Clinical trials: Mutation Abnormality
CML (Chronic Myelogenous Leukemia), Chronic Phase CML
ELVN-001 for the Treatment of Chronic Myeloid Leukemia With and Without T315I Mutation in Japanese Participants
The purpose of this study is to evaluate the safety, tolerability and determine the recommended dose for further clinical evaluation of ELVN-001 in Japanese patients with chronic phase chronic myeloid leukemia with and without…
- Trial name
- CML (NCT06787144)
- Testing
- ELVN-001
- Where
- 4 sites in Japan
- Volunteers
- 21 people
- Sponsor
- Enliven Therapeutics
Dementia, Frontotemporal
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 in Healthy Participants
This is a Phase 1, randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of VES001 in a two part followed by a…
- Trial name
- SORT-IN-1 (NCT06226064)
- Testing
- VES001
- Where
- Netherlands
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- Vesper Biotechnologies ApS
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
Non-Small Cell Lung Cancer, Epidermal Growth Factor Receptor
Bolstering Outcomes After Induction With Osimertinib Plus Chemotherapy Through Optimized Site-Directed Primary Tumor Therapy (BOOST Trial)
This is a single-arm, open-label, phase II study evaluating the clinical outcomes of local therapy (surgery or radiotherapy) to the primary tumor in patients with EGFR-mutant advanced non-small cell lung cancer (NSCLC) who have…
- Trial name
- BOOST (NCT07073365)
- Testing
- Local Therapy (Surgery or Radiotherapy)
- Where
- South Korea
- Volunteers
- 70 people
- Sponsor
- Pusan National University Hospital
Solid Tumors Harboring a TP53 Y220C Mutation
A Phase I First-in-Human Study of GenSci128 in Patients With Solid Tumors Harboring a TP53 Y220C Mutation
The purpose of this study is to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of GenSci128 Tablet in Patients with Locally Advanced or Metastatic Solid Tumors Harboring a TP53 Y220C Mutation
- Trial name
- NCT06908434
- Testing
- GenSci128 tablets
- Where
- China
- Volunteers
- 82 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
PIK3CA Mutation-Related Tumors
A Study of WX390 in Patients With Advanced Solid Tumors With PIK3CA Mutations
The goal of this clinical trial is to evaluate the safety and preliminary efficacy of WX390 in patients with advanced solid tumors. The main question it aims to answer is: safety and preliminary efficacy in WX390 therapy.
- Trial name
- NCT06132932
- Testing
- WX390
- Where
- China
- Volunteers
- 38 people
- Sponsor
- Shanghai Jiatan Pharmatech Co., Ltd
Cystic Fibrosis
A Phase 3 Study of VX-445 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-445 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
- NCT03525444
- Testing
- VX-445/TEZ/IVA, IVA
- Where
- 115 sites in United States, Australia and 11 more
- Volunteers
- 405 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Gastric Cancer, Gastric Neoplasms
Phase II Randomized Trial of Bethesda Protocol Compared to Cambridge Method for Detection of Early Stage Gastric Cancer in CDH1 Mutation Carriers
Background: Some people have a mutation in the cadherin-1 gene (CDH1) gene that is known to lead to stomach cancer. They are advised to get regular endoscopies with biopsies even if their stomach appears normal.
- Conditions
- Gastric Cancer, Gastric Neoplasms, Gastric Adenocarcinoma
- Trial name
- NCT04535414
- Testing
- Cellvizio (Registered trademark) Real-Time In Vivo Cellular Imaging Platform with Confocal Miniprobes, Olympus Graphics Interchange Format (GIF) 190 endoscope, Gastric mucosal biopsy
- Where
- United States
- Volunteers
- 195 people
- Sponsor
- National Cancer Institute (NCI)
Acute Myeloid Leukemia, Mixed Lineage Leukemia Gene Mutation
A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML
Ziftomenib is an investigational drug in development for the treatment of patients with acute myeloid leukemia (AML) with certain genetic alterations.
- Conditions
- Acute Myeloid Leukemia, Mixed Lineage Leukemia Gene Mutation, Refractory AML, AML With Mutated NPM1
- Trial name
- NCT05735184
- Testing
- Ziftomenib, Venetoclax, Azacitidine
- Where
- 44 sites in United States
- Volunteers
- 420 people
- Sponsor
- Kura Oncology, Inc.
Women With BRCA 1 or BRCA 2 Mutation, Non-tested Female Family Members
Lessons Learned From the Family Gene Toolkit
Mutations in the BRCA1/2 genes are the primary cause of hereditary breast/ovarian cancer syndrome. Genetic testing identifies mutation carriers and enables them to manage their cancer risk (i.e.
- Trial name
- FGT (NCT02154633)
- Testing
- Family Gene Toolkit, Delayed Family Gene Toolkit
- Where
- United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Cholangiocarcinoma
Combination Chemotherapy Plus Panitumumab or Bevacizumab for Inoperable Cholangiocarcinoma Without KRAS Mutations
The purpose of this study is to determine the rate of progression free survival of patients with inoperable cholangiocarcinoma 6 months after enrollment in the study.
