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
Clinicaltrials.gov Identifier
Research summary
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.Summary of Results
"About this study
Hemophilia B
Hemophilia B is a rare bleeding condition that is usually inherited and affects mostly men.
When you get a cut or injury, your body uses proteins called clotting factors to stop the bleeding. People with hemophilia don’t have enough of one of these proteins, so their blood doesn’t clot properly. This means they can bleed for a longer time after injuries. In serious cases, they might even bleed inside their body, which can harm organs or joints.
People with Hemophilia B don’t have enough of a clotting factor called Factor IX (9). Currently, the treatment available for Hemophilia B is an injection of Factor IX. This treatment can be provided “on demand”, which means it is given only when the patient bleeds, or regularly, to prevent bleeds from happening.
People with Hemophilia B receiving treatment with their missing clotting factor can sometimes develop inhibitors against the clotting factor, reducing its effects.
Main purpose of this study
The purpose of this study was to learn about the safety and effects of SerpinPC for people with Hemophilia B with inhibitors.
This study was terminated early due to business reasons.
Study population
Who could take part on this study?
Males could be included in this study if they:were aged 12 to 65 years
had Hemophilia B with inhibitors
were not on a regular treatment program with Factor IX and had at least 6 bleeding events in 1 year before study start
were on a regular treatment program with Factor IX and had at least 2 bleeding events per year
Study treatment
The treatment given in this study was:
• SerpinPC: SerpinPC reduces the levels of a protein that regulates blood clotting by limiting the production of an enzyme involved in this process. SerpinPC is an “investigational” medicine that was being studied to treat people with Hemophilia B. Investigational means that the medicine has not yet been approved. SerpinPC was given as an injection under the skin. This is known as “Subcutaneous”.
Overall results of the clinical trial
What were the main results of this study?
How many bleeding events happened during the Observation Period and during the first 24 weeks of the Treatment Period?
This question could not be answered, as the study was terminated early due to business reasons.
What side effects did participants have in this study?
Researchers keep track of all medical problems that participants have when a new treatment is tested. A medical problem may or may not be related to the treatments given in the study. Side effects are unwanted medical problems that happened during the study which the researchers considered related to the study treatment. Researchers need to compare the results of many studies to know whether a medical problem is related to a treatment.
The website listed at the end of this summary may have more information about the side effects that happened.
How many participants stopped treatment because of side effects?
None of the participants given SerpinPC stopped treatment early because of side effects.
What was learned from this study?
The researchers could not make any conclusions because the study was terminated early. The researchers found no new safety concerns with SerpinPC.
Other studies for SerpinPC are not ongoing. The sponsor does not plan to conduct any more studies in the future.
This summary only shows the results of this study. Other studies may have different findings. Researchers and health authorities’ look at the results of many studies to understand which treatments work and if they are safe.
It takes lots of people in many studies around the world to advance medical science and healthcare. Please do not use the results of this study to make healthcare decisions. Always talk to your doctor
before changing your treatment or if you have questions about the study results.
Where can I learn more about this study?
You can find more information about this study on the following websites:
• www.clinicaltrials.gov. Use the NCT identifier NCT05789537 in the search field.
• https://euclinicaltrials.eu/ Use the EU CT identifier 2022 502881 25 00 in the search field.
If you were a study participant and have questions about the results of this study, please speak with the doctor or staff at your study site."REC name
Wales REC 5
REC reference
23/WA/0300
Date of REC Opinion
14 Dec 2023
REC opinion
Further Information Favourable Opinion