PRESent-2
Research type
Research Study
Full title
A Global, Open-label, Adaptive Design Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
IRAS ID
1006538
Contact name
Shana Abbott
Contact email
Sponsor organisation
ApcinteX Limited
Eudract number
2021-003775-33
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 severe haemophilia A with or without inhibitors or moderately severe to severe haemophilia B without inhibitors. A separate research study is being conducted to investigate SerpinPC as a potential treatment for haemophilia B with inhibitors.
Haemophilia A and B are genetic bleeding conditions that are caused by lack of proteins, called factors, responsible for blood clotting. Treatments for haemophilia involve infusions of missing factors or bypassing agents.
There is a need to develop new treatments for patients with haemophilia that are more convenient and use better ways to prevent bleeding. SerpinPC is a new treatment, being developed as a “non-factor replacement therapy” (does not reply on replacing factors), to prevent bleeding episodes in patients with haemophilia A and 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 which is responsible for decreased thrombin production. It is believed that, by inhibiting this protein, 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.
A total of approximately 120 participants will be recruited for this study globally. Participating countries are: Armenia, Brazil, Barbados, Canada, Egypt, Japan, India, South Africa, Turkey, USA, France, Spain, Italy, Germany, Belgium and Poland. Approximately 60 participants with either haemophilia A or B will participate in Part 1 and an additional 60 participants will participate in Part 2. At least 30 participants in Part 2 will have haemophilia B. 10 patients are planned for the UK with a total study duration up to 76 weeks.REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
23/YH/0050
Date of REC Opinion
27 Sep 2023
REC opinion
Further Information Favourable Opinion