Clinical trials: Genetic Mutation
Common Variable Immunodeficiency (CVID), APDS / PASLI
Study of Efficacy of CDZ173 in Patients With APDS/PASLI
This study was designed to explore CDZ173, a selective PI3Kδ inhibitor, in patients with genetically activated PI3Kδ, i.e., patients with Activated phosphoinositide 3-kinase delta syndrome/ p110δ-activating mutation causing…
- Trial name
- NCT02435173
- Testing
- CDZ173
- Where
- 10 sites in United States, Belarus and 7 more
- Volunteers
- 37 people
- Sponsor
- Novartis Pharmaceuticals
Cancer, Cancer Metastatic
A Basket Trial of an ERK1/2 Inhibitor (LY3214996) in Combination With Abemaciclib.
The purpose of CTO-IUSCC-0730 study is to assess the clinical efficacy of LY3214996 in combination with abemaciclib at the recommended phase 2 dose of LY3214996 200 mg orally daily and abemaciclib 150 mg orally twice daily.
- Conditions
- Cancer, Cancer Metastatic, BRAF V600E, MEK1 Gene Mutation
- Trial name
- NCT04534283
- Testing
- Abemaciclib, LY3214996
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Anita Turk
Cystic Fibrosis
A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation
To evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF), homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation
- Trial name
- NCT02514473
- Testing
- VX-809, VX-770
- Where
- 53 sites in United States, Australia and 7 more
- Volunteers
- 206 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
NF1, Neurofibromatosis
Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults
This study, "A Phase II Study of Cabozantinib (XL l84) for Plexiform Neurofibromas in Subjects with Neurofibromatosis Type I in Children and Adults diagnosed with Neurofibromatosis Type 1 (NF1) and have a type of tumor called a…
- Conditions
- NF1, Neurofibromatosis, Plexiform Neurofibromas
- Trial name
- NF105-CABO (NCT02101736)
- Testing
- Cabozantinib
- Where
- 12 sites in United States
- Volunteers
- 45 people
- Sponsor
- University of Alabama at Birmingham
BRCA1 Mutation, BRCA2 Mutation
Pre-Salpingo-Oophorectomy Pilot Study of MAKO 7 Device Performance
This study evaluates the ability of the MAKO 7 device to collect various cells
- Conditions
- BRCA1 Mutation, BRCA2 Mutation, Atypia Suspicious for Malignancy
- Trial name
- NCT02974842
- Testing
- MAKO 7
- Where
- 3 sites in United States
- Volunteers
- 50 people
- Sponsor
- nVision Medical
Leukemia, Acute Myeloid (AML)
A Study of ASP2215 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-like Tyrosine Kinase (FLT3) Mutation
The purpose of this study was to determine the clinical benefit of ASP2215 therapy in participants with FMS-like tyrosine kinase (FLT3) mutated acute myeloid leukemia (AML) who were refractory to or had relapsed after first-line…
- Trial name
- NCT02421939
- Testing
- gilteritinib, LoDAC (Low Dose Cytarabine), Azacitidine
- Where
- 126 sites in United States, Belgium and 12 more
- Volunteers
- 371 people
- Sponsor
- Astellas Pharma Global Development, Inc.
Advanced Cholangiocarcinoma, FGFR2 Gene Mutation
Phase 3 Study of BGJ398 (Oral Infigratinib) in First Line Cholangiocarcinoma With FGFR2 Gene Fusions/Translocations
Infigratinib is an oral drug which selectively binds to fibroblast growth factor receptor (FGFR) 2 and is being developed to treat participants with FGFR2 mutated cholangiocarcinoma.
- Trial name
- NCT03773302
- Testing
- BGJ398, Gemcitabine, Cisplatin
- Where
- 116 sites in United States, Australia and 13 more
- Volunteers
- 48 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Recurrent Non-Small Cell Lung Carcinoma, Stage IV Non-Small Cell Lung Cancer
S1403, Afatinib Dimaleate With or Without Cetuximab in Treating Patients With Newly Diagnosed Stage IV or Recurrent, EGFR Mutation Positive Non-small Cell Lung Cancer
This randomized phase II/III trial studies how well afatinib dimaleate with cetuximab works and compares it with afatinib dimaleate alone in treating patients with newly diagnosed stage IV or recurrent (has come back), epidermal…
- Trial name
- NCT02438722
- Testing
- Afatinib Dimaleate, Cetuximab, Laboratory Biomarker Analysis
- Where
- 638 sites in United States
- Volunteers
- 174 people
- Sponsor
- SWOG Cancer Research Network
Duchenne Muscular Dystrophy
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is designed to evaluate the ability of ataluren to increase dystrophin protein levels in muscle cells of participants with nmDMD.
- Trial name
- NCT03648827
- Testing
- Ataluren
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- PTC Therapeutics
Non Small Cell Lung Cancer
Atezolizumab Plus Bevacizumab in First Line NSCLC Patients
This is a multi-center phase II clinical trial of atezolizumab in combination with bevacizumab as first line treatment for locally advanced or metastasic high-intermediate tumour mutation burden selected NSCLC patients.
