Clinical trials: Genetic Mutation
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
Acute Myeloid Leukemia
Milademetan Plus Quizartinib Combination Study in FLT3-ITD Mutant Acute Myeloid Leukemia (AML)
Participants with AML that have gone into remission and come back (relapsed) or gone into remission with a number of leukemia cells still in their system (refractory) will be recruited for this study.
- Trial name
- NCT03552029
- Testing
- Quizartinib, Milademetan
- Where
- 8 sites in United States
- Volunteers
- 10 people
- Sponsor
- Daiichi Sankyo
Cystic Fibrosis
A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for F508del.
- Trial name
- NCT03125395
- Testing
- LUM/IVA
- Where
- 20 sites in United States, Canada
- Volunteers
- 57 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy
This study will evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the…
- Trial name
- NCT03447262
- Testing
- VX-659/TEZ/IVA, IVA
- Where
- 100 sites in United States, Australia and 9 more
- Volunteers
- 484 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Recurrent Endometrial Carcinoma, Recurrent Ovarian Carcinoma
Bevacizumab and/or Niraparib in Patients With Recurrent Endometrial and/or Ovarian Cancer With ARID1A Mutation
The purpose of this research study is to test the proportion of tumor response to the combination treatment with niraparib and bevacizumab and see what effects (good and bad) this combination treatment has on patients with…
- Conditions
- Recurrent Endometrial Carcinoma, Recurrent Ovarian Carcinoma, ARID1A Gene Mutation
- Trial name
- NCT05523440
- Testing
- Bevacizumab, Niraparib
- Where
- 2 sites in United States
- Volunteers
- 9 people
- Sponsor
- University of Oklahoma
Progressive Familial Intrahepatic Cholestasis (PFIC)
An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
- Trial name
- NCT04185363
- Testing
- Maralixibat
- Where
- 28 sites in United States, Argentina and 14 more
- Volunteers
- 84 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Non-squamous, Non-Small Cell Lung Cancer
The Study Observes Afatinib as First-line Treatment in Patients With Epidermal Growth Factor Receptor (EGFR) Mutation-positive Advanced Non-small Cell Lung Cancer
This study aims to observe the sequential strategy with afatinib as first-line treatment and to find the optimal treatment strategy for long-term chemotherapy-free regimens in Chinese patients with EGFR-mutated advanced NSCLC.
- Trial name
- NCT04206787
- Testing
- Afatinib
- Where
- 10 sites in China
- Volunteers
- 72 people
- Sponsor
- Boehringer Ingelheim
Leukemia, Myeloid, Acute
Phase 2 Study of Quizartinib in Participants With Acute Myeloid Leukemia (AML) FLT3 Internal Tandem Duplication (FLT3/ITD) Mutation
This is a Phase 2, multi-center, open-label study to evaluate the efficacy, safety and pharmacokinetics of quizartinib monotherapy in Japanese subjects with FLT3-ITD positive refractory or relapsed acute myeloid leukemia.
- Trial name
- NCT02984995
- Testing
- Quizartinib
- Where
- 27 sites in Japan
- Volunteers
- 37 people
- Sponsor
- Daiichi Sankyo Co., Ltd.
Cystic Fibrosis
Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have the R117H-CF Transmembrane Conductance Regulator (CFTR) Mutation (KONDUCT)
The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis (CF) who have the R117H-CFTR mutation.
- Trial name
- KONDUCT (NCT01614457)
- Testing
- Ivacaftor
- Where
- 31 sites in United States, United Kingdom
- Volunteers
- 70 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Relapsed Ovarian Cancer, BRCA Mutation, Platinum Sensitivity
Olaparib Tablets as a Treatment for Ovarian Cancer Subjects With Different HRD Tumor Status
This is a non-randomized, open-label study to assess olaparib tablets as a treatment for subjects with different homologous recombination deficiency (HRD) tumor status and with platinum-sensitive, relapsed, high-grade serous or…
- Trial name
- NCT02983799
- Testing
- OLAPARIB
- Where
- 47 sites in United States, Canada
- Volunteers
- 272 people
- Sponsor
- AstraZeneca
Lobular Breast Carcinoma, Gastric Cancer
Crizotinib in Lobular Breast, Diffuse Gastric and Triple Negative Lobular Breast Cancer or CDH1-mutated Solid Tumours
The purpose of this study was to find out how effective the combination of crizotinib and fulvestrant was in shrinking lobular breast cancer tumours.
