Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B

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What is this study about?

This clinical trial is focused on studying the effects of a treatment called SerpinPC for individuals with Severe Hemophilia A or Moderately Severe to Severe Hemophilia B. Hemophilia is a condition where the blood does not clot properly, leading to excessive bleeding. The treatment being tested, SerpinPC, is a solution for injection that contains a modified form of a protein called human alpha-1 proteinase inhibitor. This study aims to evaluate how effective and safe SerpinPC is when given as a regular preventive treatment through subcutaneous injection, which means it is injected under the skin.

The purpose of the study is to assess the efficacy and safety of SerpinPC in people with these types of hemophilia. Participants in the study will receive the treatment over a period of time, and their health will be monitored to see how well the treatment works in reducing bleeding episodes. The study will also look at the overall health and quality of life of the participants while they are receiving the treatment.

Throughout the study, participants will be observed for any changes in their condition, and the number of bleeding episodes they experience will be recorded. The study will help determine if SerpinPC can be a beneficial treatment option for those living with Severe Hemophilia A or Moderately Severe to Severe Hemophilia B. The trial is designed to gather important information that could lead to improved care and management of these conditions in the future.

1 joining the study

Upon joining the study, you will be informed about the purpose and procedures involved. The study aims to evaluate the effectiveness and safety of a medication called SerpinPC for individuals with severe hemophilia A or moderately severe to severe hemophilia B.

2 initial assessment

An initial assessment will be conducted to ensure eligibility. This includes confirming your age, medical history, and current health status. You must be a male aged between 12 and 65 years with the specified type of hemophilia.

Your recent bleeding episodes and current treatment regimen will be reviewed. You should have had at least six documented bleeding episodes in the past six months if you are on an on-demand treatment regimen.

3 observation period

Before starting the medication, there will be an observation period to document any bleeding episodes. This period lasts for at least 12 weeks in Part 1 or 24 weeks in Part 2 of the study.

No bleeding should occur in the seven days before the baseline assessment. If bleeding occurs, the observation period may be extended.

4 medication administration

You will receive SerpinPC as a solution for injection under the skin, known as a subcutaneous injection. The dosage and frequency will be determined by the study protocol.

The medication aims to reduce the number of bleeding episodes and improve your quality of life.

5 monitoring and follow-up

Throughout the study, your health and response to the medication will be closely monitored. This includes regular check-ups and assessments to track any changes in your condition.

The study will evaluate the number of treated bleeds, spontaneous bleeds, and joint bleeds, as well as your overall consumption of coagulation factors.

6 completion of the study

The study is expected to continue until July 2026. Upon completion, the results will be analyzed to determine the efficacy and safety of SerpinPC.

Your participation will contribute valuable information to the understanding and treatment of hemophilia.

Who Can Join the Study?

Must be a male between 12 and 65 years old.
Must have severe Hemophilia A or moderately severe to severe Hemophilia B.
If you have Hemophilia B, you must not have a high titer inhibitor (a high titer inhibitor is a specific level of antibodies in the blood, defined as 5 or more Bethesda Units per milliliter).
Must not need current treatment with bypass agents (medications used to manage bleeding in patients with inhibitors).
Must be part of a prophylaxis program (a preventive treatment plan) or have had at least 6 bleeding episodes that needed treatment in the last 6 months if on an on-demand treatment plan.
Must have at least 12 weeks (for Part 1) or 24 weeks (for Part 2) of recorded bleeding episodes in a previous study or be willing to complete an observation period in another study.
Must not have any bleeding in the 7 days before starting the study (the observation period can be extended if actively bleeding).
Must have adequate blood, liver, and kidney function, and a D-dimer level of 750 micrograms per liter or less (D-dimer is a blood test that helps rule out the presence of an inappropriate blood clot).

Who Cannot Join the Study?

