation Aniridia
This study is designed to evaluate the effect of ataluren on Maximum Reading Speed as measured using the Minnesota Low Vision Reading Test (MNREAD) Acuity Charts in participants with nonsense mutation aniridia.
- Trial name
- STAR (NCT02647359)
- Testing
- Ataluren
- Where
- 3 sites in United States, Canada
- Volunteers
- 39 people
- Sponsor
- PTC Therapeutics
Melanoma
Dabrafenib Plus Trametinib vs Vemurafenib Alone in Unresectable or Metastatic BRAF V600E/K Cutaneous Melanoma
This was a two-arm, open-label, randomized, Phase III study comparing dabrafenib (GSK2118436) and trametinib (GSK1120212) combination therapy with vemurafenib.
- Trial name
- COMBI-v (NCT01597908)
- Testing
- Dabrafenib, Vemurafenib, Trametinib
- Where
- 205 sites in United States, Argentina and 26 more
- Volunteers
- 704 people
- Sponsor
- Novartis Pharmaceuticals
Malignant Melanoma
A Study of the Effect of Food on the Pharmacokinetics of Single Dose RO5185426 And the Safety And Efficacy of Continuous Administration in Patients With BRAF V600E Mutation-Positive Metastatic Melanoma
This randomized, open-label, two period crossover study will evaluate the effect of food on the pharmacokinetics of a single dose of RO5185426 and the efficacy and safety of continuous administration in patients with BRAF V600E…
- Trial name
- NCT01264380
- Testing
- RO5185426
- Where
- 10 sites in United States
- Volunteers
- 16 people
- Sponsor
- Hoffmann-La Roche
Recessive Dystrophic Epidermolysis Bullosa
Gentamicin for RDEB
Recessive dystrophic epidermolysis bullosa (RDEB) is an incurable, devastating, inherited skin disease caused by mutations in the COL7A1 gene that encodes for type VII collagen (C7), the major component of anchoring fibrils…
- Trial name
- NCT03012191
- Testing
- Gentamicin Sulfate
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Southern California
Breast Cancer, Breast Cancer Stage IV
A Study of Mirdametinib on Its Own or in Combination With Fulvestrant in People With Solid Tumor Cancer
The purpose of this study to find out whether mirdametinib is a safe treatment for people with advanced solid tumor cancer that has certain mutations.
- Conditions
- Breast Cancer, Breast Cancer Stage IV, HER2-negative Breast Cancer, Solid Carcinoma
- Trial name
- NCT05054374
- Testing
- Mirdametinib, Fulvestrant
- Where
- 4 sites in United States
- Volunteers
- 6 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
NSCLC
Study of Poziotinib in Japanese Patients With NSCLC
A Phase 1/2, open-label, multicenter study to determine dose, tolerability, safety and efficacy of poziotinib in Japanese patients non-small cell lung cancer (NSCLC).
- Trial name
- NCT04402008
- Testing
- Poziotinib Once Daily Dosing, Poziotinib Twice Daily Dosing, Poziotinib Once Daily Dosing or Twice Daily Dosing as determined in Phase 1
- Where
- 3 sites in Japan
- Volunteers
- 42 people
- Sponsor
- Spectrum Pharmaceuticals, Inc