We have previously demonstrated that low levels of serum IGF-1 and IGFBP-3 in children born preterm are strongly associated with ROP as well as other morbidities in preterm infants (e.g. poor growth, lung disease and poor brain development).
Based on these results we have clinically investigated the concept of restoring IGF-1/IGFBP-3 to in utero levels in premature infants. We have conducted and finalized a Phase I, pharmacokinetic study of intravenously administered rhIGF-I/rhIGFBP-3 complex (mecasermin rinfabate) to five very preterm children (GA 26-29 weeks). No side effects (b-glucose, heart rate, blood pressure) were seen and the dose and mode of delivery was established for Phase II study (Hellström A et al Ped Res 2009) (Ley D et al Ped Res 2013).
Carrying out this project is a major multi-disciplinary project with many participants, contributing with a broad range of competences, involved. For a successful implementation of the project, national and international experts in ophthalmology, neonatology, pharmacokinetics, pharmaceutics, GMP (Good Manufacturing Practice), GCP (Good Clinical Practice), GLP (Good Laboratory Practice) are needed. The Phase II study is an international multi-center trial with participation from the neonatal clinics in Lund and Stockholm.
The pharmaceutical company, Premacure AB, Uppsala, Sweden now acquired by Shire®, is coordinating all activities before, during and after the trial and is also in charge of obtaining all regulatory authorizations before the study can commence. Pharma Consulting Group AB (PCG), Uppsala, Sweden has been contracted as independent monitor of the study. This will guarantee that the work is carried out in accordance with the regulatory provisions (GCP).
As we are focusing on factors that promote neural, vascular and metabolic development, our findings regarding ROP are likely to be applicable on several aspects of complications of premature birth and will hopefully provide benefits for the whole lifespan.