Patients who do not have moderately severe to severe hemophilia B cannot participate.
Only male patients are eligible to participate.
Patients who are part of a vulnerable population may not be eligible. A vulnerable population includes groups like children, elderly, or those with certain disabilities.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Uniwersytecki Szpital Kliniczny Im Jana Mikulicza Radeckiego We Wroclawiu	Wroclaw	Poland
IRCCS Humanitas Research Hospital	Rozzano	Italy
Technische Universitaet Dresden	Dresden	Germany

Other Sites

Site Name	City	Country
University Clinical Hospital Virgen De La Arrixaca	Murcia	Spain
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego	Warsaw	Poland
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Universita’ Degli Studi Di Verona	Verona	Italy
Assistance Publique Hopitaux De Paris	Paris	France
Centre Hospitalier Universitaire De Nantes	Nantes	France
Hospices Civils De Lyon	Lyon	France
Azienda Sanitaria Universitaria Friuli Centrale	Udine	Italy
University Childrens Hospital Queen Fabiola	Brussels	Belgium
Multispecialty hospital for active treatment Sveta Sofia EOOD	Sofia	Bulgaria
Aydkcmzkma Piyxeqdv Hmyoaelr Db Pmzrz	Le Kremlin-Bicetre	France
Vikhatcs Neudmdym fcer Gesspzqkri Gjqd	Berlin	Germany
Cfmzynjgj Uzgdnanxllvxvt Sozhnxrss	Woluwe-Saint-Lambert	Belgium
Ahcojwu Oxpzrzqtkyr Ueujgonvioojh Ceildrgagboo Dsvvn Sqfzfz E Dnpzi Sompesy Dr Teqopn	Turin	Italy
Gbipjw Umgoixpykk Foqoaypti	Frankfurt	Germany
Fldnjscmb Ptjz Lt Iyxqcxwcmwyzh Bnigpwnwe Dfn Hcfbadmw Uborfijcisdvz Lm Psy	Madrid	Spain
Hoolllza Vjfi dbcafbpu	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	21.08.2023
Bulgaria	Not recruiting	21.08.2023
France	Not recruiting	21.08.2023
Germany	Not recruiting	21.08.2023
Italy	Not recruiting	21.08.2023
Poland	Not recruiting	21.08.2023
Spain	Not recruiting	21.08.2023

Trial locations

SerpinPC is a medication being studied for its potential to help people with severe hemophilia A or moderately severe to severe hemophilia B. Hemophilia is a condition where the blood doesn’t clot properly, leading to excessive bleeding. SerpinPC is designed to be given as an injection under the skin, known as a subcutaneous injection. The goal of this medication is to prevent bleeding episodes by helping the blood to clot more effectively. This study is looking at how well SerpinPC works and how safe it is for people with these types of hemophilia. By taking SerpinPC regularly, it is hoped that patients will experience fewer bleeding problems and have a better quality of life.

Severe Hemophilia A – Severe Hemophilia A is a genetic disorder caused by a deficiency of clotting factor VIII, leading to impaired blood clotting. This condition results in frequent and prolonged bleeding episodes, often occurring spontaneously or after minor injuries. Bleeding can occur internally, especially in joints and muscles, causing pain and swelling. Over time, repeated joint bleeds can lead to joint damage and reduced mobility. The severity of bleeding episodes can vary, but in severe cases, they are frequent and can be difficult to control. Individuals with this condition require careful management to prevent and address bleeding episodes.

Moderately Severe to Severe Hemophilia B – Moderately Severe to Severe Hemophilia B is a genetic disorder characterized by a deficiency of clotting factor IX, which is essential for normal blood clotting. This condition leads to frequent bleeding episodes, which can occur spontaneously or after minor injuries. Bleeding often affects joints and muscles, causing pain, swelling, and potential joint damage over time. The severity of bleeding episodes can vary, but in more severe cases, they are frequent and challenging to manage. Individuals with this condition need ongoing care to prevent and treat bleeding episodes effectively.

Trial ID:

2022-502880-39-00

Protocol code:

AP-0102

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Safety and Effectiveness of SerpinPC for Patients with Severe Hemophilia A or B

What is this study about?

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