ViRap
Project data
Project financing: 23 119 125 PLN (including financing for UMED: 1 528 640,00 PLN), financed by the Medical Research Agency (ABM)
Project description:
The project will test safety and efficacy of a new therapy for neurological disorders (epilepsy, mental retardation, autism) and tumours (brain, heart, kidney, retinal tumours) in children with a genetically determined rare disease (Tuberous sclerosis – TSC). The study aims to compare two drugs, an anti-epileptic and an mTOR inhibitor, for preventive treatment in tuberous sclerosis. The ViRap study will also prospectively analyse biomarkers of epileptogenesis and oncogenesis, which will contribute to the knowledge on the mechanisms of epilepsy and cancer development.
The primary objective of the ViRap clinical trial is to compare the safety, tolerability and efficacy of an anti-epileptic drug (vigabatrin) and an mTOR inhibitor (rapamycin) in the prevention of epilepsy and mental retardation and autism spectrum disorders in infants with TSC. The secondary objective of the study will be to evaluate the effect of the use of both drugs on the development of tumour lesions in children with TSC. The tertiary objective will be to assess the effect of treatment on biomarkers of epileptogenesis and oncogenesis in TSC.
Beneficiary
Consortium composed of:
Institute “Children’s Health Centre Memorial” (Leader)
Medical University of Lodz (Partner)
TRANSITION TECHNOLOGIES SCIENCE SP. Z O.O. (Partner)
Medical University of Warsaw (WUM) (Partner)
Brigham and Women’s Hospital (Partner)
Coordinating Principal Investigator
prof. Katarzyna Kotulska-Jóźwiak, MD, PhD
UMED Site Principal Investigator
prof. Wojciech Młynarski, MD, PhD