Phase 2a non-commercial and non-randomized intervention study. evaluating the effectiveness of crizotinib in the treatment of children with severe form of neurofibromatosis type 2, in particular those who cannot undergo surgery and radiotherapy

Project Title
Niekomercyjne i nierandomizowane badanie interwencyjne fazy 2a. oceniające skuteczność produktu leczniczego kryzotynib w terapii dzieci z ciężką postacią neurofibromatozy typu 2., w szczególności niepoddających się leczeniu chirurgicznemu oraz/lub radioterapii.
Financing Institution

Medical Research Agency

Medical Research Agency

dr n. med. Marek Karwacki
Project Objective

Type 2 neurofibromatosis (NF-2) is an ultra-rare primary cancer and genetically determined disease, most often manifested in young adults. The childhood form of this disease is a unique presentation, but unfortunately it is burdened with many complications, it develops much faster than in adults, it is responsible for its severe course and an outstanding shortening of the survival time, and severe, complex disability.

Until recently, the therapies used, which undoubtedly made a real revolution in oncology and significantly improved the prognosis of patients with many cancers, were reserved only for the treatment of malignant tumors. Treatment of benign tumors has been and is the domain of oncological surgery, but the activities of a specialist doctor are usually associated with many serious complications and are rarely effective (the procedure is not radical).

Based on the described premises, and due to the inability to offer an effective therapy for reducing or at least inhibiting the growth of tumors, a study on the treatment of children with NF-2 with crizonitib was developed. Treatment that, even if it does not prevent the formation of tumors, should significantly reduce their complex disability and bring them back to society.

The study will include patients aged 4-15 years with confirmed type 2 neurofibromatosis (NF-2). They will be cared for for a period of 15 months, during which they will be taking the drug (daily dosing) and will undergo monthly check-ups to assess the safety of the therapy. Additionally, patients will also undergo an ophthalmological examination. The aim of the clinical trial is to evaluate the effectiveness of crizotinib therapy, resulting in at least the arrest of the growth dynamics of the tumor or many tumors of both the central and peripheral nervous systems.

The research is carried out by a team of doctors led by the main researcher - Dr. Marek Karwacki, specialist in pediatrics (2nd degree) as well as pediatric hematology and oncology with a doctorate in clinical genetics, employee of the University Clinical Center of the Medical University of Warsaw, Academic and Clinical Department of Oncology, Pediatric Hematology, Clinical Transplantology and Pediatrics.