Exemestane for Polycystic Ovarian Syndrome

(ENDEAVOR Trial)

This trial tests exemestane, an aromatase inhibitor, to determine if it can help women with polyendocrine metabolic ovarian syndrome (PMOS, formerly known as PCOS) ovulate. It also monitors any side effects of the drug. Participants will take either exemestane or a placebo (an inactive substance) for a short period and attend follow-up clinic visits. Women diagnosed with PMOS, who have experienced difficulty conceiving, and have not previously tried ovulation induction drugs may be suitable for this trial. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants a chance to be among the first to receive this treatment.

Yes, you must stop taking certain medications that interact with exemestane, such as those listed in the exclusion criteria. If you are on any of these medications, you cannot participate in the trial.

Previous studies have generally found exemestane to be well-tolerated. This drug, often used to treat breast cancer, has side effects similar to others in its category. Common side effects include menopause-like symptoms, joint pain, and bone loss. Research involving over 1,000 patients found that only one death was possibly linked to exemestane treatment, indicating that serious side effects are rare.

Unlike the standard treatments for Polycystic Ovarian Syndrome (PCOS), which often include hormonal contraceptives and insulin-sensitizing drugs, Exemestane offers a unique approach by targeting estrogen production directly. Exemestane is an aromatase inhibitor, meaning it works by blocking the enzyme responsible for converting androgens into estrogen, potentially reducing symptoms more effectively. Researchers are particularly excited about its potential to offer quicker symptom relief, as it may show results in just a few days. Additionally, the study explores different dosages and durations, such as a 25 mg dose for 10 days and a 50 mg dose for 5 days, which could provide flexible treatment options for patients.

Research has shown that exemestane, a drug primarily used for breast cancer, lowers estrogen levels in the body. This is crucial because high estrogen can disrupt ovulation, often problematic in conditions like PMOS. Studies on similar treatments have found that reducing estrogen can lead to ovulation in about 65% of patients with these conditions. In this trial, participants will receive varying doses of exemestane or a placebo to assess its effectiveness for PMOS. Although direct data on exemestane for PMOS is limited, its mechanism suggests it might enhance the chances of ovulation. Early signs indicate promise for this potential new use.¹²³⁴⁶