Clinical trials: Genetic Mutation
Ovarian Cancer
A Study of Fluzoparib（SHR-3162）in BRCA1/2-mutant Relapsed Ovarian Cancer
This is a multicenter， open-label study to evluate the efficacy and safety of a novel PARP 1/2 inhibitor fluzoparib （SHR-3162）in BRCA1/2-mutant Relapsed Ovarian Cancer.
- Trial name
- NCT03509636
- Testing
- Fluzoparib
- Where
- China
- Volunteers
- 113 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Cholangiocarcinoma With IDH1 Mutation, Solid Tumors With IDH1 Mutation
IDH1 Inhibitor AB-218 in Patients With Advanced IDH1 Mutant Cholangiocarcinoma and Other Solid Tumor
This is an open label, single-arm Phase I study to evaluate the safety, tolerability, PK and preliminary efficacy of AB-218, an oral IDH1 inhibitor, for the treatment of adult patients with advanced IDH1 mutant cholangiocarcinoma…
- Trial name
- NCT05814536
- Testing
- AB-218 capsule
- Where
- 14 sites in China
- Volunteers
- 9 people
- Sponsor
- AnHeart Therapeutics Inc.
Cystic Fibrosis
A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
The primary purpose of study is to evaluate the treatment effect of tezacaftor in combination with ivacaftor (TEZ/IVA) on chest imaging endpoints using low-dose computed tomography (LDCT) at Week 72, and to evaluate the safety of…
- Trial name
- NCT02730208
- Testing
- Tezacaftor/Ivacaftor, Ivacaftor
- Where
- 9 sites in Australia
- Volunteers
- 41 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Non Small Cell Lung Cancer, Brain Metastases
A Study to Determine the Efficiency For Brain Metastasis NSCLC Patients Treated With Icotinib Alone or Combined With Radiation Therapy
The purpose of this study is to evaluate the efficacy of icotinib alone or in combination with radiation therapy for NSCLC patients harboring EGFR mutation with brain metastases. The primary endpoint is overall survival .
- Trial name
- SMART (NCT04058704)
- Testing
- Icotinib, SRS/WBRT/HA-WBRT/SMART
- Where
- China
- Volunteers
- 296 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Non Small Cell Lung Cancer
A Study to Assess the Efficacy of WSD0922-FU in Patients With C797S+ Advanced Non-small Cell Lung Cancer
This is a Phase II, Open Label, Multicenter, Single Arm Study of WSD0922-FU for Patients with Locally Advanced or Metastatic Non-Small Cell Lung Cancer whose Disease has Progressed with First-Line Osimertinib Treatment and whose…
- Trial name
- NCT06868485
- Testing
- WSD0922-FU Tablets, Dose level A, WSD0922-FU Tablets, Dose level B
- Where
- 18 sites in United States, China and 1 more
- Volunteers
- 40 people
- Sponsor
- Wayshine Biopharm, Inc.
Non-Small Cell Lung Cancer
Pivotal Study to Evaluate YL202 Versus Docetaxel in Patients With Locally Advanced or Metastatic EGFR Sensitive Mutation Non-Squamous Non-Small Cell Lung Cancer
Non-small cell lung cancer (NSCLC) is a common type of lung cancer where abnormal cells in the lungs grow out of control. The purpose of this study is to evaluate YL202 monotherapy versus Docetaxel in participants with locally…
- Trial name
- NCT07416994
- Testing
- YL202, Docetaxel
- Where
- 2 sites in China
- Volunteers
- 440 people
- Sponsor
- MediLink Therapeutics (Suzhou) Co., Ltd.
Clonal Mast Cell Disease, KIT D816V Mutation
Screening Study for KIT D816V Mutated Mast Cell Disease in Select Populations
This is a multicenter screening study to characterize the prevalence of the KIT D816V mutation in participants with suspected clonal mast cell disease.
- Conditions
- Clonal Mast Cell Disease, KIT D816V Mutation, Suspected KITD816V Mutated Clonal Mast Cell Disease
- Trial name
- POLARIS (NCT07143669)
- Testing
- Screening
- Where
- 26 sites in United States, Belgium
- Volunteers
- 750 people
- Sponsor
- Blueprint Medicines Corporation
Advanced Breast Cancer
Study to Assess the Safety of Alpelisib Plus Fulvestrant, in Men and Post-menopausal Women With HR-positive, HER2-negative, Advanced Breast Cancer (aBC) With PIK3CA Mutation, Whose Disease Progressed on or After Endocrine Treatment
The purpose of this study was to determine the safety of alpelisib plus fulvestrant in men and post-menopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, advanced or…
- Trial name
- ALPINIST (NCT05631795)
- Testing
- Alpelisib, Fulvestrant
- Where
- 12 sites in India
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Non-small Cell Lung Cancer
Study to Evaluate Sutetinib Maleate Capsule in Locally Advanced or Metastatic Non-small Cell Lung Cancer
This is a Phase 2b, multicenter, open-label study to evaluate the safety and efficacy of Sutetinib Maleate Capsule in Locally Advanced or Metastatic NSCLC (Non-resistant Uncommon EGFR Mutations Only, Including L861Q, G719X…
- Trial name
- NCT05168566
- Testing
- Sutetinib Maleate Capsule
- Where
- 31 sites in United States, Australia and 1 more
- Volunteers
- 128 people
- Sponsor
- Teligene US
Malignant Melanoma, Neoplasms
A Pharmacokinetics Study to Investigate the Effect of Ketoconazole on Vemurafenib in Patients With BRAFV600 Mutation-Positive Metastatic Melanoma
This open-label, multi-center, three-period, one sequence study will investigate the effect of ketoconazole on the pharmacokinetics of vemurafenib in patients with unresectable BRAFV600-mutation positive metastatic melanoma or…
- Trial name
- NCT01765556
- Testing
- ketoconazole, vemurafenib
- Sponsor
- Hoffmann-La Roche
Non-small Cell Lung Cancer
Efficacy and Safety of Osimertinib for HK Chinese With Metastatic T790M Mutated NSCLC-real World Setting.
