Clinical trials: Gene Mutations
Thalassemia
iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations
This is a single centre、single arm、open-label study，to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.
- Trial name
- NCT03728322
- Testing
- iHSCs treatment group
- Volunteers
- 12 people
- Sponsor
- Allife Medical Science and Technology Co., Ltd.
Advanced Solid Tumor, Metastatic Solid Tumors
Study of TPX-0022 in Patients With Advanced NSCLC, Gastric Cancer or Solid Tumors Harboring Genetic Alterations in MET
A phase 1/2, first-in-human, open-label study of the safety, tolerability, PK, and efficacy of the novel MET/CSF1R/SRC inhibitor TPX-0022 in adult subjects with advanced or metastatic NSCLC, Gastric Cancer, or solid tumors…
- Conditions
- Advanced Solid Tumor, Metastatic Solid Tumors, MET Gene Alterations
- Trial name
- SHIELD-1 (NCT03993873)
- Testing
- elzovantinib (TPX-0022)
- Where
- 24 sites in United States, France and 3 more
- Volunteers
- 95 people
- Sponsor
- Turning Point Therapeutics, Inc.
Advanced Non-small Cell Lung Cancer, EGFR Mutation
First in Human Study of BAY2927088 in Participants Who Have Advanced Non-small Cell Lung Cancer (NSCLC) With Mutations in the Genes of Epidermal Growth Factor Receptor (EGFR) and/or Human Epidermal Growth Factor Receptor 2 (HER2)
Researchers are looking for a better way to treat people who have advanced non-small cell lung cancer (NSCLC), a group of lung cancers that have spread to nearby tissues or to other parts of the body.
- Conditions
- Advanced Non-small Cell Lung Cancer, EGFR Mutation, HER2 Mutation
- Trial name
- NCT05099172
- Testing
- BAY2927088_formulation A, BAY2927088_formulation B_1, BAY2927088_formulation B_2
- Where
- 94 sites in United States, Belgium and 14 more
- Volunteers
- 430 people
- Sponsor
- Bayer
Non-small Cell Lung Cancer
An Open Study of ASP8273 in Patients With Non-Small-Cell Lung Cancer (NSCLC) Who Have Epidermal Growth Factor Receptor (EGFR) Mutations
Purpose of the study is to determine the following in patients with non-small cell lung cancer (NSCLC) harboring EGFR activating mutations. the safety and tolerability of ASP8273. the pharmacokinetics (PK) of ASP8273.
- Trial name
- NCT02192697
- Testing
- ASP8273
- Where
- 14 sites in Japan, South Korea and 1 more
- Volunteers
- 124 people
- Sponsor
- Astellas Pharma Inc
NSCLC, EGFR Activating Mutation
TY-9591 in the Patients With EGFR Mutations in Advanced NSCLC With Brain Metastases
This study is to evaluate the efficacy and safety of TY-9591 in first-line treatment of patients with EGFR-sensitive mutation-positive non-small cell lung cancer with brain metastases compared to Osimertinib.
- Conditions
- NSCLC, EGFR Activating Mutation, Brain Metastases
- Trial name
- NCT05948813
- Testing
- TY-9591, Osimertinib
- Where
- China
- Volunteers
- 420 people
- Sponsor
- TYK Medicines, Inc
Non-small Cell Lung Cancer (NSCLC)
A Chart Review Study of Adults With Advanced NSCLC
The main aim is to see how treatment patterns and drugs might improve care for adults with advanced or metastatic NSCLC with epidermal growth factor receptor (EGFR) exon-20 driven mutations. Past medical records will be reviewed.
- Trial name
- EXTRACT (NCT05207423)
- Where
- 30 sites in Canada, France and 1 more
- Volunteers
- 179 people
- Sponsor
- Takeda
Metastatic Breast Cancer
Minimally Interventional Study on Prevalence of Emerging ESR1 Mutations in Liquid Biopsy in Three Cohorts of Patients With Breast Cancer in Comparison With Patient's Baseline ESR1 Mutation Status as Defined by Tissue Profiling.
