Work
Packages

WP1 will manage the Phase II clinical trial performed in 364 patients distributed into four subgroups (cohorts) using a 1:1:1:1 allocation ratio for Placebo:PIO:SPI:SPIOMET.

This clinical trial aims to assess:

– The efficacy, tolerability and safety of a new treatment (SPIOMET) for adolescent girls and young women with PCOS;

– The superiority (in this order) of each SPIOMET, SPIO and PIO over placebo and in addition, the superiority of SPIOMET against the other study arms.

All patients will receive lifestyle guidance (WP2). The primary endpoint is ovulation rate (over 3 x 3 months). Bioequivalence of SPIOMET in a single tablet vs the co-intake of the three separate compounds has been established in a Phase I study. The randomized (1:1:1:1) clinical trial will be stratified by centre, body mass index and gynaecological age.

The proposed Clinical Investigation Protocol is designed according to the European Medicines Agency’s (EMA) response to the Paediatric Investigation Plan (PIP).

WP2 will implement a specific Lifestyle Intervention Program (LIP) that will be developed for both adolescent girls (>12.0 and <16.0 years), and young women (>16.0 and <24 years) and will be implemented during the time window of medication intake. This intervention will be mostly based on the well-established lifestyle intervention Obeldicks, which is effective to treat hyperandrogenaemia and other symptoms related to PCOS in patients with obesity.

Optimapharm will be responsible for the management of the clinical trial, including activities like:

1- Finalise the trial protocol and submission package
2- Trial coordination and central trial management
3- Monitor data quality and safety
4- Pharmacovigilance
5-Data management
6-Biostatistics

Optimapharm will closely collaborate with the partners FSJD (coordination), RJF (manufacturing and supply of the study medication) and Asphalion (regulatory strategy and exploitation).

WP4 will analyse the costs and health effects of each treatment option as Health-Related Quality of Life (HRQoL). During the Phase II clinical trial HRQoL will be assessed using validated instruments: a generic short form 36 health questionnaire (SF-36) and a specific PCOS questionnaire (PCOSQ).

The PCOSQ is an instrument that was specifically designed and validated to evaluate the impact of the symptoms and signs of PCOS on patients HRQoL.

The versions of the questionnaires in the respective languages of the participating countries will also be validated during the clinical trial.

Patients will be engaged in the revision of the clinical protocol, the material for patients (assent document, patients’ information sheet, lay summary, patient diary, etc.) and in the design of the Lifestyle Intervention Program (LIP). This will also ensure that the information about the trial and its communication is suitable for the health literacy level of the target population. This will be critical to guarantee the highest rate of inclusions, the lowest rate of withdrawals (WP1-3) and the highest commitment and adherence to the new healthcare intervention.

This WP will ensure that appropriate communication and dissemination channels are used to raise awareness around SPIOMET4Health’s activities and results in various European Union communities: policy makers, clinicians, patients, scientists and the general public. This work will contribute to enable the understanding of PCOS, as well as its social, economic and personal impact on health. The communication and dissemination strategy will provide information on PCOS in easy-to- understand and friendly formats for all social strata.

WP6 will be perfectly aligned with WP5 to maximize the impact of the project beyond the context of participants in the clinical trial and their communities.

This WP will perform the regulatory and exploitation activities in order to define the roadmap or requirements and regulatory steps to obtain the marketing authorization approval of SPIOMET for the treatment of PCOS. It will comprise different aspects essential to market a novel medicinal product, as well as to advance the proposed Technology Readiness Levels of the project (from 4 to 6), including the definition of the regulatory pathway, the price and reimbursement strategy, business plan development and intellectual property management.

This WP will include coordination and management activities among the consortium partners and between the consortium and the European Commission. This WP will ensure an efficient operational management, including administrative, financial and legal issues.