Phase II Study of Defactinib (VS6063) in Patients With Tumors With NF2 Loss
Overview of the Study
The NCI-MATCH trial focused on patients with various cancers, assigning them targeted therapies based on genetic changes found in their tumors. This study specifically looked at tumors with neurofibromatosis 2 (NF2) mutations, which are sensitive to a treatment called defactinib, a FAK inhibitor.
Study Methods
Patients with NF2 mutations were given defactinib at a dose of 400 mg twice daily until their condition worsened or they experienced severe side effects. The main goal was to see how many patients responded positively to the treatment, while secondary goals included monitoring side effects and how long patients remained stable without disease progression.
Results Summary
Out of 5,548 patients screened, 57 had NF2 mutations. Only 35 enrolled in the study, and 33 received treatment. Most patients had already undergone multiple treatments before this study. Common side effects included:
- Fatigue: 36%
- Nausea: 33%
- High bilirubin levels: 27%
Only one patient showed a partial response, leading to an overall response rate of just 3%. The average time without disease progression was 1.9 months, with a few patients experiencing longer periods of stability.
Conclusion
This study did not achieve its main goal. Defactinib showed limited effectiveness in patients with NF2 loss who had already been treated multiple times.
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