- Conditions
- Lobular Breast Carcinoma, Gastric Cancer, Triple Negative Breast Cancer, CDH1 Gene Mutation
- Trial name
- ROLo (NCT03620643)
- Testing
- Crizotinib Oral Capsule [Xalkori], Fulvestrant 50 MG/ML Prefilled Syringe [Faslodex or generic]
- Where
- 5 sites in United Kingdom
- Volunteers
- 33 people
- Sponsor
- Royal Marsden NHS Foundation Trust
Cystic Fibrosis
A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor
This is a Phase 3, randomized, double-blind, ivacaftor-controlled, parallel-group, multicenter study of tezacaftor in combination with ivacaftor in subjects aged 12 years and older with CF who are heterozygous for the…
- Trial name
- NCT02412111
- Testing
- Ivacaftor, Tezacaftor/Ivacaftor
- Where
- 68 sites in United States, Australia and 8 more
- Volunteers
- 156 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Melanoma
A Study Comparing Trametinib and Dabrafenib Combination Therapy to Dabrafenib Monotherapy in Subjects With BRAF-mutant Melanoma
This was a two-arm, double-blinded, randomized, Phase III study comparing dabrafenib and trametinib combination therapy to dabrafenib administered with a placebo (dabrafenib monotherapy).
- Trial name
- NCT01584648
- Testing
- Dabrafenib, Trametinib
- Where
- 121 sites in United States, Argentina and 12 more
- Volunteers
- 423 people
- Sponsor
- Novartis Pharmaceuticals
Melanoma
A Study of Atezolizumab Plus Cobimetinib and Vemurafenib Versus Placebo Plus Cobimetinib and Vemurafenib in Previously Untreated BRAFv600 Mutation-Positive Patients With Metastatic or Unresectable Locally Advanced Melanoma
This is a Phase III, double-blinded, placebo-controlled, randomized, multicenter study designed to evaluate the efficacy, safety, and pharmacokinetics of atezolizumab + cobimetinib + vemurafenib compared with placebo +…
- Trial name
- NCT02908672
- Testing
- Atezolizumab, Cobimetinib, Vemurafenib
- Where
- 110 sites in United States, Australia and 18 more
- Volunteers
- 514 people
- Sponsor
- Hoffmann-La Roche
Solid Tumor, Hematologic Malignancies
BGJ398 for Patients With Tumors With FGFR Genetic Alterations
The purpose of this signal seeking study was to determine whether treatment with BGJ398 demonstrates sufficient efficacy in select FGFR pathway-regulated solid tumors and/or hematologic malignancies to warrant further study.
- Trial name
- CBGJ398XUS04 (NCT02160041)
- Testing
- BGJ398
- Where
- 56 sites in United States
- Volunteers
- 84 people
- Sponsor
- Novartis Pharmaceuticals
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)
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
- REVERSE (NCT02652780)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 37 people
- Sponsor
- GenSight Biologics
B-Cell Chronic Lymphocytic Leukemia, B-Lymphocytic Leukemia, Chronic
Ibrutinib, Fludarabine, and Pembrolizumab in High-Risk or Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Background: Chronic lymphocytic leukemia and small lymphocytic lymphoma (hereby referred as CLL) are tumors of B cells. A subset of patients categorized as high-risk CLL has a poor clinical outcome when treated with conventional…
- Conditions
- B-Cell Chronic Lymphocytic Leukemia, B-Lymphocytic Leukemia, Chronic, Chronic Lymphocytic Leukemia, Leukemia, Chronic Lymphatic
- Trial name
- NCT03204188
- Testing
- Ibrutinib, Fludarabine, Pembrolizumab
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Malignant Melanoma
Comparison of In-House Methods and Cobas BRAF V600 Mutation Assay in Melanoma Tumor Samples
This non-interventional study will compare the Cobas BRAF V600 mutation assay with in-house methods used in molecular laboratories for the assessment of the BRAF mutation status in melanoma tumor samples.
- Trial name
- NCT01744860
- Where
- 12 sites in France
- Volunteers
- 420 people
- Sponsor
- Hoffmann-La Roche
Leber's Hereditary Optic Neuropathy
Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy
The study is a dose-escalation study, phase 1. The objective of this proposed clinical trial is to evaluate the safety of mitochondrially targeted ND4 gene therapy with the adeno-associated viral vector in appropriate LHON…
- Trial name
- LHON (NCT02161380)
- Testing
- injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),, injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med), injection of scAAV2-P1ND4v2 2.4 X10e10vg (High)
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Byron Lam
Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease
A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age)…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis
- Trial name
- NCT04935879
- Testing
- Inclacumab
- Where
- 58 sites in United States, Brazil and 10 more
- Volunteers
- 241 people
- Sponsor
- Pfizer