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Genetic Determinants of the Outcome of Immune Tolerance Induction therapy in patients with severe haemophilia A and inhibitors

The aim of this study is to identify genetic determinants that are associated with successful immune tolerance induction in patients with haemophilia A and inhibitors.

A phase 1B of crizotinib either in combination or as single agent in pediatric patients with ALK, ROS1 or MET positive malignancies;Study ITCC 053

This study has been transitioned to CTIS with ID 2023-504880-18-00 check the CTIS register for the current data. Primary• To determine the RP2D of crizotinib in combination with temsirolimus • To determine the safety and preliminary activity of…

Paediatric Inflammatory Bowel Diseases Network for Safety, Efficacy, Treatment and Quality improvement of care: The PIBDNet inception cohort and safety registry

The primary objective of the PIBD-NET inception cohort is to search for factors predictive for outcome, specific severe adverse events (SAEs) and for predictors of response or non-response to therapy. The secondary objective is the identification of…

Phase 1b/2 Study of Carfilzomib in Combination With Induction Chemotherapy in Children With Relapsed or Refractory Acute Lymphoblastic Leukemia

The Primary Objectives of the Phase 1b are: • To assess the safety and tolerability of carfilzomib, alone and in combination with induction chemotherapy, for the treatment of children with relapsed or refractory ALL • To determine the maximum…