Clinical trials: Mutations
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
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
Gynecologic Neoplasms, Epithelial Ovarian Cancer
PLX2853 as a Single Agent in Advanced Gynecological Malignancies and in Combination With Carboplatin in Platinum-Resistant Epithelial Ovarian Cancer
The purpose of this research study is to evaluate safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of the investigational drug PLX2853 in Advanced Gynecological Malignancies with a Known ARID1A Mutation and…
- Trial name
- NCT04493619
- Testing
- PLX2853, Carboplatin
- Where
- 9 sites in United States, Canada
- Volunteers
- 37 people
- Sponsor
- Opna Bio LLC
Solid Tumors, KRAS Mutation
A Study to Test Different Doses of BI 3011441 in Japanese People With Different Types of Advanced Cancer (NRAS/KRAS Mutation Positive)
This study is open to Japanese adults with different types of advanced cancer that are positive for NRAS/KRAS mutations. This is a study in people for whom previous treatment was not successful or no standard treatment exists.
- Trial name
- NCT04742556
- Testing
- BI 3011441
- Where
- 3 sites in Japan
- Volunteers
- 15 people
- Sponsor
- Boehringer Ingelheim
Carcinoma, Non-Small-Cell Lung
A Study of Lazertinib in Participants With Epidermal Growth Factor Receptor (EGFR) Mutation Positive Advanced Non-Small Cell Lung Cancer (NSCLC)
The main purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of Lazertinib when given orally to participants with epidermal growth factor receptor single activating mutation positive (EGFRm+)…
- Trial name
- NCT04075396
- Testing
- Lazertinib
- Where
- 9 sites in United States, Spain and 1 more
- Volunteers
- 29 people
- Sponsor
- Janssen Research & Development, LLC
Myelofibrosis With High Molecular Risk Mutations
Phase III Study Investigating the Efficacy and Safety of Ruxolitinib in Early Myelofibrosis Patients With High Molecular Risk Mutations.
Myelofibrosis patients with high molecular risk mutations have an intrinsically aggressive disease with increased risk of leukemic transformation and reduced overall survival.
- Trial name
- ReTHINK (NCT02598297)
- Testing
- Ruxolitinib
- Where
- 110 sites in Australia, Austria and 23 more
- Volunteers
- 49 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation
This study evaluates the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 years and older, homozygous or heterozygous for the F508del…
- Trial name
- NCT03537651
- Testing
- TEZ/IVA, IVA
- Where
- 55 sites in United States, Australia and 9 more
- Volunteers
- 130 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
TSC1, TSC2
Everolimus for Cancer With TSC1 or TSC2 Mutation
In this research study, the investigators are evaluating the clinical benefit of everolimus in cancer patients with inactivating TSC1 or TSC2 mutations or activating MTOR mutations.
- Conditions
- TSC1, TSC2, Tuberous Sclerosis Complex, MTOR
- Trial name
- NCT02201212
- Testing
- Everolimus
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Dana-Farber Cancer Institute
Metastatic Non Small Cell Lung Cancer
Dacomitinib for Treatment of Patients in India With Metastatic Non Small Cell Lung Cancer With EGFR Activating Mutations
This is a Phase 4, open label, single arm, multi-center, prospective clinical trial of dacomitinib that will be performed in India.
- Trial name
- NCT04511533
- Testing
- Dacomitinib
- Where
- 15 sites in India
- Volunteers
- 101 people
- Sponsor
- Pfizer
Melanoma
Combined BRAF-Targeted Therapy & Immunotherapy for Melanoma
This research study is a Phase II clinical trial of an investigational combination of drugs (vemurafenib and aldesleukin) to learn whether the combination works in treating a specific cancer.
- Trial name
- NCT01754376
- Testing
- Aldesleukin, Vemurafenib
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Massachusetts General Hospital
Chronic Myelomonocytic Leukemia (CMML)
Cobimetinib in Newly Diagnosed or HMA-treated CMML Patients With RAS Pathway Mutations
This is an open-label, nonrandomized phase 2 trial to assess the efficacy of cobimetinib in RAS pathway activated CMML. All eligible patients will be treated daily with cobimetinib in 28-day cycles.
