The following is a very concise summary of the work performed split out per area of work
1. Management: All boards were established with regular meeting times (in person and/or digitally) and clear goals. A management plan was made to coordinate the different partners in our group.
2. Study protocols: All documents for ethical and regulatory approval were prepared. These have been fully approved in the Netherlands and in Germany. Approval in the UK has not been achieved and mitigation measures were applied (see point 4 below). We made several small changes to the protocol to better streamline processes and to increase safety and conform for included patients.
3. Data collection and planning: A data management plan was created and completed. An electronic data reporting/questionnaire collection form was created. Regular reports are sent to the Data Safety Board.
4. Randomized controlled trial (RCT): After initial delays due to COVID19, the RCT phase of our the STOPFOP clinical trial has been successfully completed. More specifically, 17 patients have enrolled in the study: 11 in VUMC Amsterdam, the Netherlands and 6 in Garmisch partenkirchen (GAP), Germany. All patients finished the RCT and started the open label phase. The London site (RNOH) was unable to open. British patients have therefore travelled to Amsterdam to participate in the study via the VUMC site, which was a manageable journey for them. No serious side effects occurred during the RCT.
5. Open label extension (OLE): All participants who completed the RCT phase progressed to the OLE phase of the study, as foreseen in the DoA. The OLE study was successfully completed too. No serious side effects occurred during the RCT and overall, the participants of the study experienced benefits from the repurposed drug. However, further analysis of the data is required before drawing conclusions and make them public.
6. Prolonged extension open-label study (beyond the STOPFOP project): A prolonged extension study was designed for those patients who completed the study and wanted to continue due to the benefit from the repurposed drug experienced. All 17 patients that completed the OLE phase of the study continued to the prolonged OLE phase (GAP 6; VUMC 11). The OLE part of the study has been continued after the completion of the IMI2 STOPFOP project, as it was beyond the project’s scope.
7. Sustainability plan: Contact with EMA was made for orphan drug designation and all relevant documents have been prepared. Further progress on this subject may take place after the end of the project.
8. Dissemination and communication: Dissemination and exploitation plans have been drawn up and regularly updated. A logo and website were created that has been frequently visited. The project has been presented and discussed internationally on a very regular basis at scientific conferences, FOP patient meetings and educational events. A peer reviewed article has been published on the original design of the study:
https://pubmed.ncbi.nlm.nih.gov/35650602/(se abrirá en una nueva ventana).
9. Ethics: An independent ethics advisor has been involved throughout the lifetime of the project who supported and guided the it. This advisor has regularly checked the status of the project and provided reports with recommendations.
