Clinical trials: Mutation
Non-Squamous Non-Small Cell Lung Cancer
An Observational Study of Tarceva (Erlotinib) in First Line in Patients With Advanced EGFR Mutation-Positive Non-Small Cell Lung Cancer (GERTAC)
This observational study will evaluate the efficacy and safety of Tarceva (erlotinib) in first-line therapy in patients with locally advanced or metastatic EGFR mutation-positive non-small cell lung cancer.
- Trial name
- NCT01790217
- Where
- Germany
- Volunteers
- 52 people
- Sponsor
- Hoffmann-La Roche
Cystic Fibrosis
Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation
The purpose of this study is to evaluate the safety of long-term ivacaftor treatment in participants with cystic fibrosis (CF) from Studies 110 (NCT01614457), 111 (NCT01614470), and 113 (NCT01685801).
- Trial name
- KONTINUE (NCT01707290)
- Testing
- Ivacaftor
- Where
- 38 sites in United States, Belgium and 2 more
- Volunteers
- 125 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
NSCLC (Advanced Non-small Cell Lung Cancer)
GFH375 Monotherapy and Combination Therapy as First-Line Treatment for Advanced KRAS G12D-Mutant Non-Small Cell Lung Cancer
This study is a multicenter, open-label, randomized clinical trial aimed at exploring the efficacy and safety of three treatment regimens for treatment-naive advanced NSCLC patients with KRAS G12D mutation: GFH375 monotherapy…
- Trial name
- NCT07554859
- Testing
- ArmA:GFH375, Arm B:GFH375, Arm C:GFH375
- Where
- 2 sites in China
- Volunteers
- 90 people
- Sponsor
- Genfleet Therapeutics (Shanghai) Inc.
Fragile X Syndrome
Validation of Saliva in the Esoterix Genetic Laboratories Fragile X Assay
The purpose of this research is to determine if saliva samples can be used as an alternate sample type to test for fragile X. By using saliva instead of blood, it would be easier for patients to have fragile X testing.
- Trial name
- NCT01616589
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Esoterix Genetic Laboratories, LLC
Solid Tumor Cancer, PDAC
IN10018 in Combination With RNK08954 for the Treatment of KRASG12D Mutation-Positive Locally Advanced or Metastatic Solid Tumors
This is a multicenter, open-label, Phase Ib/II clinical study. The study includes Phase Ib-Dose Exploration Stage and Phase II - Efficacy Exploration and Determination Stage.
- Trial name
- NCT07441174
- Testing
- IN10018 in combination with RNK08954
- Where
- 14 sites in China
- Volunteers
- 92 people
- Sponsor
- InxMed (Shanghai) Co., Ltd.
Caucasian Patients With EGFR Mutation Positive Advanced NSCLC
Efficacy, Safety, Tolerability of Gefitinib as 1st Line in Caucasian Patients With EGFR Mutation Positive Advanced NSCLC
This study is carried out to see how Caucasian patients with lung cancer which has EGFR mutation will respond to gefitinib (IRESSA™) as a first line treatment.
- Trial name
- IFUM (NCT01203917)
- Testing
- Gefitinib
- Where
- 61 sites in Bulgaria, France and 11 more
- Volunteers
- 1,060 people
- Sponsor
- AstraZeneca
Low Grade Ovarian Serous Adenocarcinoma, Ovarian Cancer
A Study of Avutometinib (VS-6766) v. Avutometinib (VS-6766) + Defactinib in Recurrent Low-Grade Serous Ovarian Cancer With and Without a KRAS Mutation
This study will assess the safety and efficacy of avutometinib (VS-6766) monotherapy and in combination with defactinib in subjects with recurrent Low-Grade Serous Ovarian Cancer (LGSOC)
- Trial name
- RAMP 201 (NCT04625270)
- Testing
- avutometinib (VS-6766), avutometinib (VS-6766) and defactinib
- Where
- 47 sites in United States, Belgium and 5 more
- Volunteers
- 225 people
- Sponsor
- Verastem, Inc.
