Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)


Özen A. O. , Harari O.(Executive), Aydıner E. , Barış S. , Kocamış B.

Other Supported Projects, 2020 - 2023

  • Project Type: Other Supported Projects
  • Begin Date: January 2020
  • End Date: March 2023

Project Abstract

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