Clinical trials: Genetic Mutation
Advanced/Metastatic Solid Tumors With KRAS p.G12C Mutation
AMG 510 Ethnic Sensitivity Study (CodeBreaK 105).
To evaluate safety, tolerability, PK, and preliminary efficacy of AMG 510 PO QD in subjects of Chinese descent with KRAS p.G12C-mutant advanced/metastatic solid tumors.
- Trial name
- NCT04380753
- Testing
- AMG 510
- Where
- 4 sites in Hong Kong, Taiwan
- Volunteers
- 12 people
- Sponsor
- Amgen
Non Small Cell Lung Cancer
A Study of Abemaciclib (LY2835219) in Participants With Previously Treated KRAS Mutated Lung Cancer
The main purpose of this study is to evaluate how safe and effective the study drug known as abemaciclib is in participants with lung cancer.
- Trial name
- JUNIPER (NCT02152631)
- Testing
- Abemaciclib, Erlotinib
- Where
- 231 sites in United States, Argentina and 19 more
- Volunteers
- 453 people
- Sponsor
- Eli Lilly and Company
Cystic Fibrosis
Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109…
- Trial name
- NCT02544451
- Testing
- LUM/IVA
- Where
- 60 sites in United States, Australia and 7 more
- Volunteers
- 246 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma
Acalabrutinib in Patients With Relapsed/Refractory and Treatment naïve Deletion 17p CLL/SLL
This study is to determine the response to acalabrutinib in patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
- Trial name
- NCT02337829
- Testing
- Acalabrutinib (Arm A), Acalabrutinib (Arm B)
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Acerta Pharma BV
Cowden's Disease, Hamartoma Syndrome, Multiple
Sirolimus to Treat Cowden Syndrome and Other PTEN Hamartomatous Tumor Syndromes
Background: People with phosphatase and tensin homolog deleted on chromosome 10 (PTEN) hamartomatous tumor syndromes (PHTS) have a mutation in one of their genes called PTEN that can lead to benign tumors called hamartomas…
- Trial name
- NCT00971789
- Testing
- fludeoxyglucose F 18, sirolimus, Clinical Videography
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- National Cancer Institute (NCI)
Malignant Rhabdoid Tumors (MRT), Rhabdoid Tumors of the Kidney (RTK)
A Study of Tazemetostat in Adult Participants With Soft Tissue Sarcoma
This study will include participants with various types of cancer known as soft-tissue sarcomas. Tissues that can be affected by soft tissue sarcomas include fat, muscle, blood vessels, deep skin tissues, tendons and ligaments.
- Conditions
- Malignant Rhabdoid Tumors (MRT), Rhabdoid Tumors of the Kidney (RTK), Atypical Teratoid Rhabdoid Tumors (ATRT), Selected Tumors With Rhabdoid Features
- Trial name
- NCT02601950
- Testing
- Tazemetostat
- Where
- 32 sites in United States, Australia and 7 more
- Volunteers
- 267 people
- Sponsor
- Epizyme, Inc.
Solid Tumors Harboring a BRAF V600 Mutation
A Phase Ib/II Study of LGX818 in Combination With MEK162 in Adult Patients With BRAF Dependent Advanced Solid Tumors
This is a multi-center, open-label, dose finding, Phase Ib dose escalation study to estimate the MTD(s) and/or RP2D(s) for the dual combination of LGX818 and MEK162 and the triple combination of LGX818 and MEK162 and LEE011…
- Trial name
- NCT01543698
- Testing
- LGX818, MEK162, LEE011
- Where
- 26 sites in United States, Australia and 7 more
- Volunteers
- 189 people
- Sponsor
- Pfizer
Melanoma
A Study of AMNN107 in the Treatment of Metastatic and/or Inoperable Melanoma Harboring a c-Kit Mutation
The purpose of this study is to determine whether nilotinib is efficacious in the treatment of metastatic and/or inoperable melanoma harboring a c-Kit mutation.
- Trial name
- TEAM (NCT01028222)
- Testing
- Nilotinib, DTIC
- Where
- 49 sites in United States, Argentina and 12 more
- Volunteers
- 55 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation
This is a Phase 4, randomized, double-blind, placebo-controlled, parallel-group study in subjects aged 12 years and older with CF who are homozygous for the F508del-CFTR mutation.
- Trial name
- NCT02875366
- Testing
- LUM/IVA
- Where
- 12 sites in Australia, United Kingdom
- Volunteers
- 70 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
KRAS Mutation-Related Tumors, Metastatic Solid Tumor
A Study of Repotrectinib in Combination With Other Anticancer Therapies for the Treatment of Subjects With KRAS-Mutant Solid Tumors
A Phase 1b/2 Study of Repotrectinib in Combination with Other Anticancer Therapies for the Treatment of Subjects with KRAS-Mutant Advanced Solid Tumors (TRIDENT-2)
- Conditions
- KRAS Mutation-Related Tumors, Metastatic Solid Tumor, Advanced Solid Tumor
- Trial name
- NCT05071183
- Testing
- TPX-0005, Trametinib
- Where
- 6 sites in United States
- Volunteers
- 9 people
- Sponsor
- Turning Point Therapeutics, Inc.
