Clinical trials: Genetic Mutation
Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations
Midostaurin and Decitabine in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia and FLT3 Mutation
This phase II trial studies how well midostaurin and decitabine work in treating older patients with newly diagnosed acute myeloid leukemia and FLT3 mutations.
- Conditions
- Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, FLT3 Tyrosine Kinase Domain Point Mutation, Secondary Acute Myeloid Leukemia
- Trial name
- NCT02634827
- Testing
- Decitabine, Laboratory Biomarker Analysis, Midostaurin
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Mayo Clinic
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
Neoplasms, Breast, Carcinoma of Breast
A Phase III Trial of Niraparib Versus Physician's Choice in HER2 Negative, Germline BRCA Mutation-positive Breast Cancer Patients
The purpose of this study is to compare progression-free survival (PFS) in patients with advanced/metastatic breast cancer who have a BRCA mutation when treated with niraparib as compared to those treated with physician's choice
- Conditions
- Neoplasms, Breast, Carcinoma of Breast, Human Epidermal Growth Factor 2 Negative Carcinoma of Breast, BRCA1 Gene Mutation
- Trial name
- BRAVO (NCT01905592)
- Testing
- niraparib, Physician's choice
- Where
- 104 sites in United States, Belgium and 12 more
- Volunteers
- 216 people
- Sponsor
- Tesaro, Inc.
Malignant Neoplasm
Ponatinib for Patients Whose Advanced Solid Tumor Cancer Has Activating Mutations Involving the Following Genes: FGFR1, FGFR2, FGFR3, FGFR4, RET, KIT.
This phase II trial studies how well ponatinib hydrochloride works in treating patients with cancer that has spread to other parts of the body (metastatic), has failed previous treatment (refractory), and has one of several…
- Trial name
- NCT02272998
- Testing
- ponatinib hydrochloride, laboratory biomarker analysis
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Sameek Roychowdhury
Major Depressive Disorder
Deplin® P.L.U.S. Program (Progress Through Learning Understanding & Support)
This study will be an observational study in which patients who have been prescribed Deplin® are invited to participate in surveys regarding their experiences with Deplin®.
- Trial name
- NCT01369030
- Testing
- Deplin®
- Where
- 2 sites in United States
- Volunteers
- 554 people, healthy volunteers welcome
- Sponsor
- Pamlab, Inc.
Carcinoma, Non-Small-Cell Lung
Trial of Continuous Once Daily Oral Treatment Using BIBW 2992 (Afatinib) Plus Sirolimus (Rapamune®) in Patients With Non-small Cell Lung Cancer Harbouring an EGFR Mutation and/or Disease Progression Following Prior Erlotinib or Gefitinib
The primary objective of this trial is to identify the Maximum Tolerated Dose of BIBW 2992 therapy when given continuously in combination with Sirolimus.
- Trial name
- NCT00993499
- Testing
- BIBW 2992, Sirolimus (rapamycin)
- Where
- 8 sites in Spain
- Volunteers
- 44 people
- Sponsor
- Boehringer Ingelheim
Atopic Dermatitis, Ichthyosis Vulgaris
Genetic Screening for Filaggrin Mutation in Atopic Dermatitis and Ichthyosis Vulgaris in the African American Population
The investigators' primary objective is to identify common and rare mutations in the filaggrin gene in African American patients with a diagnosis of atopic dermatitis and ichthyosis vulgaris.
- Trial name
- NCT01016106
- Testing
- Buccal Swab
- Where
- 2 sites in United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Northwestern University
Atherosclerosis
Arterial Function and Atherosclerosis in Essential Thrombocythemia
The aim of the study is to examine (a) whether patients with JAK2 V617F positive ET in comparison to age-and sex-matched, apparently healthy control subjects show more advanced progression of arterial stiffness, pulse-wave…
- Trial name
- NCT03828422
- Testing
- imaging
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- University Medical Centre Ljubljana
Melanoma
Comparison of AZD6244 in Combination With Dacarbazine Versus (vs) Dacarbazine Alone in BRAF Mutation Positive Melanoma Patients
To assess the efficacy in terms of overall survival of AZD6244 in combination with dacarbazine, compared with dacarbazine alone, in first line patients with BRAF mutation positive advanced cutaneous or unknown primary melanoma
- Trial name
- NCT00936221
- Testing
- AZD6244, Dacarbazine
- Where
- 38 sites in United States, Brazil and 10 more
- Volunteers
- 385 people
- Sponsor
- AstraZeneca
Relapsed or Refractory B-cell Non-Hodgkin's Lymphoma
Study of Tazemetostat in Participants With Relapsed or Refractory B-cell Non-Hodgkin's Lymphoma With EZH2 Gene Mutation
This is a multicenter, open-label, Phase 2 study to assess the efficacy and safety of tazemetostat in participants with relapsed or refractory B-cell non-Hodgkin's lymphoma (NHL) with EZH2 gene mutation.
- Trial name
- NCT03456726
- Testing
- Tazemetostat
- Where
- 28 sites in Japan
- Volunteers
- 20 people
- Sponsor
- Eisai Co., Ltd.
