Evaluation of the efficacy and safety of empagliflozin in the treatment of neutropenia in patients with glycogenosis 1b "- EMPAtia

Project Title
„Ocena skuteczności i bezpieczeństwa empagliflozyny w leczeniu neutropenii u pacjentów z glikogenozą 1b” - EMPAtia
Financing Institution

Medical Research Agency

Medical Research Agency

dr Piotr Sobieraj
Project Objective

The aim of the clinical trial is to evaluate the safety and efficacy of empagliflozin therapy in patients with GSD 1b. Type Ib glycogenosis is an ultra-rare, congenital, inherited autosomal and recessive disease. It manifests itself, inter alia, with severe hypoglycaemia, hepatomegaly and severe neutropenia with impaired neutrophil function, resulting in recurrent severe, sometimes life-threatening bacterial and fungal infections and inflammatory bowel disease. In the treatment of neutropenia in patients with GSD Ib, filgrastim is used, a drug that increases neutrophil count but does not improve neutrophil function in this group of patients, and is given by subcutaneous injection, usually daily. Some patients do not respond to filgrastim treatment, and all persist with recurrent infections despite treatment. Treatment with the preparation in most patients causes complications in the form of splenomegaly, sometimes accompanied by hypersplenism, gingival hyperplasia, and long-term complication of filgrastim treatment may be acute myeloblastic leukemia and myelodysplastic syndrome.

It is an open study. The study group will consist of pediatric and adult patients with biochemically and / or molecularly diagnosed glycogenosis Ib and neutropenia.

The study is carried out in two clinical centers in Poland: at the Children's Health Institute in Warsaw (IPCZD) and at the Medical University of Warsaw (MUW).

Project implemented in the period from  1 October 2020 to  31 March 2025