Study on the Safety and Effectiveness of SerpinPC for Patients with Hemophilia B with Inhibitors

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What is this study about?

This clinical trial is focused on studying a condition known as Hemophilia B, which is a genetic disorder that affects the blood’s ability to clot properly. People with this condition can experience excessive bleeding even from minor injuries. The study is testing a new treatment called SerpinPC, which is a modified form of a protein known as human alpha-1 proteinase inhibitor. This treatment is given as a solution for injection under the skin.

The purpose of the study is to evaluate how effective and safe SerpinPC is for people with Hemophilia B who have developed inhibitors. Inhibitors are antibodies that can make standard treatments less effective. Participants in the study will receive regular injections of SerpinPC and will be monitored over a period of time to see how well the treatment works in reducing bleeding episodes. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects.

The study will last for a total of 48 weeks, during which participants will be closely observed for any changes in their condition and any side effects they might experience. The main focus will be on the number of bleeding episodes that require treatment, as well as the overall health and quality of life of the participants. This trial aims to provide valuable information on whether SerpinPC can be a beneficial treatment option for those living with Hemophilia B with inhibitors.

1 joining the study

Upon joining the study, you will be required to provide written informed consent. This means you agree to participate after understanding the study details.

You will have the opportunity to discuss the study with the investigator or their delegate to ensure all your questions are answered.

2 initial assessments

You will undergo initial assessments to confirm eligibility. These assessments include checking your blood count, liver function, and kidney function.

You must have a platelet count of at least 100,000 per microliter and a hemoglobin level of at least 10 grams per deciliter.

3 observational period

If you have not already completed a 12-week documentation of bleeding episodes, you will need to do so before starting the medication.

This period helps establish a baseline for your bleeding rate.

4 medication administration

You will receive the medication SerpinPC as a subcutaneous injection. This means the solution is injected under your skin.

The main objective is to evaluate the efficacy and safety of SerpinPC in reducing bleeding episodes.

5 treatment period

During the first 24 weeks, your bleeding rate will be closely monitored. This is known as the primary endpoint of the study.

You will continue to document any bleeding events and medication usage in a diary.

6 extended treatment period

The study will also evaluate your bleeding rate over the first 48 weeks and beyond, as part of the secondary endpoints.

Your overall health and quality of life will be assessed using a questionnaire designed for individuals with hemophilia.

7 end of study

The study is estimated to end by October 2025. At this point, final assessments will be conducted to evaluate the long-term effects of the treatment.

You will have a final discussion with the study team to review your experience and any findings.

Who Can Join the Study?

Must be a male between 12 and 65 years old.
Must have adequate blood health, meaning:
- Platelet count of at least 100,000 per microliter of blood.
- Hemoglobin level of at least 10 grams per deciliter of blood.
Must have adequate liver health, meaning:
- Total bilirubin level no more than 1.5 times the normal limit (except for Gilbert syndrome).
- Liver enzymes (AST and ALT) no more than 3 times the normal limit.
- No signs of liver cirrhosis.
Must have adequate kidney health, meaning:
- Serum creatinine level no more than 2 times the normal limit.
Must be able to use a diary to record bleeding events and medication use (help from a caregiver is allowed for teenagers).
If sexually active with a partner who can become pregnant, must agree to use effective birth control during the study.
Must be able to provide written consent to participate in the study (teenagers need assent and parental/guardian consent).
Must have a history of Hemophilia B with a factor IX level of 5% or less.
If currently on a preventive treatment program, must be willing to stop the treatment before starting the study medication.
Must have a history of factor IX inhibitor requiring treatment with bypass agents.
Must have a documented annual bleeding rate (ABR) of at least:
- 6 in the past year if not on a preventive treatment.
- 2 if on a preventive treatment.
Must have at least 12 weeks of documented bleeding episodes in a previous study or be willing to complete a 12-week observation period.
Must have no bleeding in the 7 days before starting the study (the observation period can be extended if there is an active bleed).
Must have a D-dimer level of 750 micrograms per liter or less (or 1750 mg/L if there is a resolving bleed).

Who Cannot Join the Study?

Patients who do not have Hemophilia B cannot participate. Hemophilia B is a bleeding disorder where blood does not clot properly.
Only male patients can participate. Female patients are not eligible.
Patients who are not within the specified age range cannot participate. The study is for certain age groups only.
Patients who are considered part of a vulnerable population may not be eligible. This means groups who might need special protection or care.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Centre Hospitalier Universitaire De Lille	Lille	France

Other Sites

Site Name	City	Country
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Assistance Publique Hopitaux De Paris	Paris	France
Hospices Civils De Lyon	Lyon	France
Azwkhivyvr Pmxukvrr Hcvvfkzm Da Pfjiv	Le Kremlin-Bicetre	France
Gdalqy Uybhcgesum Fojqxjpgq	Frankfurt	Germany
Fabydvyow Pcad Lf Impkmgpzielwy Bpririyyd Dzs Hhmdwtiq Uxsbhvqgqodps Lt Pyf	Madrid	Spain

Trial status

Country	Status	Recruitment Start
France	Not recruiting	21.08.2023
Germany	Not recruiting	21.08.2023
Italy	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 Hemophilia B, a condition where blood doesn’t clot properly. This medication is given as an injection under the skin. The goal of using SerpinPC is to see if it can reduce bleeding episodes in people who have developed inhibitors, which are antibodies that make it harder for standard treatments to work. By using SerpinPC, researchers hope to find a new way to manage Hemophilia B and improve the quality of life for those affected by this condition.

Hemophilia B – Hemophilia B is a genetic bleeding disorder caused by a deficiency of clotting factor IX, which is essential for blood coagulation. This condition leads to prolonged or spontaneous bleeding episodes, often occurring in joints, muscles, and soft tissues. The severity of bleeding can vary, with some individuals experiencing frequent and severe bleeds, while others may have milder symptoms. Over time, repeated bleeding into joints can cause joint damage and chronic pain. The disease is inherited in an X-linked recessive pattern, primarily affecting males, while females are typically carriers. Hemophilia B is a lifelong condition that requires careful management to prevent and control bleeding episodes.

Trial ID:

2022-502881-25-00

Protocol code:

AP-0103

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Safety and Effectiveness of SerpinPC for Patients with Hemophilia B with Inhibitors

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?