Özen A. O., Harari O.(Yürütücü), Aydıner E., Barış S., Kocamış B.
Diğer Uluslararası Fon Programları, 2020 - 2023
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Proje Türü:
Diğer Uluslararası Fon Programları
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Başlama Tarihi:
Ocak 2020
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Bitiş Tarihi:
Mart 2023
Proje Özeti
Brief Summary:
The primary objective of the study is to determine the effect of pozelimab on CD55-deficient protein-losing enteropathy (PLE; CHAPLE).
The secondary objectives of the study are:
- To evaluate the safety and tolerability of pozelimab in patients with CD55-deficient PLE disease
- To determine the effects of pozelimab on albumin and other serum proteins (total protein, immunoglobulins)
- To determine the effects of pozelimab on ascites
- To determine the effect of pozelimab on health-related quality of life
- To determine the effect of pozelimab on lab abnormalities observed in CD55-deficient PLE such as hypertriglyceridemia, thrombocytosis, and hypovitaminosis B12
- To describe the effects of pozelimab on the sparing of concomitant medications and reduction in hospitalization days
- To determine the effects of pozelimab on growth
- To characterize the concentration of pozelimab in patients with CD55-deficient PLE
- To assess the occurrence of ADA for pozelimab in patients with CD55-deficient PLE disease