A Phase II, randomised, multi–centric, multi-national clinical trial to evaluate the efficacy, tolerability and safety of a fixed dose combination of Spironolactone/Pioglitazone/Metformin (SPIOMET) for adolescent girls and young adult women (AYAs) with polycystic ovary syndrome (PCOS)

UFUK 2020 Projesi, 2021 - 2026

Proje Türü: UFUK 2020 Projesi
Başlama Tarihi: Nisan 2021
Bitiş Tarihi: Nisan 2026

Proje Özeti

Polycystic Ovary Syndrome (PCOS) is the most prevalent, chronic endocrine-metabolic disorder in women of reproductive age, affecting 5-10% of women worldwide. It is the most frequent cause of anovulatory subfertility. There is no approved therapy for PCOS. Usual care with oral contraceptives -prescribed off-label- does not revert the underlying pathophysiology or associated co-morbidities. Pilot studies have generated new insights into the pathophysiology of PCOS, leading to the identification of a new treatment able to revert the PCOS phenotype without side effects. The novel medication consists of a fixed, low-dose combination of two insulin sensitisers [Pioglitazone (Pio) and Metformin (Met)] and one mixed anti-androgen and anti-mineralcorticoid (Spironolactone (Spi)] within a single tablet - SPIOMET. SPIOMET4HEALTH will test, in a multicentre Phase II trial, the effects of SPIOMET components on top of lifestyle measures in adolescent girls and young adult women with PCOS. SPIOMET aims at normalizing ovulation rate and the endocrinemetabolic status through reduction of hepato-visceral fat excess in an early phase of the disorder. This approach will presumably reduce the risks of co-morbidities, including anovulatory subfertility. These accomplishments should improve the quality of life in these patients and significantly mitigate the economic burden for the European healthcare system. The consortium clusters experts from all European research groups working on PCOS in AYAs. SPIOMET4Health will engage patients from the very beginning and throughout the project and incorporate their experiences in the design and evaluation of the trial. The update and validation of PCOS-specific Patient Reported Outcome Measures (PROMs) will provide the first large-scale evidence on the psycho-social benefits of the studied treatment. These results, together with economic modelling, will lead to conclusions that will be helpful for sound decision-making in healthcare systems.