Work Plan

Work Package 1 - Management and Coordination:

The ultimate objectives of the project management activities are to provide efficient management and administration of the project, while fulfilling all legal and European Commission requirements.

Work Package 2 - Production and Release of Clinical-grade MSC:

In this Work Package we will manufacture GMP clinical grade fetal MSC according to standardized protocols, analyse the cell product, define a set of cell product parameters, release the cell product and control the cell product quality at the receiver institution.

Work Package 3 - Molecular Diagnosis Including Non-invasive Prenatal Diagnosis of OI:

We will deliver timely definitive molecular pre- and postnatal diagnosis of cases eligible for MSC therapy and improve access to safe prenatal diagnosis by developing comprehensive non-invasive prenatal diagnosis based on analysis of cell free DNA in maternal plasma.

Work Package 4 - Phase I/II Clinical Trial:

Here we will conduct an open-label multi-centre phase I/II clinical trial to study the safety and efficacy of prenatal and/or postnatal repeated transplantation of human fetal liver MSC to ameliorate severe OI in comparison to a matched historical and prospective controls.

Work Package 5 - Clinical Trial: Patient Follow-up:

The aim of this work is to assess the clinical course after transplantation of MSC.

Work Package 6 - Patient Experience and Ethical Issues:

Here we will explore the views and experiences of parents regarding OI and the MSC therapy. We will assess the quality of life of patients undergoing MSC therapy and examine current ethical standards, debates and risks associated with stem cell transplantation and clinical trials.

Work Package 7 - Health Economic Assessment:

We will assess the health economic impact of the use of MSC in the treatment of severe OI by developing a health economic model for prenatal and postnatal transplantation of MSC to OI patients.

Work Package 8 - Data Management:

The output of this work package will be to develop a robust IT solution for the BOOSTB4 project. This will be achieved using a proven commercially available internet-based platform to develop a specific database for an intervention and follow-up study of children suffering from OI and treated with fetal MSC.

Work Package 9 - Communication, Dissemination and Exploitation:

Here we will ensure the successful dissemination and exploitation of the project results and promote the project through well planned and effective communication activities. We will also support the engagement of all consortium participants in the function of the project.