PRESent-3
Research type
Research Study
Full title
A Global, Open-label Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Hemophilia B With Inhibitors
IRAS ID
1007032
Contact name
Shana Abbott, Senior Director Clinical Operations
Contact email
Sponsor organisation
ApcinteX Limited
Eudract number
2022-000879-39
Research summary
ApcinteX Limited has begun a study of an investigational drug (also known as the “study drug”) called SerpinPC as a possible treatment for haemophilia B.
Haemophilia B is a genetic bleeding condition that is caused by lack of proteins responsible for blood clotting. The main treatments for haemophilia involve frequent infusions of missing factors or bypassing agents when inhibitors are present. There is a need to develop new treatments for patients with haemophilia that are more convenient and use other ways to prevent bleeding. SerpinPC is a new treatment being developed as a ‘non-factor replacement therapy’ in patients with haemophilia. Because SerpinPC does not rely on replacing a specific clotting protein, like factor IX (factor 9), it can be used to prevent bleeding episodes in haemophilia B with or without inhibitors. SerpinPC is given as an injection under the skin and seeks to restore haemostatic balance by inhibiting an anti-coagulant protein called ‘activated protein C’, that circulates in the blood and decreases the production of thrombin, the most important clotting protein. It is believed, that by decreasing activated protein C, SerpinPC will allow the formation of enough thrombin to prevent bleeding.
The main purpose of this study is to learn how well the study drug works and how safe the study drug is when given via an injection under the skin. Approximately 12 participants with haemophilia B with inhibitors will be participating.REC name
Wales REC 5
REC reference
23/WA/0300
Date of REC Opinion
14 Dec 2023
REC opinion
Further Information Favourable Opinion