To assess the efficacy of single-agent osimertinib in relation to EGFR T790M mutant allele fraction (AF) in a real-world setting.
- Trial name
- NCT03219970
- Testing
- Osimertinib
- Where
- 4 sites in China
- Volunteers
- 156 people
- Sponsor
- AstraZeneca
Cystic Fibrosis
Study of Ataluren (PTC124) in Cystic Fibrosis
The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study…
- Trial name
- NCT02107859
- Testing
- Ataluren
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 61 people
- Sponsor
- PTC Therapeutics
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
Low-grade Glioma
Expanded Access Program (EAP) for Tovorafenib (DAY101) in RAF-Altered, Relapsed or Refractory Low-Grade Glioma
The DAY101-EAP is a multicenter, open-label, expanded access treatment protocol designed to provide access to tovorafenib (DAY101) for eligible patients.
- Trial name
- NCT05760586
- Testing
- Tovorafenib
- Sponsor
- Day One Biopharmaceuticals, Inc.
Non-small Cell Lung Cancer, EGFR Gene Mutation
First Line Treatment in EGFR Mutation Positive Advanced NSCLC Patients With Central Nervous System (CNS) Metastases
The first-line treatment with single agent AZD3759 results in superior Progression Free Survival (PFS) compared to Standard of Care (SoC) Epidermal Growth Factor Receptor Tyrosine Kinase Inhibitors (EGFR-TKI), in patients with…
- Conditions
- Non-small Cell Lung Cancer, EGFR Gene Mutation, Brain Metastases
- Trial name
- BM (NCT03653546)
- Testing
- AZD3759, Erlotinib, Gefitinib
- Where
- 58 sites in China, Singapore and 2 more
- Volunteers
- 492 people
- Sponsor
- Alpha Biopharma (Jiangsu) Co., Ltd.
NSCLC, Stage IV, NSCLC Stage IIIB
Study of Tarloxotinib in Pts With NSCLC (EGFR Exon 20 Insertion, HER2-activating Mutations) & Other Solid Tumors With NRG1/ERBB Gene Fusions
Open-label, Phase 2, single treatment arm, 3 cohorts
- Conditions
- NSCLC, Stage IV, NSCLC Stage IIIB, NSCLC, Stage IIIC, NSCLC, Recurrent
- Trial name
- RAIN (NCT03805841)
- Testing
- tarloxotinib bromide
- Where
- 14 sites in United States, Canada and 1 more
- Volunteers
- 41 people
- Sponsor
- Rain Oncology Inc
Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant
Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation
The purpose of this study is to characterize the disease progression of confirmed OPA1 mutation-associated autosomal dominant optic atrophy (ADOA) by evaluating the changes in ocular structural and functional outcomes.
- Conditions
- Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant, Optic Atrophies, Hereditary, Kjer Optic Atrophy
- Trial name
- NCT06140329
- Where
- 7 sites in United States, Australia and 4 more
- Volunteers
- 1 people
- Sponsor
- PYC Therapeutics
Non-Small Cell Lung Cancer, KRAS G12C Lung Cancer
A Study Evaluating the Efficacy and Safety of Divarasib Versus Sotorasib or Adagrasib in Participants With Previously Treated KRAS G12C-positive Advanced or Metastatic Non-Small Cell Lung Cancer
The purpose of this study is to assess the safety and efficacy of divarasib compared to locally approved KRAS G12C inhibitors (sotorasib or adagrasib) in participants with KRAS G12C-positive (KRAS G12C +) advanced or metastatic…
- Trial name
- Krascendo 1 (NCT06497556)
- Testing
- Divarasib, Sotorasib, Adagrasib
- Where
- 151 sites in United States, Argentina and 24 more
- Volunteers
- 338 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
DMD/BMD is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Trial name
- NCT00592553
- Testing
- Ataluren
- Where
- 37 sites in United States, Australia and 9 more
- Volunteers
- 174 people
- Sponsor
- PTC Therapeutics
Acute Myeloid Leukemia (AML)
Treatment Patterns and Key Healthcare Resource Use in Acute Myeloid Leukemia (AML) With or Without FMS-like Tyrosine Kinase-3 (FLT3) Mutation Study Based on Retrospective Chart Review
The purpose of this study is to retrospectively evaluate the treatment patterns and AML-related key healthcare resource use among AML patients, stratified by FLT3 mutation status, intensive chemotherapy (IC) eligibility, and…
- Trial name
- NCT03047083
- Testing
- Treatment patterns among AML patients, AML-related healthcare resources
- Where
- United States
- Volunteers
- 1,027 people
- Sponsor
- Astellas Pharma Global Development, Inc.
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