Minimally interventional study on prevalence of emerging ESR1 mutations in liquid biopsy in three cohorts of patients with breast cancer (with and without prior therapies in metastatic setting, and during first-line aromatase…
- Trial name
- Pangeia-2 (NCT06417801)
- Where
- 6 sites in Brazil
- Volunteers
- 170 people
- Sponsor
- AstraZeneca
Amyotrophic Lateral Sclerosis
GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS)
GM604 is an endogenous human embryonic stage neural regulatory and signaling peptide that controls the development, monitoring and correction of the human nervous system.
- Trial name
- GALS (NCT01854294)
- Testing
- GM604
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Genervon Biopharmaceuticals, LLC
Advanced Solid Tumors With HER2 Mutation,eg:Colorectal,Urothelial,Gastric, Hepatobiliary,Endometrial,Melanoma,Ovarian,Cervical,Salivary Gland,Pancreatic,Breast
A Study of T-DXd for the Treatment of Solid Tumors Harboring HER2 Activating Mutations
This is an open-label, multi-center, single arm, Phase II study to evaluate the efficacy and safety of T-DXd for the treatment of unresectable and/or metastatic solid tumors harboring specific HER2 activating mutations regardless…
- Trial name
- DPT01 (NCT04639219)
- Testing
- Trastuzumab deruxtecan
- Where
- 30 sites in United States, Belgium and 7 more
- Volunteers
- 102 people
- Sponsor
- AstraZeneca
Cholangiocarcinoma, FGFR2 Fusion
Study to Evaluate the Efficacy and Safety of TT-00420 (Tinengotinib) in Cholangiocarcinoma
This study is an open-label, multicenter study to evaluate the efficacy and safety of TT-00420 tablet in adult patients with advanced cholangiocarcinoma.
- Conditions
- Cholangiocarcinoma, FGFR2 Fusion, FGFR2 Gene Mutation, FGFR1 Alteration
- Trial name
- NCT04919642
- Testing
- TT-00420
- Where
- 32 sites in United States
- Volunteers
- 55 people
- Sponsor
- TransThera Sciences (Nanjing), Inc.
Non-Small Cell Lung Cancer
A Study of Prevalence of EGFR Mutations in Participants With NSCLC
This is a non-interventional, local, multicenter, cross-sectional study to determine the prevalence of epidermal growth factor receptor (EGFR) mutations in participants diagnosed with non-squamous non-small cell lung cancer…
- Trial name
- NCT02695849
- Where
- 2 sites in Jordan
- Volunteers
- 69 people
- Sponsor
- Hoffmann-La Roche
Non-small Cell Lung Cancer (NSCLC)
Sacituzumab Tirumotecan (MK-2870) Versus Chemotherapy in Previously Treated Advanced or Metastatic Nonsquamous Non-small Cell Lung Cancer (NSCLC) With EGFR Mutations or Other Genomic Alterations (MK-2870-004)
The purpose of this study is to evaluate sacituzumab tirumotecan versus chemotherapy (docetaxel or pemetrexed) for the treatment of previously-treated non-small cell lung cancer (NSCLC) with exon 19del or exon 21 L858R EGFR…
- Trial name
- NCT06074588
- Testing
- Sacituzumab tirumotecan, Docetaxel, Pemetrexed
- Where
- 195 sites in United States, Australia and 23 more
- Volunteers
- 556 people
- Sponsor
- Merck Sharp & Dohme LLC
Advanced or Metastatic NSCLS With Exon 20 Insertion Mutation
REZILIENT3 (REsearching ZIpaLertinib In Egfr Non-small Cell Lung Cancer Tumors)
The purpose of this study is to evaluate the safety and efficacy of zipalertinib in combination with standard first-line platinum-based chemotherapy compared to chemotherapy alone, in patients with locally advanced or metastatic…
- Trial name
- REZILIENT3 (NCT05973773)
- Testing
- TAS6417
- Where
- 130 sites in United States, Belgium and 21 more
- Volunteers
- 285 people
- Sponsor
- Taiho Oncology, Inc.
