Dihydroartemisinin for Treatment of Polycystic Ovary Syndrome: study protocol for a multi-centre placebo-controlled Randomized Clinical Trial

Introduction

Polycystic ovary syndrome (PCOS) is the most common endocrine disorder that affects reproductive-aged individuals. Our previous single-arm pilot trial showed that dihydroartemisinin (DHA) normalized menstrual cycles, ameliorated hyperandrogenemia, and reduced antral follicles in individuals with PCOS. However, there is still lack of comparative evidence that confirms the efficacy of DHA in individuals with PCOS when compared to placebo.

Methods and analysis

This is a multicentre randomized, double-blinded, placebo-controlled trial in individuals with PCOS who have irregular menstrual cycles and hyperandrogenism, with or without polycystic ovary morphology. A total of 150 participants will be randomized in a 1:1 ratio to receive oral tablets of 40mg DHA three times per day or placebo for 90 days. The primary outcome is return of regular menstrual cycles within 6 months after start of treatment, with antral follicle count and metabolic profile being secondary outcomes. The primary analysis will follow the intention-to-treat principle.

Ethics and dissemination

This study has been approved by the ethics committees of Zhongshan Hospital, Fudan University and all participating centres. All participants will provide written informed consent before randomization. The results will be published in a peer-reviewed journal and will be presented at international meetings.

Trial registration number

NCT06842524

Protocol version

1.1 (2025/07/18)

Strengths and limitations of this study

This is a multicentre, randomised, double-blinded, placebo-controlled trial.

This study is the first randomized trial to explore the efficacy of DHA in PCOS.

The results may be underpowered to study a smaller than expected effect of DHA.