Dihydroartemisinin for the Treatment of Polycystic Ovary Syndrome

NCT06842524 | Recruiting Phase 2 DMC

• 1 other identifier: B2024-417(2)
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 4

Brief Summary

Polycystic ovarian syndrome (PCOS) is the most frequent endocrine disorder affecting women of reproductive age, with a prevalence of 10 to 13%. PCOS is characterized by irregular menstrual cylcles/ovulatory dysfunction, hyperandrogenism, and polycystic ovarian morphology. For infertile patients seeking ovulation induction, letrozole is the drug of first choice. For PCOS patients not seeking pregnancy, there exists a variety of treatments to alleviate symptoms. It has been demonstrated that artemisinin derivatives can promote energy expenditures and insulin sensitivity by activating thermogenic adipocytes, thereby protecting against diet-induced obesity and metabolic disorders in rodents. Recently, we showed in a single arm pilot study including 19 PCOS-patients, that dihydroartemisinin ameliorated hyperandrogenemia reduced antral follicle count and normalized menstrual cycles. Based on these findings, we aim to evaluate the efficacy of dihydroartemisinin in women with PCOS in a placebo controlled randomized clinical trial. 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 results will potentially impact the standard of care for patients diagnosed with PCOS.

Conditions

Polycystic Ovary Syndrome (PCOS)

Keywords

Polycystic Ovary Syndrome; Dihydroartemisinin

Outcome Measures

Primary Outcomes (1)

• The occurrence of a regular menstrual cycle

  The occurrence of a regular menstrual cycle is defined as at least three consecutive spontaneous vaginal bleedings lasting for 2-7 days, with intervals between the start of each cycle of 21 and 35 days (inclusive), during the 26-week period after initiating treatment.

  From the start of treatment to the end of follow-up at 26 weeks

Secondary Outcomes (7)

• The presence of a dominant follicle

  From the start of treatment to the end of follow-up at 26 weeks

• The number of bilateral antral follicles

  Before and immediately after 90-day treatment

• Serum AMH

  Before and immediately after 90-day treatment

• Serum total testosterone

  Before and immediately after 90-day treatment

• Serum sex hormone binding globulin (SHBG)

  Before and immediately after 90-day treatment

Study Arms (2)

Dihydroartemisinin Arm

ACTIVE COMPARATOR

Dihydroartemisinin tablets 40mg tid for 90 days

Drug: Dihydroartemisinin

Placebo Arm

PLACEBO COMPARATOR

Idential placebo tid for 90 days

Drug: Placebo

Interventions

Dihydroartemisinin DRUG

Dihydroartemisinin tablets 40mg tid po for 90 days

Dihydroartemisinin Arm

Placebo DRUG

Identical placebo tid for 90 days

Placebo Arm

Eligibility Criteria

• Age: 18 Years - 40 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Women with PCOS defined as having irregular menstrual cycles and hyperandrogenism. Irregular menstrual cycles are defined as \< 21 or \> 35 days or \< 8 cycles per year. Hyperandrogenism refers to either hyperandrogenemia or hirsutism. Hyperandrogenemia will be defined as an elevated total testosterone \>1.67 nmol/L measured by Elecsys Testosterone II (Roche Diagnostics). Hirsutism is determined by a modified Ferriman-Gallwey Score \>4 at screening exam.
• Body Mass Index (BMI) between 18.5 and 28 kg/M2
• Negative pregnancy test
• No plan for pregnancy in the coming 6 months

You may not qualify if:

• Patients on oral contraceptives. A two-month washout period will be required prior to screening for patients on these agents. A one-month washout will be required for patients on oral cyclic progestins. Patients on depo-progestins or hormonal implants are excluded.
• Patients with liver disease defined as ALT or AST above normal range of each participating center, or total bilirubin\>30umol/L. Metabolic dysfunction-associated steatotic liver disease (MASLD) with normal ALT and AST can be included.
• Patients with anemia (Hemoglobin \< 12 g/dL) or neutropenia (neutrocyte \<1.8×10\^9/L).
• Patients with renal disease defined as serum creatinine\> 115umol/L.
• Patients diagnosed with other endocrine diseases that are known to cause secondary polycystic ovary morphology, e.g., Cushing's syndrome, hyperprolactinemia, congenital adrenal hyperplasia (21-hydroxylase deficiency or other enzyme deficiency), hypothyroidism, etc.
• Patients diagnosed with Type 1 or Type 2 diabetes.
• Patients with known heart disease, like heart failure, atrial fibrillation, coronary heart disease, etc.
• Patients with a history of any type of cancer.
• Patients taking other medications known to affect reproductive function or metabolism. These medications include GnRH agonists and antagonists, antiandrogens, gonadotropins, GLP-1 receptor agonist, SGLT2i, metformin and thiazolidinediones. The washout period on all these medications will be two months.
• Patients who have undergone a bariatric surgery procedure within the past 12 months.

Sponsors & Collaborators

• Shanghai Zhongshan Hospital: lead

Study Sites (4)

The First Affiliated Hospital of Xiamen University

Xiamen, Fujian, 361003, China

RECRUITING

Women and Children's Hospital, School of Medicine, Xiamen University

Xiamen, Fujian, 361003, China

RECRUITING

Zhongshan Hospital Fudan University

Shanghai, Shanghai Municipality, 200032, China

RECRUITING

The First Affiliated Hospital of Wenzhou Medical University

Wenzhou, Zhejiang, 325000, China

RECRUITING

MeSH Terms

Conditions

Polycystic Ovary Syndrome

Interventions

artenimol

Condition Hierarchy (Ancestors)

Ovarian Cysts; Cysts; Neoplasms; Ovarian Diseases; Adnexal Diseases; Genital Diseases, Female; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Genital Diseases; Gonadal Disorders; Endocrine System Diseases

Study Officials

• Xi Dong, MD

  Reproductive Medicine Center, Zhongshan Hospital, Fudan University

  STUDY CHAIR

• Xiaoying Li, MD, PhD

  Department of Endocrinology and Metabolism, Zhongshan Hospital, Fudan University

  STUDY DIRECTOR

• Jingjing Jiang, MD, PhD

  Department of Endocrinology and Metabolism, Zhongshan Hospital, Fudan University

  PRINCIPAL INVESTIGATOR

• Ben Willem Mol, MD, PhD

  Department of Obstetrics and Gynaecology, Monash University

  PRINCIPAL INVESTIGATOR

• Wentao Li, MD, PhD

  National Perinatal Epidemiology and Statistics Unit, Centre for Big Data Research in Health, University of New South Wales

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Liangshan Mu, MD, PhD

CONTACT

86-021-64041990; mu.liangshan@zs-hospital.sh.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 19, 2025
• First Posted: February 24, 2025
• Study Start: April 16, 2025
• Primary Completion (Estimated): September 30, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: April 20, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ANALYTIC CODE
• Time Frame: After publication.
• Access Criteria: IPD and supporting information will be avaible to researchers upon reasonable request (e.g. with a practical and meaningful research proposal).

Locations

CN (4)