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
Clinicaltrials.gov Identifier
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.Lay Summary of Results
"About this study
Hemophilia A and B
Hemophilia A and B are rare bleeding conditions that are usually inherited and affect 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 A don’t have enough of a clotting factor called Factor VIII (8), whereas people with Hemophilia B don’t have enough Factor IX (9). Currently, the treatment available for Hemophilia A is an injection of Factor VIII, and for Hemophilia B is an injection of Factor IX. These treatments can be provided “on demand”, which means they are given only when the patient bleeds, or regularly, to prevent bleeds from happening.
People with Hemophilia A or B receiving treatment with their missing clotting factor can sometimes develop inhibitors against the clotting factor, reducing its effects.
Hemophilia A is considered severe when clotting factor activity levels are below 1%. Hemophilia B is considered moderately severe to severe when clotting factor activity levels are between below or equal to 2%. The normal range of clotting factor activity levels is between 50% to 150%
Main purpose of this study
The purpose of this study was to learn about the safety and effects of SerpinPC for people with severe Hemophilia A, with or without inhibitors, or moderately severe to severe Hemophilia B, without inhibitors.
Researchers thought that SerpinPC could help promote blood clotting, regardless of disease severity or the presence of 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 severe Hemophilia A with or without inhibitors
had moderately severe or severe Hemophilia B without inhibitors
were on a regular treatment program or receiving “on demand” treatment with clotting factor
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 A or 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 Part 2 of the Treatment Period in participants with Hemophilia B who were previously receiving “on demand” treatment?
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 NCT05789524 in the search field.
• https://euclinicaltrials.eu/ Use the EU CT identifier 2022-502880-39-00 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
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