Clinical trials: Genetic Mutation
Cystic Fibrosis
A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
To evaluate the efficacy of ivacaftor treatment, as measured by lung clearance index (LCI), in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation
- Trial name
- NCT02742519
- Testing
- ivacaftor
- Where
- 6 sites in Australia, Canada and 1 more
- Volunteers
- 14 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
EGFR Mutation Status in aNSCLC Patients (Locally Advanced and/or Metastatic Disease) With Adenocarcinoma and Non-adenocarcinoma Histologies.
Europe-Japan Diagnostic Study for EGFR Testing
This is a non-interventional diagnostic, international, multicenter and non-comparative study of EGFR mutation status in aNSCLC patients (locally advanced and/or metastatic disease) with adenocarcinoma and non-adenocarcinoma…
- Trial name
- ASSESS (NCT01785888)
- Where
- 57 sites in France, Germany and 6 more
- Volunteers
- 1,311 people
- Sponsor
- AstraZeneca
Cancer
A Rollover Study to Provide Continued Treatment With GSK2118436 to Subjects With BRAF Mutation-Positive Tumors
This rollover study is designed to provide continued access to GSK2118436 for eligible subjects with BRAF mutation-positive tumors who have previously participated in a GlaxoSmithKline (GSK)-sponsored GSK2118436 study (parent…
- Trial name
- NCT01231594
- Testing
- GSK2118436, GSK1120212, Other approved anti-cancer agent
- Where
- 44 sites in United States, Australia and 7 more
- Volunteers
- 214 people
- Sponsor
- Novartis Pharmaceuticals
Non-small-cell Lung Cancer
Icotinib as Adjuvant Therapy in Treating Non-small-cell Lung Cancer Patients With Positive EGFR Mutation
This study is designed to evaluate the efficacy of icotinib as adjuvant therapy in treating such patients. The primary endpoint is to compare the recurrence-free survival after 1-year or 2-year treatment with icotinib.
- Trial name
- NCT01929200
- Testing
- 1-year treatment with icotinib, 2-year treatment with icotinib
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Ovarian Cancer
The Clinical Trial to Evaluate the Pharmacokinetics, Safety and Tolerability of ZL-2306 (Niraparib) in Patients With Ovarian Cancer
Niraparib is a potent and highly selective PARP-1/-2 inhibitor. The primary objective of this trial is to evaluate the pharmacokinetic (PK) properties of ZL-2306 (niraparib) and its metabolite M1 in patients from Mainland China…
- Trial name
- NCT03551171
- Testing
- ZL-2306 (niraparib)
- Where
- 6 sites in China
- Volunteers
- 42 people
- Sponsor
- Zai Lab (Shanghai) Co., Ltd.
Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD)
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able to improve physical abilities of…
- Conditions
- Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
- Trial name
- KH176-301 (NCT06451757)
- Testing
- Sonlicromanol
- Where
- 10 sites in United States, Denmark and 5 more
- Volunteers
- 220 people
- Sponsor
- Khondrion BV
Leukemia
Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia
The purpose of this study was to determine the efficacy and safety of twice daily (bid) oral midostaurin in patients with Aggressive Systemic Mastocytosis (ASM) or Mast Cell Leukemia (MCL) with or without an Associated…
- Trial name
- NCT00782067
- Testing
- Midostaurin (PKC412)
- Where
- 29 sites in United States, Australia and 10 more
- Volunteers
- 116 people
- Sponsor
- Novartis Pharmaceuticals
Non-small Cell Lung Cancer
A Study of JAB-21822 in Advanced or Metastatic NSCLC With KRAS p.G12C and STK11 Co-mutation and Wild-type KEAP1
Evaluate the safety and tolerability, drug levels, and clinical activity of JAB-21822 in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) whose tumors with KRAS p.G12C mutation and a…
- Trial name
- NCT05276726
- Testing
- JAB 21822
- Where
- 21 sites in China
- Volunteers
- 104 people
- Sponsor
- Allist Pharmaceuticals, Inc.
Myelofibrosis
A Study of APG-1252 in Patients With Myelofibrosis Who Progressed After Initial Therapy
The study is a designed to evaluate safety and activity of APG-1252 when administered as monotherapy and in combination with ruxolitinib in previously ruxolitinib treated myelofibrosis patients.
- Trial name
- NCT04354727
- Testing
- APG-1252, Ruxolitinib
- Where
- 2 sites in United States
- Sponsor
- Ascentage Pharma Group Inc.
Leukemia, Myeloid
Open Study of CEP-701 in Patients With Refractory Acute Myeloid Leukemia With FLT-3 Mutation
The purpose of this study is to determine the response rate of patients with refractory, relapsed or poor risk AML expressing FLT-3 activating mutations, when administered CEP-701 at a dosage of 60 mg 2 times a day.
- Trial name
- NCT00030186
- Testing
- CEP-701 60mg, Cep-701 80mg, Cep-701 40mg
- Volunteers
- 37 people
- Sponsor
- Cephalon
Cystic Fibrosis
A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibrosis Who Have an R117H-CFTR Mutation, Including Pediatric Patients
The purpose of this study is to confirm the long-term safety and effectiveness of Kalydeco® (ivacaftor) in US CF patients with the R117H-CFTR mutation <18 years of age and to describe the long-term safety and effectiveness of…
- Trial name
- NCT02722057
- Volunteers
- 368 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Non-Squamous Non-Small Cell Lung Cancer
A Study to Assess the Incidence of EGFR Mutation in Patients With Newly Diagnosed Locally Advanced or Metastatic Non-Small Cell Lung Cancer in the UK, And of Tarceva (Erlotinib) as First-Line Therapy in EGFR Mutation Positive Patients.