Myoclonus Epilepsies, Progressive
Safety, Blood Levels and Effects of AUT00201 in Patients With MEAK
A randomized, double-blind, placebo-controlled, crossover study to assess the safety, tolerability, and pharmacokinetics of single doses of AUT00201 at 100 mg or matching placebo in patients with myoclonus epilepsy and ataxia due…
- Trial name
- AUT022201 (NCT05873062)
- Testing
- AUT00201
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Autifony Therapeutics Limited
Advanced or Metastatic Solid Tumors Irrespective of Gene Alterations, Non-Small Cell Lung Cancer
Phase 1b/2 Study of Futibatinib in Combination With Binimetinib in Patients With Advanced KRAS Mutant Cancer
Phase 1b/2 study to evaluate the FGFRi futibatinib in combination with the MEKi binimetinib in patients with advanced KRASmt tumors.
- Conditions
- Advanced or Metastatic Solid Tumors Irrespective of Gene Alterations, Non-Small Cell Lung Cancer, KRAS Gene Mutation
- Trial name
- NCT04965818
- Testing
- Futibatinib and Binimetinib
- Where
- 3 sites in United States
- Volunteers
- 38 people
- Sponsor
- Taiho Oncology, Inc.
X-Linked Retinitis Pigmentosa
Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa
A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).
- Trial name
- NCT05874310
- Testing
- FT-002
- Where
- 2 sites in China
- Volunteers
- 18 people
- Sponsor
- Frontera Therapeutics
Relapsed Ovarian Cancer, BRCA Mutation, Platinum Sensitivity
Olaparib Treatment in Relapsed Germline Breast Cancer Susceptibility Gene (BRCA) Mutated Ovarian Cancer Patients Who Have Progressed at Least 6 Months After Last Platinum Treatment and Have Received at Least 2 Prior Platinum Treatments
Comparison of olaparib vs. physician's choice of single agent standard of care non-platinum based chemotherapy in patients with germline Breast Cancer susceptibility gene (gBRCA) mutated ovarian cancer who have progressed at…
- Trial name
- SOLO3 (NCT02282020)
- Testing
- OLAPARIB, Single agent chemotherapy
- Where
- 94 sites in United States, Argentina and 11 more
- Volunteers
- 266 people
- Sponsor
- AstraZeneca
Pancreatic Adenocarcinoma Metastatic, BRCA1 Mutation
SHARON: A Clinical Trial for Metastatic Cancer Using Chemotherapy and Patients' Own Stem Cells
The clinical trial is a phase 1, single-arm trial that will evaluate the safety of the investigational treatment on metastatic pancreatic cancer and metastatic breast cancer.
- Conditions
- Pancreatic Adenocarcinoma Metastatic, BRCA1 Mutation, BRCA2 Mutation, Pancreatic Acinar Cell Carcinoma
- Trial name
- SHARON (NCT04150042)
- Testing
- Melphalan, BCNU, Vitamin B12B
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- General Oncology, Inc.
Parkinson Disease
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Parkinson's Disease
The main goal of this clinical trial is to learn about the safety and tolerability of GT-02287. The questions it aims to answer are: What medical problems do participants have when taking GT-02287? How is GT-02287 absorbed…
- Trial name
- NCT06732180
- Testing
- GT-02287
- Where
- 7 sites in Australia
- Volunteers
- 20 people
- Sponsor
- Gain Therapeutics, Inc.
