(P 322) PHASE 2 SINGLE-ARM CLINICAL TRIAL TO EVALUATE THE EFFICACY AND SAFETY OF AURANOFIN IN ADULT PATIENTS WITH PROGRESSIVE DESMOID

Objective: Drugs with evidence for desmoid have the problem of side effects. There is a need for safe drugs that are useful for desmoids and have few side effects. The drug repositioning method showed that auranofin was effective in suppressing the growth of desmoid cells. Auranofin also suppressed desmoids formation in vivo. Auranofin is an FDA-approved disease-modifying anti-rheumatic drug that has been used for decades for treatment of rheumatoid arthritis with a well-studied safety profile for human use. In this presentation, we will introduce a phase 2 single-arm clinical trial to evaluate the efficacy and safety of auranofin in adults with progressive desmoid.

Methods: The inclusion criteria are (1) patients who have been confirmed to be desmoid by the time informed consent is obtained, (2) patients who are 18 years of age or older, (3) patients with evaluable lesions, and (4) within 6 months before the start of the study. Patients with tumor growth or exacerbation of symptoms in the patient, ⑤ Patients for whom informed consent has been obtained in writing by the patient or his / her surrogate. Exclusion criteria are (1) patients who took auranofin within 4 weeks before informed consent was obtained, (2) patients who took anticancer drugs or molecular-targeted therapies within 4 weeks before informed consent was obtained, (3) patients with serious complications, (4) patients who are judged to be inappropriate for conducting this clinical trial by the investigator. Scheduled number of cases: assuming that the threshold progression-free survival rate is 40%, the expected progression-free survival rate is 70%, the significance level is 10% on both sides, and the power is 80%, the required number of cases is 21 cases. Considering the cases of dropout, the planned number of cases is set to 23. Patients take auranofin 3 mg tablets twice daily after breakfast and dinner (same as the dosage and administration of rheumatoid arthritis). The primary endpoint is progression-free survival at 6 months after the start of treatment as efficacy. As safety, the event name, degree, and incidence of adverse events and side effects are determined. Secondary endpoints: The following items (1) to (3) at 6 months after the start of administration and the following items (1) to (4) at 52 weeks. (1) Response rate (image evaluation by MRI, RECIST): CR / PR / SD ratio, (2) Image evaluation by MRI T2-enhanced image, (3) Pain scale evaluation by NRS (Numerical Rating Scale), (4) Progression-free survival rate.

Results: We are currently recruiting patients with aggressive desmoid for clinical trials. No severe side effects such as discontinuation of auranofin administration have occurred.

Conclusion: Results of this phase 2 clinical trials for patients with progressive desmoid will be reported in the next CTOS.