- Trial name
- GOC-B-P (NCT01206049)
- Testing
- Gemcitabine, Oxaliplatin, Capecitabine
- Where
- Denmark
- Volunteers
- 88 people
- Sponsor
- Vejle Hospital
Solid Tumor, Lymphoma
Precision Medicine for Patients With Identified Actionable Mutations
The goal of the current pragmatic trial is to evaluate the impact of a simple method of selecting a treatment approach for identified mutations on participants' progression free survival (PFS).
- Conditions
- Solid Tumor, Lymphoma, Multiple Myeloma, Malignant Neoplasm
- Trial name
- NCT04111107
- Testing
- Investigational Agent, Supportive Care Regimens, Next Gen Sequencing Report
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Wake Forest University Health Sciences
Non-small-cell Lung Cancer
Neoadjuvant Therapy of Icotinib in Epidermal Growth Factor Receptor Mutated NSCLC Patients
The purpose of this study is to evaluate the overall response rate and disease free survival for IIIAN2 non-small-cell lung cancer patients with EGFR 19 or 21 exon mutation.
- Trial name
- NCT01843647
- Testing
- Icotinib, Chemotherapy
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Chronic Hepatitis B
Molecular Characteristics of Hepatitis B Virus Integration, Mutation, and Drug Resistance
Patients with a confirmed diagnosis of chronic hepatitis B (CHB) who have complete clinical data and retained blood or liver tissue samples will be enrolled.
- Trial name
- HBV IMDR (NCT06779864)
- Where
- China
- Volunteers
- 2,000 people
- Sponsor
- Beijing Friendship Hospital
Acute Myeloid Leukemia
Gilteritinib vs Midostaurin in FLT3 Mutated Acute Myeloid Leukemia
Eligible untreated patients with FLT3 acute myeloid leukemia (AML) between the ages of 18 and 70 will be randomized to receive gilteritinib or midostaurin during induction and consolidation.
- Trial name
- NCT03836209
- Testing
- Gilteritinib, Midostaurin, Daunorubicin
- Where
- 44 sites in United States
- Volunteers
- 181 people
- Sponsor
- PrECOG, LLC.
HRAS Gene Mutation, HNSCC
Safety and Efficacy of Tipifarnib in Head and Neck Cancer With HRAS Mutations and Impact of HRAS on Response to Therapy
An international, multicenter, open-label, 2 cohort, non-comparative, pivotal study evaluating the efficacy of tipifarnib in HRAS mutant HNSCC (AIM-HN).
- Trial name
- AIM-HN/SEQ-HN (NCT03719690)
- Testing
- Tipifarnib, HRAS Detection Assay
- Where
- 102 sites in United States, Australia and 14 more
- Volunteers
- 296 people
- Sponsor
- Kura Oncology, Inc.
Leber's Hereditary Optic Neuropathy (LHON)
Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy
The objective of this clinical study is to select the optimal dose and evaluate the safety and efficacy of NR082 in treatment of LHON caused by mitochondrial ND4 gene mutation.
- Trial name
- GOLD (NCT04912843)
- Testing
- NR082 injection
- Where
- China
- Volunteers
- 102 people
- Sponsor
- Wuhan Neurophth Biotechnology Limited Company
Non-Small Cell Lung Cancer
First-line Treatment With Icotinib in Elder NSCLC EGFR Mutated Patients
A single arm, open label, phase IV trial to evaluate safety and efficacy of icotinib as first-line treatment for the elder patients with advanced or metastatic (IIIb and IV) NSCLC and mutated EGFR.
- Trial name
- NCT01646450
- Testing
- Icotinib
- Where
- China
- Volunteers
- 35 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Retinoblastoma, Melanoma
Retinoblastoma Biomarker Study
Retinoblastoma is a rare pediatric ocular tumor caused by germline and/or somatic mutations in the tumor suppressor gene RB1. Survivors of retinoblastoma, particularly those with the hereditary form of the disease (germline RB1…
- Conditions
- Retinoblastoma, Melanoma, Sarcoma
- Trial name
- NCT00342797
- Where
- 4 sites in United States
- Volunteers
- 2,136 people
- Sponsor
- National Cancer Institute (NCI)
NSCLC Stage IV
Cranial Radiotherapy Plus Chemoimmunotherapy in Untreated Driver-mutation Negative NSCLC With Active Brain Metastasis
Non-small cell lung cancer (NSCLC), the most prevalent form of lung cancer, has a significant risk of brain metastasis (BM). Historically, the median overall survival for advanced NSCLC patients with BM was under six months with…
- Trial name
- NCT06501378
- Testing
- PD-1/PD-L1 inhibitor plus chemotherapy, SRT/WBRT
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Fudan University
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