- Trial name
- TELMA (NCT03836066)
- Testing
- Atezolizumab-Bevacizumab
- Where
- 25 sites in Spain
- Volunteers
- 41 people
- Sponsor
- Fundación GECP
Cystic Fibrosis
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of VX-661/Ivacaftor in Pediatric Subjects With Cystic Fibrosis (CF)
This is a Phase 3, 2-part (Part A and Part B), open label, multicenter study evaluating the pharmacokinetic (PK), safety, and tolerability of multiple doses of tezacaftor (TEZ) in combination with ivacaftor (IVA) in subjects 6…
- Trial name
- NCT02953314
- Testing
- TEZ, TEZ/IVA, IVA
- Where
- 33 sites in United States, Canada
- Volunteers
- 83 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Hodgkin Lymphoma
Tazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With EZH2, SMARCB1, or SMARCA4 Gene Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well tazemetostat works in treating patients with brain tumors, solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that have come back (relapsed) or do not respond to…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Hodgkin Lymphoma, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Hodgkin Lymphoma
- Trial name
- NCT03213665
- Testing
- Tazemetostat
- Where
- 117 sites in United States, Puerto Rico
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)
Non-Small Cell Lung Cancer
Study For Patients With NSCLC EGFR Mutations (Del 19 or L858R +/- T790M)
This is a Phase 1/2 study of PF-06747775 as a single agent and in combination with other cancer treatments in patients with advanced EGFRm NSCLC.
- Trial name
- NCT02349633
- Testing
- PF-06747775, Palbociclib, Avelumab
- Where
- 15 sites in United States, Australia and 2 more
- Volunteers
- 65 people
- Sponsor
- Pfizer
Glioblastoma
Adult Study: ABT-414 Alone or ABT-414 Plus Temozolomide vs. Lomustine or Temozolomide for Recurrent Glioblastoma Pediatric Study: Evaluation of ABT-414 in Children With High Grade Gliomas
This study was conducted to evaluate the efficacy and safety of depatuxizumab mafodotin (ABT-414) alone or with temozolomide versus temozolomide or lomustine alone in adult participants with recurrent glioblastoma.
- Trial name
- INTELLANCE-2 (NCT02343406)
- Testing
- Depatuxizumab mafodotin, Temozolomide, Lomustine
- Where
- 102 sites in United States, Australia and 19 more
- Volunteers
- 266 people
- Sponsor
- AbbVie
Newly Diagnosed Acute Myeloid Leukemia (AML), Untreated AML
Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation
Study AG120-C-009 is a global, Phase 3, multicenter, double-blind, randomized, placebo-controlled clinical trial to evaluate the efficacy and safety of AG-120 (ivosidenib) + azacitidine vs placebo + azacitidine in adult…
- Conditions
- Newly Diagnosed Acute Myeloid Leukemia (AML), Untreated AML, AML Arising From Myelodysplastic Syndrome (MDS), Leukemia, Myeloid, Acute
- Trial name
- AGILE (NCT03173248)
- Testing
- AG-120, Azacitidine
- Where
- 90 sites in United States, Australia and 18 more
- Volunteers
- 146 people
- Sponsor
- Institut de Recherches Internationales Servier
Melanoma
The Effects of Treatment With Vemurafenib on the Immune System in Advanced Melanoma
Approximately 40-60 % of cutaneous melanomas select for a mutation in a protein called BRAF which is part of a signaling pathway called the Mitogen Activated Protein Kinase (MAPK) pathway.
- Trial name
- NCT01942993
- Testing
- Vemurafenib
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Philip Friedlander
Breast Cancer
Study of Safety and Efficacy of Dapagliflozin + Metformin XR Versus Metformin XR in Participants With HR+, HER2-, Advanced Breast Cancer While on Treatment With Alpelisib and Fulvestrant
This study was designed to assess the safety and efficacy of the combination of dapagliflozin plus metformin extended release (XR) compared with metformin XR during treatment with alpelisib plus fulvestrant in participants with…
- Trial name
- EPIK-B4 (NCT04899349)
- Testing
- Alpelisib, Fulvestrant, Metformin XR
- Where
- 2 sites in United States, Malaysia
- Volunteers
- 2 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F)
- Trial name
- NCT03525548
- Testing
- VX-445/TEZ/IVA, TEZ/IVA, IVA
- Where
- 44 sites in United States, Belgium and 2 more
- Volunteers
- 113 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Advanced Solid Tumor
Berzosertib Human Mass Balance Study (DDRiver Solid Tumors 208)
The study was conducted in two periods, Period 1 (mass balance) and Period 2 (extension). The purpose of Period 1 of this study was to provide a quantitative characterization of the mass balance, rates and routes of elimination…
- Trial name
- NCT05246111
- Testing
- [14C]Berzosertib, Berzosertib, Topotecan
- Where
- 2 sites in Hungary
- Volunteers
- 6 people
- Sponsor
- Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
Testing Binimetinib as a Potential Targeted Treatment in Cancers With NRAS Genetic Changes (MATCH-Subprotocol Z1A)
This phase II MATCH treatment trial investigates the good and bad effects of binimetinib in patients whose cancer has a genetic change called NRAS mutation.
- Conditions
- Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Hematopoietic and Lymphoid Cell Neoplasm, Refractory Lymphoma
- Trial name
- NCT04439344
- Testing
- Binimetinib
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- National Cancer Institute (NCI)