NCT04073498

The Safety and Tolerability of SerpinPC in Healthy Men and in Men with Severe Blood Disorders (haemophilia a and B)

An Adaptive Design, Phase I/IIa, Open Label, Multicentre, Single Dose Escalation and Multiple Dose Study to Investigate the Safety, Tolerability and Pharmacokinetics of Intravenous and Subcutaneous Doses of SerpinPC in Healthy Male Volunteers and Male Participants with Severe Haemophilia a or B with or Without Inhibitors

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 39 (actual)
• Sponsor: ApcinteX Ltd · Industry
• Sex: Male
• Age: 18 Years – 55 Years
• Healthy volunteers: Accepted

Summary

The purpose of this study is to investigate the safety and activity in the body of a new drug called SerpinPC. The study will be split into 7 parts: Part 1a will be conducted in healthy male volunteers in the UK (up to 15) and Parts 1b, 2, 3, 4, 5 and 6 will be conducted in haemophilia A \& B patients in Moldova and Georgia. Part 1a of the study will look at how safe the drug is when given as single doses to healthy volunteers at different strengths and via 2 different routes of administration (through a vein or via an injection under the skin). Parts 1b, 2, 3, 4, 5 and 6 of the study will look at the safety of the drug when given as an injection under the skin to patients with severe haemophilia A or B. The study will also investigate how the levels of the drug in the blood change over a period of time and how the drug acts in the body by taking blood samples. These blood samples will measure the concentration of the drug in the blood and measure certain aspects of the blood to determine how the drug affects them. The study sponsor (ApcinteX) is developing this drug for the treatment of haemophilia A and haemophilia B, which are 2 types of rare blood disorders which affect the body's ability to form blood clots. Patients who have haemophilia A and B do not have certain clotting factors in their blood which means that they experience difficulty in stopping bleeding after injury and can be prone to extended periods of bleeding. Current treatments for haemophilia involves injections which replace the missing factors in the blood. However these treatments are short term and therefore patients require regular treatments in order to manage the condition. Therefore, there is a need to develop more effective treatments which provide longer term benefits. The aim of SerpinPC is to prevent bleeding rather than to have to treat bleeds to minimise pain and damage after they have occurred.

Conditions

• Hemophilia a
• Hemophilia B

Interventions

Type	Name	Description
DRUG	SerpinPC	Administered as IV infusion (over 30 minutes) or SC injection (abdomen) according to cohort dosing instructions.
DRUG	Placebo	Matching placebo administered by SC injection according to cohort dosing instructions.

Timeline

• Start date: 2019-08-14
• Primary completion: 2024-09-26
• Completion: 2024-09-26
• First posted: 2019-08-29
• Last updated: 2025-02-06

Locations

3 sites across 3 countries: Georgia, Moldova, United Kingdom