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. 2024 Oct 7;16(10):e71033.
doi: 10.7759/cureus.71033. eCollection 2024 Oct.

Efficacy and Safety of Auranofin for Progressive Desmoid-Type Fibromatosis: The Study Protocol of an Open-Label Phase II Trial

Yoshihiro Nishida  1 , Kan Ito  2 , Tomohisa Sakai  3 , Fumie Kinoshita  4 , Yachiyo Kuwatsuka  4 , Saori Kinoshita  5 , Shiro Imagama  3
Affiliations

Efficacy and Safety of Auranofin for Progressive Desmoid-Type Fibromatosis: The Study Protocol of an Open-Label Phase II Trial

Yoshihiro Nishida et al. Cureus. .

Abstract

Background As desmoid-type fibromatosis (DF) exhibits a high recurrence rate after surgery, initial active surveillance followed by medical therapy is the mainstay of the treatment. However, there are few effective drugs with acceptable side effects. Methodology Among drugs that have been used for a long period and possess a known safety profile, auranofin was observed to be effective in suppressing DF using the drug repositioning method in our laboratory. This clinical study has been designed to examine the efficacy and safety of auranofin, an approved anti-rheumatic drug, in patients with progressive DF. Results This study is conducted as a single-center, single-arm, open-label study. Auranofin 3 mg tablets will be administered twice daily to DF patients with progressive disease. The primary endpoint is progression-free survival at 26 weeks after starting treatment. Secondary endpoints include response rate, T2-weighted MRI evaluation, pain intensity, quality of life (QOL), and safety assessment. Conclusions This is the first clinical trial of auranofin in patients with aggressive DF. The study will allow an in-depth understanding of the efficacy of auranofin for response rate as well as for changes in MRI findings, pain, and QOL in patients with aggressive DF.

Keywords: auranofin; clinical trial; desmoid; drug repositioning; protocol.

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Conflict of interest statement

Human subjects: All authors have confirmed that this study did not involve human participants or tissue. Animal subjects: All authors have confirmed that this study did not involve animal subjects or tissue. Conflicts of interest: In compliance with the ICMJE uniform disclosure form, all authors declare the following: Payment/services info: All authors have declared that no financial support was received from any organization for the submitted work. Financial relationships: Yoshihiro Nishida declare(s) a grant from Nagoya University Hospital. This research is supported by Nagoya University Hospital Funding for Clinical Development. Other relationships: All authors have declared that there are no other relationships or activities that could appear to have influenced the submitted work.

Figures

Figure 1
Figure 1. Flowchart of the study procedure.
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