Advanced Solid Tumor, PIK3CA Mutation
Combination of Serabelisib and Insulin Suppressing Diet With or Without Nab-paclitaxel in Subjects With Advanced Solid Tumors With PIK3CA Mutations
This study will evaluate the feasibility of optimizing the safety and tolerability of serabelisib (an investigational PI3K inhibitor) when combined with an ISD and with or without nab-paclitaxel with a goal of reducing side…
- Conditions
- Advanced Solid Tumor, PIK3CA Mutation, PTEN Loss of Function Mutation
- Trial name
- NCT05300048
- Testing
- Serabelisib, Insulin Suppressing Diet, Nab paclitaxel
- Where
- 17 sites in United States
- Volunteers
- 34 people
- Sponsor
- Faeth Therapeutics
Retinitis Pigmentosa
Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa.
- Trial name
- NCT06291935
- Testing
- VG901
- Where
- Germany
- Volunteers
- 6 people
- Sponsor
- VeonGen Therapeutics GmbH
Metastatic Colorectal Cancer, KRAS Gene Mutation
Onvansertib in Combination With FOLFIRI and Bevacizumab for Second Line Treatment of Metastatic Colorectal Cancer Patients With a KRAS Mutation
The purpose of the Phase 1b/2 study is to determine the safety and efficacy of Onvansertib, administered orally, daily on Day 1-5 and Day 15-19 of each 28-day cycle, in combination with FOLFIRI + Bevacizumab, as second-line…
- Trial name
- NCT03829410
- Testing
- Onvansertib, Bevacizumab, FOLFIRI
- Where
- 7 sites in United States
- Volunteers
- 68 people
- Sponsor
- Cardiff Oncology
Advanced or Metastatic Solid Tumors, FGFR Mutations
Efficacy and Safety of Pemigatinib in Participants With Solid Tumors With FGFR Mutations or Translocations (FIGHT-208)
The purpose of this study is to evaluate the efficacy and safety of pemigatinib in participants with previously treated locally advanced/metastatic or surgically unresectable solid tumors harboring activating FGFR mutations or…
- Conditions
- Advanced or Metastatic Solid Tumors, FGFR Mutations, FGFR Translocations
- Trial name
- NCT04003623
- Testing
- Pemigatinib
- Where
- 11 sites in United States
- Volunteers
- 1 people
- Sponsor
- Incyte Corporation
Carcinoma, Non-Small-Cell Lung
A Clinical Study of Bemcentinib With Standard of Care Chemoimmunotherapy in Untreated Advanced/Metastatic Non-small Cell Lung Cancer Patients With a Mutation in the STK11 Gene
The primary purpose of this study is to determine the safety and tolerability of the combination of bemcentinib with chemo-immunotherapy (CIT) to identify the recommended phase 2 dose (RP2D) when administered as first line (1L)…
- Trial name
- NCT05469178
- Testing
- Bemcentinib, Pembrolizumab, Pemetrexed
- Where
- 34 sites in United States, France and 5 more
- Volunteers
- 26 people
- Sponsor
- BerGenBio ASA
Neoplasms, Solid Tumors
My Pathway: A Study Evaluating Herceptin/Perjeta, Tarceva, Zelboraf/Cotellic, Erivedge, Alecensa, and Tecentriq Treatment Targeted Against Certain Molecular Alterations in Participants With Advanced Solid Tumors
This multicenter, non-randomized, open-label study will evaluate the efficacy and safety of six treatment regimens in participants with advanced solid tumors for whom therapies that will convey clinical benefit are not available…
- Conditions
- Neoplasms, Solid Tumors, Biliary Cancer, Salivary Cancer
- Trial name
- NCT02091141
- Testing
- Trastuzumab, Pertuzumab, Erlotinib
- Where
- 60 sites in United States
- Volunteers
- 673 people
- Sponsor
- Genentech, Inc.
EGFR-TKI-naïve Patients With NSCLC Harboring EGFR Activating Mutations
A Study of ASP8273 in Epidermal Growth Factor Receptor (EGFR) Tyrosine Kinase Inhibitor-Naïve Patients With Non-Small Cell Lung Cancer Harboring EGFR Mutations
The purpose of this study is to determine the safety, the antitumor activity and the pharmacokinetics of ASP8273 in EGFR tyrosine kinase inhibitor (EGFR-TKI)-naïve patients with non-small cell lung cancer (NSCLC) harboring EGFR…
- Trial name
- NCT02500927
- Testing
- ASP8273 Capsules, ASP8273 Capsules A
- Where
- 11 sites in Japan
- Volunteers
- 31 people
- Sponsor
- Astellas Pharma Inc