- Trial name
- CONCERTO (NCT04409639)
- Testing
- Cobimetinib
- Where
- 3 sites in United States
- Volunteers
- 14 people
- Sponsor
- University of Utah
Cystic Fibrosis
Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
The purpose of this study is to evaluate of the safety, efficacy, pharmacokinetics (PK) and pharmacodynamic (PD) effects of lumacaftor (VX-809) alone and when coadministered with ivacaftor (VX-770) in participants with cystic…
- Trial name
- NCT01225211
- Testing
- Lumacaftor, Ivacaftor
- Where
- 55 sites in United States, Australia and 5 more
- Volunteers
- 312 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Grade 2 Glioma, Residual Glioma
Study of Vorasidenib (AG-881) in Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 or IDH2 Mutation (INDIGO)
Study AG881-C-004 is a phase 3, multicenter, randomized, double-blind, placebo-controlled study comparing the efficacy of vorasidenib to placebo in participants with residual or recurrent Grade 2 glioma with an IDH1 or IDH2…
- Conditions
- Grade 2 Glioma, Residual Glioma, Recurrent Glioma
- Trial name
- NCT04164901
- Testing
- Vorasidenib
- Where
- 84 sites in United States, Canada and 9 more
- Volunteers
- 331 people
- Sponsor
- Institut de Recherches Internationales Servier
Chronic Myeloid Leukemia
Homoharringtonine (Omacetaxine Mepesuccinate) in Treating Patients With Chronic Myeloid Leukemia (CML) With the T315I BCR-ABL Gene Mutation
To evaluate the safety and efficacy of subcutaneous administration of omacetaxine mepesuccinate (HHT) in achieving a clinical response in CML patients in chronic, accelerated, or blast phase who have failed prior imatinib therapy…
- Trial name
- NCT00375219
- Testing
- Omacetaxine mepesuccinate
- Where
- 32 sites in United States, Canada and 8 more
- Volunteers
- 103 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Cystic Fibrosis
Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
This study evaluated the long-term safety, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are…
- Trial name
- NCT04058366
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 84 sites in United States, Australia and 10 more
- Volunteers
- 251 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Melanoma
Adjuvant Dabrafenib (GSK2118436) in Patients With Surgically Resected AJCC Stage IIIC Melanoma Characterized by a BRAFV600E/K Mutation
In this study, the investigator's want to find out if dabrafenib can stop stage IIIC melanoma from coming back after surgery.
- Trial name
- NCT01682213
- Testing
- Dabrafenib
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Duchenne Muscular Dystrophy
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Trial name
- NCT00759876
- Testing
- Ataluren
- Where
- 3 sites in United States
- Volunteers
- 36 people
- Sponsor
- PTC Therapeutics
Bile Duct Adenocarcinoma, Fanconi Anemia Complementation Group Gene Mutation
Olaparib in Treating Patients With Metastatic Biliary Tract Cancer With Aberrant DNA Repair Gene Mutations
This phase II trial studies how well olaparib works in treating patients with biliary tract cancer that has spread to other places in the body (metastatic) and with aberrant DNA repair gene mutations.
- Conditions
- Bile Duct Adenocarcinoma, Fanconi Anemia Complementation Group Gene Mutation, Metastatic Bile Duct Carcinoma, PTEN Gene Deletion
- Trial name
- NCT04042831
- Testing
- Biospecimen Collection, Computed Tomography, Magnetic Resonance Imaging
- Where
- 5 sites in United States
- Volunteers
- 32 people
- Sponsor
- Academic and Community Cancer Research United
Endometrial Adenocarcinoma, Endometrial Adenosquamous Carcinoma
Akt Inhibitor MK2206 in Treating Patients With Recurrent or Advanced Endometrial Cancer
This phase II trial studies how well Akt inhibitor MK2206 works in treating patients with recurrent or advanced endometrial cancer.
- Conditions
- Endometrial Adenocarcinoma, Endometrial Adenosquamous Carcinoma, Endometrial Clear Cell Adenocarcinoma, Endometrial Serous Adenocarcinoma
- Trial name
- NCT01307631
- Testing
- Akt Inhibitor MK2206, Laboratory Biomarker Analysis
- Where
- 7 sites in United States
- Volunteers
- 37 people
- Sponsor
- National Cancer Institute (NCI)
Non-Small Cell Lung Carcinoma
Study of Capmatinib in Indian Patients With MET Exon 14 Skipping Mutation Positive Advanced NSCLC.
The Drugs Controller General of India (DCGI) granted approval for Rahika® (capmatinib) film-coated tablets (150 mg and 200 mg) for the treatment of adult patients with advanced or metastatic non-small cell lung cancer (NSCLC)…
- Trial name
- NCT05110196
- Testing
- Capmatinib 150 mg, Capmatinib 200 mg
- Where
- 15 sites in India
- Volunteers
- 50 people
- Sponsor
- Novartis Pharmaceuticals