Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.
Status:
Completed
Trial end date:
2019-08-26
Target enrollment:
Participant gender:
Summary
The objectives of the study are to assess the safety, tolerability, preliminary efficacy and
PK of multiple subcutaneous (SC) doses of pegcetacoplan in subjects with paroxysmal nocturnal
hemoglobinuria (PNH) who have not received treatment with eculizumab in the past.
An exploratory objective of the study is to assess the pharmacodynamics (PD) of multiple SC
doses of pegcetacoplan when administered to PNH patients.