CompletedPhase 1/2

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

Georgia, Moldova, United Kingdom39 participantsStarted 2019-08-14

Plain-language 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.

Who can participate

Age range18 Years – 55 Years

SexMALE

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Inclusion criteria

✓. Males age ≥18 years and ≤55 years.
✓. Capable of giving written informed consent to participate after reading the information and consent form, and after having the opportunity to discuss the trial with the investigator or delegate.
✓. Willingness to give written consent to have data entered into The Over-volunteering Prevention System (TOPS).
✓. Sufficient intelligence to understand the nature of the trial and any hazards of participating in it. Ability to communicate satisfactorily with the investigator and to participate in, and comply with the requirements of the entire trial.
✓. Screening D-dimer ≤ 750 µg/L
✓. Non-user of nicotine products i.e. non-smokers or ex-smokers who have stopped smoking or who had stopped using cigarette replacements for at least 6 months before the first dose of IMP.
✓. Male subject willing to use 2 highly effective methods of contraception from the first dose administration until 3 months after dosing.
✓. Subject with a body mass index (BMI) of 18 - 30 kg/m2 and weight ≥ 60 kg.

Exclusion criteria

What they're measuring

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

Timeframe: From Day 1 up to 74 weeks

Trial details

NCT IDNCT04073498

SponsorApcinteX Ltd

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-09-26

View on ClinicalTrials.gov More Hemophilia a trials