Non-small Cell Lung Cancer (NSCLC)
Sacituzumab Tirumotecan (MK-2870) Versus Pemetrexed and Carboplatin Combination Therapy in Participants With Epidermal Growth Factor (EGFR)-Mutated, Advanced Nonsquamous Non-small Cell Lung Cancer (NSCLC) Who Have Progressed on Prior EGFR Tyrosine Kinase Inhibitors (MK-2870-009)
The purpose of this study is to evaluate sacituzumab tirumotecan versus pemetrexed in combination with carboplatin for the treatment of epidermal growth factor receptor (EGFR)-mutated advanced non-squamous non-small cell lung…
- Trial name
- NCT06305754
- Testing
- Sacituzumab tirumotecan, Pemetrexed, Carboplatin
- Where
- 156 sites in United States, Argentina and 17 more
- Volunteers
- 550 people
- Sponsor
- Merck Sharp & Dohme LLC
Advanced Solid Tumors, Breast Cancer
A Phase 1 Study of ATV-1601 in Patients With Advanced Cancer That Have AKT1 E17K Mutations
This is a Phase 1, open-label study to evaluate the safety and tolerability of ATV-1601 administered orally in adults with AKT1 E17K-mutant, advanced solid tumors and also in HR+/HER2- advanced and metastatic breast cancer, with…
- Conditions
- Advanced Solid Tumors, Breast Cancer, Breast Carcinoma, Breast Neoplasms
- Trial name
- NCT07038369
- Testing
- ATV-1601, ATV-1601 + Fulvestrant
- Where
- 8 sites in United States, France and 2 more
- Volunteers
- 134 people
- Sponsor
- Atavistik Bio, Inc
Solid Tumor, Breast Tumor
Niraparib in the Treatment of Patients With Advanced PALB2 Mutated Tumors
The purpose of this study is to further evaluate the efficacy and safety of niraparib in patients with locally advanced or metastatic solid tumors and a pathogenic or likely pathogenic tumor PALB2 (tPALB2) mutation.
- Conditions
- Solid Tumor, Breast Tumor, Colon Tumor, Malignant, Lung Tumor
- Trial name
- PAVO (NCT05169437)
- Testing
- Niraparib
- Where
- 80 sites in United States
- Volunteers
- 22 people
- Sponsor
- Tempus AI
Non-small Cell Lung Cancer
A Confirmatory Clinical Study in NSCLC Patients With MET Exon 14 Mutation (KUNPENG-2)
The goal of this clinical trial is to test if PLB1001 works well and safely in Non-small cell lung cancer patients with MET exon 14 mutation.
- Trial name
- KUNPENG-2 (NCT05989542)
- Testing
- PLB1001
- Where
- China
- Volunteers
- 136 people
- Sponsor
- Beijing Pearl Biotechnology Limited Liability Company
Acute Myeloid Leukemia, Relapsed Acute Myeloid Leukemia
A Study to Evaluate Lanraplenib (LANRA) in Combination With Gilteritinib in Participants With FLT3-mutated Relapsed or Refractory Acute Myeloid Leukemia (AML)
The primary objective of this study is to evaluate the safety of lanraplenib (LANRA) in combination with the FMS-like tyrosine kinase 3 (FLT3) inhibitor gilteritinib, in participants with relapsed or refractory (R/R) FLT3-mutated…
- Conditions
- Acute Myeloid Leukemia, Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
- Trial name
- NCT05028751
- Testing
- Lanraplenib, Gilteritinib
- Where
- 17 sites in United States, Spain
- Volunteers
- 24 people
- Sponsor
- Kronos Bio
Amino Acid Metabolism, Inborn Errors
Ataluren for Nonsense Mutation Methylmalonic Acidemia
Methylmalonic acidemia (MMA) is a rare genetic disorder caused by mutations in the gene for mitochondrial enzyme methylmalonyl-CoA mutase (MCM) or in one of the genes for adenosylcobalamin (AdoCbl).
- Trial name
- NCT01141075
- Testing
- Ataluren
- Where
- 9 sites in Belgium, France and 4 more
- Volunteers
- 11 people
- Sponsor
- PTC Therapeutics
Non-small Cell Lung Cancer
A Phase 1b/2 Study of Safety and Efficacy of Rociletinib in Combination With MPDL3280A in Patients With Advanced or Metastatic EGFR-mutant NSCLC
This clinical research study is being carried out in two parts, Phase 1 and Phase 2. The primary purpose of the Phase 1 portion of the study is to observe the safety of the combination of rociletinib and MPDL3280A in EGFR-mutant…
- Trial name
- NCT02630186
- Testing
- Rociletinib, MPDL3280A
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Clovis Oncology, Inc.