Dihydroartemisinin for the treatment of polycystic ovary syndrome: study protocol for a multi-centre placebo-controlled randomised clinical trial.

introductionPolycystic ovary syndrome (PCOS) is the most common endocrine disorder that affects reproductive-aged individuals. Our previous single-arm pilot trial showed that dihydroartemisinin (DHA) normalised menstrual cycles, ameliorated hyperandrogenaemia and reduced antral follicles in individuals with PCOS. However, no comparative evidence confirms the efficacy of DHA in individuals with PCOS when compared with placebo. METHODS AND ANALYSIS: This is a multi-centre randomised, double-blinded, placebo-controlled trial of individuals with PCOS who have irregular menstrual cycles and hyperandrogenism, with or without polycystic ovary morphology. A total of 150 participants will be randomised in a 1:1 ratio to receive oral tablets of 40 mg DHA three times per day or placebo for 90 days. The primary outcome is the return of regular menstrual cycles within 6 months after treatment initiation, 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 randomisation. The results will be published in a peer-reviewed journal and will be presented at international meetings. TRIAL REGISTRATION NUMBER: NCT06842524.