NCT04073498

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

Completed Phase 1, PHASE2 Last updated 6 February 2025

What this trial tests

Phase 1, PHASE2 trial testing SerpinPC in Hemophilia a in 39 participants. Completed in 26 September 2024.

Timeline

14 August 2019

Primary endpoint
26 September 2024

26 September 2024

Quick facts

Lead sponsor	ApcinteX Ltd
Phase	Phase 1, PHASE2
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	single
Primary purpose	treatment
Enrollment	39
Start date	14 August 2019
Primary completion	26 September 2024
Estimated completion	26 September 2024
Sites	3 locations across Georgia, United Kingdom, Moldova

Drugs / interventions tested

• SerpinPC — full drug profile →
• Placebo

Conditions studied

• Hemophilia a — all drugs for Hemophilia a →
• Hemophilia B — all drugs for Hemophilia B →

Sponsor

ApcinteX Ltd — full company profile →

Who can join

Adults 18 to 55, male only, with Hemophilia a or Hemophilia B. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Therapeutic SERPINs: Improving on Nature.
   Maas C, de Maat S. · · 2021 · cited 23× · PMID 33869308 · DOI 10.3389/fcvm.2021.648349
2. Updates on Novel Non-Replacement Drugs for Hemophilia.
   Gualtierotti R, Pasca S, Ciavarella A, Arcudi S, et al · · 2022 · cited 22× · PMID 36297295 · DOI 10.3390/ph15101183
3. Non-factor therapies for bleeding disorders: A primer for the general haematologist.
   Swan D, Mahlangu J, Thachil J. · · 2022 · cited 18× · PMID 36051064 · DOI 10.1002/jha2.442
4. Advances in biopharmaceutical products for hemophilia.
   Wu J, Liu X, Yang H, He Y, et al · · 2024 · cited 4× · PMID 39717090 · DOI 10.1016/j.isci.2024.111436
5. Hemostats in the clinic.
   Joshi M, Zhao Z, Mitragotri S. · · 2024 · cited 4× · PMID 39545083 · DOI 10.1002/btm2.10673
6. Absence of Effect of Emicizumab on D-Dimer Concentrations in Adult Patients with Severe Hemophilia A.
   Iarossi M, Lambert C, Hermans C. · · 2022 · cited 1× · PMID 36474347 · DOI 10.1177/10760296221143382
7. Proteomic Profiling of Protease-Primed Virus-Permissive Caco-2 Cells Display Abortive-Interferon Pathway and Deregulated Thromboinflammatory SERPINS
   Sharma M, Panda NK. · · 2020 · DOI 10.20944/preprints202006.0206.v1