This study will assess the prevalence of epidermal growth factor receptor (EGFR) mutations in newly diagnosed patients with locally advanced or metastatic non-small cell lung cancer (NSCLC).
- Trial name
- NCT01250119
- Testing
- erlotinib [Tarceva]
- Where
- 22 sites in United Kingdom
- Volunteers
- 688 people
- Sponsor
- Hoffmann-La Roche
Solid Tumor
PF-07284892 in Participants With Advanced Solid Tumors
The purpose of this first-in-patient, open label study is to determine the maximum tolerated dose and/or recommended dose for further study of PF-07284892 as a single agent and in combination with lorlatinib, encorafenib and…
- Trial name
- NCT04800822
- Testing
- PF-07284892, lorlatinib, binimetinib
- Where
- 11 sites in United States
- Volunteers
- 53 people
- Sponsor
- Pfizer
Cystic Fibrosis
Lumacaftor/Ivacaftor Combination Therapy in Subjects With CF Who Have an A455E CFTR Mutation
This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter, crossover study that will evaluate the efficacy of LUM/IVA in subjects with CF 12 years of age and older who have at least one A455E mutation.
- Trial name
- NCT03061331
- Testing
- LUM/IVA
- Where
- 2 sites in Netherlands
- Volunteers
- 20 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
KRAS G12C Mutant Solid Tumors, Carcinoma, Non-Small-Cell Lung
Study of JDQ443 in Patients With Advanced Solid Tumors Harboring the KRAS G12C Mutation
This is a phase Ib/II open label study. The escalation part will characterize the safety and tolerability of JDQ443 single agent and JDQ443 in combination with the other study treatments (TNO155 and tislelizumab) in advanced…
- Conditions
- KRAS G12C Mutant Solid Tumors, Carcinoma, Non-Small-Cell Lung, Carcinoma, Colorectal, Cancer of Lung
- Trial name
- KontRASt-01 (NCT04699188)
- Testing
- JDQ443, TNO155, tislelizumab
- Where
- 38 sites in United States, Australia and 14 more
- Volunteers
- 344 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
This is an open-label extension study for participants who completed a Phase 3, placebo-controlled study of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.
- Trial name
- NCT02456103
- Testing
- Ataluren
- Where
- 70 sites in United States, Argentina and 14 more
- Volunteers
- 246 people
- Sponsor
- PTC Therapeutics
Solid Tumor, RAS Mutation
Evaluation of RAS Inhibitor Treatment in Participants With Advanced or Metastatic Solid Tumors Harboring RAS Mutations
This is a phase I study to evaluate the safety, tolerability, pharmacokinetics, and anti-tumor activity of ASKC189 in in Participants With Advanced or Metastatic Solid Tumors Harboring RAS Mutations
- Trial name
- NCT07836036
- Testing
- ASKC189
- Where
- 2 sites in China
- Volunteers
- 200 people
- Sponsor
- Jiangsu Aosaikang Pharmaceutical Co., Ltd.
Advanced Non-small Cell Lung Cancer, EGFR Mutation
A Study to Learn About How BAY2927088 Affects the Level of Dabigatran or Rosuvastatin in the Blood When These Drugs Are Taken Together in Healthy Participants
Researchers are looking for a better way to treat people who have advanced non-small cell lung cancer (NSCLC) with specific genetic changes called epidermal growth factor receptor (EGFR) and human epidermal growth factor receptor…
- Conditions
- Advanced Non-small Cell Lung Cancer, EGFR Mutation, HER2 Mutation, Healthy Volunteers
- Trial name
- NCT06329895
- Testing
- BAY2927088, Dabigatran etexilate, Rosuvastatin
- Where
- United Kingdom
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Bayer
Non-Small Cell Lung Cancer, EGFR Gene Mutation
D-0316 Versus Icotinib in Patients With Locally Advanced or Metastatic EGFR Sensitising Mutation Positive NSCLC
To assess the efficacy and safety of D-0316 versus Icotinib, a standard of care epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKI), in patients with locally advanced or Metastatic Non Small Cell Lung Cancer…
- Trial name
- NCT04206072
- Testing
- D-0316 Capsule, Icotinib Hydrochloride Tablets
- Where
- 2 sites in China
- Volunteers
- 362 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Pancreatic Cancer, Pancreatic Cancer Metastatic
Study of Daraxonrasib and Daraxonrasib + GnP as First-line Treatment in Patients With Metastatic Pancreatic Adenocarcinoma
The purpose of this study is to evaluate the safety and efficacy of an investigational RAS(ON) inhibitor administered as monotherapy or in combination with chemotherapy, compared with standard of care (SOC) chemotherapy alone.
- Conditions
- Pancreatic Cancer, Pancreatic Cancer Metastatic, PDAC, PDAC - Pancreatic Ductal Adenocarcinoma
- Trial name
- RASolute 303 (NCT07491445)
- Testing
- daraxonrasib, gemcitabine, nab-paclitaxel
- Where
- 82 sites in United States, Australia and 14 more
- Volunteers
- 900 people
- Sponsor
- Revolution Medicines, Inc.
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