Locally Advanced Pancreatic Carcinoma, Metastatic Pancreatic Carcinoma
Binimetinib and Encorafenib for the Treatment of Pancreatic Cancer in Patients With a Somatic BRAF V600E Mutation
This phase II trial studies the side effects and how well the combination of binimetinib and encorafenib work in treating patients with pancreatic cancer with a somatic BRAF V600E mutation.
- Conditions
- Locally Advanced Pancreatic Carcinoma, Metastatic Pancreatic Carcinoma, Recurrent Pancreatic Carcinoma, Stage III Pancreatic Cancer AJCC v8
- Trial name
- NCT04390243
- Testing
- Binimetinib, Encorafenib
- Where
- 5 sites in United States
- Volunteers
- 6 people
- Sponsor
- Academic and Community Cancer Research United
Cystic Fibrosis
A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF…
- Trial name
- NCT03447249
- Testing
- VX-659/TEZ/IVA, IVA
- Where
- 101 sites in United States, Australia and 9 more
- Volunteers
- 385 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Advanced Non Small Cell Lung Cancer, Advanced (Inoperable) Non Small Cell Lung Cancer
Study to Determine the Effect of Food on the Blood Levels of AZD9291 Following Oral Dosing of a Tablet Formulation in Patients With Non-Small Cell Lung Cancer
This is a 2-part study in patients with epidermal growth factor receptor mutation positive (EGFRm+) non-small cell lung cancer (NSCLC) whose disease has progressed on treatment with an epidermal growth factor receptor…
- Trial name
- NCT02163733
- Testing
- AZD9291 tablets, Pharmacokinetic sampling - AZD9291, Dietary Fasted
- Where
- 13 sites in France, South Korea and 2 more
- Volunteers
- 38 people
- Sponsor
- AstraZeneca
Amyotrophic Lateral Sclerosis
An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)
The primary objectives of Parts A and B of this study are to evaluate the safety, tolerability, and pharmacokinetics (PK) of ascending doses of tofersen in adults with ALS and a documented superoxide dismutase 1 (SOD1) mutation.
- Trial name
- VALOR (Part C) (NCT02623699)
- Testing
- Tofersen
- Where
- 40 sites in United States, Australia and 10 more
- Volunteers
- 176 people
- Sponsor
- Biogen
Advanced Breast Cancer
Pilot Decentralized Clinical Trial in Men and Pre and Post-menopausal Women With Breast Cancer and a Specific Mutation (PIK3CA) Treated With Alpelisib in Combination With Fulvestrant
The study was designed to identify and register practical observations and experiences in connection with planning and implementing decentralized, patient-centered clinical trials at a geographic distance with virtual elements.
- Trial name
- TELEPIK (NCT04862143)
- Testing
- Alpelisib, Fulvestrant, Goserelin
- Where
- Sweden
- Volunteers
- 2 people
- Sponsor
- Novartis Pharmaceuticals
Non-Small Cell Lung Cancer, KRAS Mutation-Related Tumors
Binimetinib and Hydroxychloroquine in Patients With Advanced KRAS Mutant Non-Small Cell Lung Cancer
This study will evaluate using hydroxychloroquine (HCQ) along with binimetinib as an effective method for treating cancer. All patients will receive binimetinib at a standard dose approved for other cancers.
- Trial name
- NCT04735068
- Testing
- Binimetinib Pill, Hydroxychloroquine Pill
- Where
- United States
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Abramson Cancer Center at Penn Medicine
Nonsene Mutation Duchenne Muscular Dystrophy
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to <2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20…
- Trial name
- NCT04336826
- Testing
- Ataluren
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- PTC Therapeutics
NLRC4-MAS, XIAP Deficiency
Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency
This is a Phase 3 study to assess the safety and efficacy of Tadekinig alfa in patients with monogenic, interleukin-18 (IL 18) driven autoinflammation due to Nucleotide-binding oligomerization domain, leucine-rich repeat and…
- Trial name
- NCT03113760
- Testing
- Tadekinig alfa, 0.9% sodium chloride
- Where
- 9 sites in United States, Canada and 1 more
- Volunteers
- 15 people
- Sponsor
- AB2 Bio Ltd.
Multiple Myeloma, Neoplasms
A Screening Study to Detect BRAF V600 Mutation-Positive Patients For Enrollment Into Clinical Research Studies of Zelboraf (Vemurafenib)
This is a screening study to detect BRAF V600 mutation-positive patients for enrollment into clinical research studies of Zelboraf (vemurafenib).
- Trial name
- NCT01804140
- Where
- 20 sites in United States
- Volunteers
- 662 people
- Sponsor
- Genentech, Inc.
Solid Tumours
Japan PhI/II of GSK2118436 and GSK1120212 Combination in Subjects With BRAF V600E/K Mutation Positive Advanced Solid Tumors (Phase I Part) or Cutaneous Melanoma (Phase II Part)
This is a Japanese Phase I/II, open-label, non-controlled study to evaluate the safety, tolerability, pharmacokinetic profile, and efficacy of the combination of GSK2118436 and GSK1120212 in subjects with BRAF V600E/K mutation…
- Trial name
- NCT01928940
- Testing
- dabrafenib, trametinib
- Where
- 2 sites in Japan
- Volunteers
- 12 people
- Sponsor
- GlaxoSmithKline