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Eptacog Beta (Activated)

This article discusses clinical trials investigating the use of Eptacog Beta (Activated), also known as CEVENFACTA, in the treatment of Hemophilia A patients with FVIII inhibitors. The trials aim to evaluate different treatment approaches, including immune tolerance induction (ITI) and bleeding prevention, to improve patient outcomes and quality of life.

Table of Contents

What is Eptacog Beta (Activated)?

Eptacog Beta (Activated) is a medication used in the treatment of hemophilia A, particularly for patients who have developed inhibitors. It is also known by its brand name CEVENFACTA[1]. This medication belongs to a class of drugs called recombinant factor VIIa (rFVIIa), which are laboratory-made versions of a natural clotting factor in the body[1].

What is it used for?

Eptacog Beta (Activated) is primarily used to treat and prevent bleeding in patients with hemophilia A who have developed inhibitors. Hemophilia A is a genetic disorder that impairs the body’s ability to form blood clots, leading to excessive bleeding. Some patients with hemophilia A develop inhibitors, which are antibodies that interfere with the treatment using standard factor VIII replacement therapy[1].

How is it administered?

CEVENFACTA is administered through intravenous injection, which means it is injected directly into a vein. It comes in three different strengths[1]:

  • 1 mg (45 KIU) powder and solvent for solution for injection
  • 2 mg (90 KIU) powder and solvent for solution for injection
  • 5 mg (225 KIU) powder and solvent for solution for injection

The dosage is typically measured in micrograms per kilogram (μg/Kg) of body weight. The exact dosage and frequency of administration will be determined by your healthcare provider based on your individual needs and response to treatment[1].

Clinical Trial Information

A clinical trial is currently being conducted to evaluate the effectiveness of various treatment approaches for hemophilia A patients with inhibitors, including the use of Eptacog Beta (Activated)[1]. The trial, titled “MOdern Treatment of Inhibitor-PositiVe PATiEnts with Haemophilia A,” aims to assess different treatment strategies, including:

  • Immune Tolerance Induction (ITI) therapy
  • Prophylactic treatment with bypass agents like Eptacog Beta (Activated)
  • Combination therapies

The trial is evaluating outcomes such as the success rate of ITI, bleeding rates, and the frequency and severity of bleeding episodes[1].

Potential Benefits

The use of Eptacog Beta (Activated) may offer several potential benefits for hemophilia A patients with inhibitors[1]:

  • Control and prevention of bleeding episodes
  • Management of bleeding during and after surgical procedures
  • Potential reduction in the frequency and severity of bleeding events
  • An alternative treatment option for patients who have developed inhibitors to standard factor VIII therapy

Important Considerations

While Eptacog Beta (Activated) can be an effective treatment for many patients, it’s important to be aware of the following[1]:

  • Medical Supervision: This medication should only be used under the guidance of a healthcare professional experienced in treating hemophilia.
  • Monitoring: Regular check-ups and blood tests may be necessary to monitor the effectiveness of the treatment and adjust the dosage if needed.
  • Potential Side Effects: As with any medication, there may be side effects. Your doctor will discuss these with you and monitor for any adverse reactions.
  • Individual Response: The effectiveness of the treatment can vary from person to person. Your healthcare provider will work with you to determine the best treatment plan for your specific needs.

If you have hemophilia A with inhibitors, discuss with your healthcare provider whether Eptacog Beta (Activated) might be an appropriate treatment option for you[1].

Aspect Details
Study Title MOdern Treatment of Inhibitor-PositiVe PATiEnts with Haemophilia A – An International Low-Interventional Pragmatic Investigator Initiated Trial
Main Objective Evaluate ITI outcomes and compare annualized bleeding rates in different treatment groups
Participant Eligibility Any age, Hemophilia A with historical inhibitor titre ≥ 0.6 BU/mL
Treatment Groups Group 1 and 2: ITI with various factor products; Group 3: Non-ITI treatment
Primary Endpoints ITI success criteria (Groups 1 and 2), ABR comparison (Group 3 vs. Groups 1 and 2)
Secondary Objectives Time to ITI outcome, bleeding frequency and severity, treatment costs, adverse reactions
Study Drug Eptacog Beta (Activated) – CEVENFACTA (1 mg, 2 mg, 5 mg strengths)
Administration Route Intravenous injection
Maximum Treatment Period 60 months

FAQ

  • What is the main purpose of the clinical trial using Eptacog Beta (Activated)? The main purpose is to evaluate different treatment approaches for Hemophilia A patients with FVIII inhibitors, including immune tolerance induction (ITI) outcomes and annualized bleeding rates (ABR).
  • Who can participate in this clinical trial? Participants can be of any age with Hemophilia A of any severity, who have a historical inhibitor titre ≥ 0.6 BU/mL, including those who have failed previous ITI attempts.
  • What are the primary endpoints of the trial? The primary endpoints include evaluating ITI outcomes in Groups 1 and 2, and comparing the annualized bleeding rate (ABR) in Group 3 with Groups 1 and 2.
  • How is Eptacog Beta (Activated) administered in the trial? Eptacog Beta (Activated) is administered intravenously as a solution for injection, available in different strengths (1 mg, 2 mg, and 5 mg).
  • What are some of the secondary objectives of the trial? Secondary objectives include assessing the frequency and severity of bleeding episodes, the number of infusions required to control bleeds, and evaluating treatment costs and adverse drug reactions.

Ongoing Clinical Trials on Eptacog Beta (Activated)

  • Study on Emicizumab and Drug Combination for Patients with Haemophilia A and FVIII Inhibitors

    Recruiting

    1 1 1 1
    Investigated drugs:
    Bulgaria Croatia Finland Germany Norway Spain +1

Glossary

  • Hemophilia A: A genetic bleeding disorder caused by a deficiency in blood clotting factor VIII (FVIII).
  • FVIII Inhibitors: Antibodies that neutralize the clotting function of factor VIII, making standard treatment less effective.
  • Immune Tolerance Induction (ITI): A treatment approach aimed at eliminating inhibitors by exposing the immune system to regular doses of clotting factor.
  • Bethesda Units (BU): A measurement used to quantify the level of inhibitors in the blood.
  • Annualized Bleeding Rate (ABR): The number of bleeding episodes a patient experiences in a year.
  • Eptacog Beta (Activated): A recombinant form of activated factor VII used to treat and prevent bleeding in hemophilia patients with inhibitors.
  • CEVENFACTA: The brand name for Eptacog Beta (Activated) used in this clinical trial.
  • Prophylactic Therapy: Regular treatment given to prevent bleeding episodes in hemophilia patients.
  • Adverse Drug Reactions (ADRs): Unwanted or harmful reactions that occur after administration of a medication.
  • Target Joint: A joint that has experienced repeated bleeding episodes, often leading to chronic damage.

References

  1. http://clinicaltrials.eu/trial/study-on-emicizumab-and-drug-combination-for-patients-with-haemophilia-a-and-fviii-inhibitors/