Amino Acid Metabolism, Inborn Errors
Ataluren for Nonsense Mutation Methylmalonic Acidemia
Methylmalonic acidemia (MMA) is a rare genetic disorder caused by mutations in the gene for mitochondrial enzyme methylmalonyl-CoA mutase (MCM) or in one of the genes for adenosylcobalamin (AdoCbl).
- Trial name
- NCT01141075
- Testing
- Ataluren
- Where
- 9 sites in Belgium, France and 4 more
- Volunteers
- 11 people
- Sponsor
- PTC Therapeutics
Fabry Disease
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
The primary objective of this study was to compare the effect of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
- Trial name
- NCT00925301
- Testing
- migalastat hydrochloride
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 67 people
- Sponsor
- Amicus Therapeutics
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
Cancer, Melanoma
Study to Investigate the Objective Response Rate of Dabrafenib in Combination With Trametinib in Subjects With BRAF V600 Mutation-Positive Melanoma
This was a single-arm, open-label, multi-center, Phase II study to evaluate dabrafenib and trametinib combination therapy in subjects with BRAF V600 mutation-positive, unresectable or metastatic Acral lentiginous or cutaneous…
- Trial name
- NCT02083354
- Testing
- Dabrafenib, Trametinib
- Where
- 13 sites in China, Hong Kong and 3 more
- Volunteers
- 77 people
- Sponsor
- Novartis Pharmaceuticals
IDH1 Mutation, IDH2 Mutation
BGB-290 and Temozolomide in Treating Patients With Recurrent Gliomas With IDH1/2 Mutations
This phase I/II trial studies the side effects and how well BGB-290 and temozolomide work in treating patients with gliomas (brain tumors) with IDH1/2 mutations that have come back.
- Conditions
- IDH1 Mutation, IDH2 Mutation, Recurrent Glioblastoma, Recurrent WHO Grade II Glioma
- Trial name
- NCT03914742
- Testing
- PARP Inhibitor BGB-290, Temozolomide, Therapeutic Conventional Surgery
- Where
- 12 sites in United States
- Volunteers
- 67 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Carcinoma
Osimertinib Plus Savolitinib in EGFRm+/MET+ NSCLC Following Prior Osimertinib
This study (the SAVANNAH study) will investigate the efficacy of osimertinib in combination with savolitinib in participants with epidermal growth factor receptor mutation positive (EGFRm+) and mesenchymal epithelial transition…
- Trial name
- SAVANNAH (NCT03778229)
- Testing
- Osimertinib, Savolitinib
- Where
- 90 sites in United States, Brazil and 11 more
- Volunteers
- 367 people
- Sponsor
- AstraZeneca
Malignant Melanoma
A Study of Vemurafenib (RO5185426) in Comparison With Dacarbazine in Previously Untreated Patients With Metastatic Melanoma (BRIM 3)
This randomized, open-label study evaluated the efficacy, safety and tolerability of vemurafenib (RO5185426) as compared to dacarbazine in previously untreated patients with metastatic melanoma.
- Trial name
- NCT01006980
- Testing
- Vemurafenib, Dacarbazine
- Where
- 111 sites in United States, Australia and 10 more
- Volunteers
- 675 people
- Sponsor
- Hoffmann-La Roche
Cystic Fibrosis
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation
The purpose of this study was to evaluate the safety and efficacy of ivacaftor in participants with cystic fibrosis (CF) who were aged 12 years or older and were homozygous for the F508del-CF transmembrane conductance regulator…
- Trial name
- DISCOVER (NCT00953706)
- Testing
- Ivacaftor
- Where
- 34 sites in United States
- Volunteers
- 140 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Breast Neoplasms, BRCA 1 Gene Mutation
A Study Evaluating Talazoparib (BMN 673), a PARP Inhibitor, in Advanced and/or Metastatic Breast Cancer Patients With BRCA Mutation (EMBRACA Study)
The purpose of this open-label, 2:1 randomized phase III trial is to compare the safety and efficacy of talazoparib (also known as BMN 673) versus protocol-specific physician's choice in patients who have locally advanced and/or…
- Conditions
- Breast Neoplasms, BRCA 1 Gene Mutation, BRCA 2 Gene Mutation
- Trial name
- EMBRACA (NCT01945775)
- Testing
- talazoparib, Physician's-Choice
- Where
- 362 sites in United States, Australia and 14 more
- Volunteers
- 431 people
- Sponsor
- Pfizer
Non-small Cell Lung Cancer
A Study Of Dacomitinib (PF-00299804) In Patients With Advanced Non-Small Cell Lung Cancer
This is a Phase 2 study of oral dacomitinib given every 12 hours over days 1-4 of each two-week cycle to patients with Non-small cell lung cancer.
- Trial name
- NCT01858389
- Testing
- Dacomitinib
- Where
- 14 sites in United States, South Korea
- Volunteers
- 41 people
- Sponsor
- Pfizer