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Efmoroctocog Alfa

Efmoroctocog Alfa, also known as Eloctate, is an extended half-life factor VIII product being studied in clinical trials for the treatment of hemophilia A. These trials aim to evaluate its effectiveness in preventing and treating bleeding episodes, improving quality of life, and addressing challenges like inhibitor formation in patients with this rare blood clotting disorder.

Table of Contents

What is Efmoroctocog Alfa?

Efmoroctocog alfa, also known by its brand names Eloctate or ELOCTATE, is a medication used to treat hemophilia A[1]. It belongs to a class of drugs called extended half-life factor VIII products. This means that the medication stays active in your body for a longer time compared to standard factor VIII products[2]. Efmoroctocog alfa is a recombinant factor VIII Fc fusion protein, which is a scientific way of saying it’s a laboratory-created version of the clotting factor that people with hemophilia A lack[3].

How Does It Work?

In people with hemophilia A, the blood doesn’t clot properly because they don’t have enough of a protein called factor VIII. Efmoroctocog alfa works by replacing this missing factor VIII in the body. This helps the blood to clot normally, which can prevent or stop bleeding episodes[1]. The “extended half-life” feature of efmoroctocog alfa means that it remains active in the body for a longer time. This can lead to fewer injections for patients compared to standard factor VIII products[2].

What Conditions Does It Treat?

Efmoroctocog alfa is primarily used to treat hemophilia A, also known as classic hemophilia. It can be used in several ways:

  • Prophylaxis (prevention): Regular use to prevent bleeding episodes[1]
  • On-demand treatment: To control bleeding when it occurs[1]
  • Perioperative management: To prevent excessive bleeding during and after surgery[1]

Additionally, research is being conducted to explore its use in other scenarios, such as:

  • Preventing the formation of inhibitors in people with severe hemophilia A who haven’t been treated before[5]
  • Eradicating inhibitors in people who have developed them[6]
  • Reducing heavy menstrual bleeding in women who carry the hemophilia A gene[7]

How is It Administered?

Efmoroctocog alfa is given as an intravenous injection, which means it’s injected directly into a vein. The dose and frequency of injections can vary depending on several factors, including the severity of your hemophilia, your body weight, and the purpose of treatment (prevention or on-demand)[1].

For long-term prevention of bleeding (prophylaxis), a common starting dose is 50 IU (International Units) per kilogram of body weight, given every 3 to 5 days. However, your doctor may adjust this dose based on your individual response, potentially ranging from 25 to 65 IU/kg. In some cases, especially for younger patients, shorter intervals between doses or higher doses may be necessary[1].

Current Clinical Trials

Several clinical trials are currently underway to further investigate the benefits and potential new uses of efmoroctocog alfa:

  • Quality of Life Study: A study is examining how switching from standard half-life factor VIII products to efmoroctocog alfa affects the quality of life in young people with hemophilia A in Russia[1].
  • Effectiveness Comparison: Another study is comparing the effectiveness of efmoroctocog alfa to conventional factor products in preventing bleeding episodes[2].
  • Inhibitor Prevention: A trial is investigating whether efmoroctocog alfa is better than another drug called emicizumab at preventing the formation of inhibitors in children with severe hemophilia A who haven’t been treated before[5].
  • Inhibitor Eradication: Researchers are also studying whether using efmoroctocog alfa alone or in combination with emicizumab is more effective at eliminating inhibitors in people who have developed them[6].
  • Menstrual Bleeding in Carriers: A study is exploring whether efmoroctocog alfa can help reduce heavy menstrual bleeding in women who carry the hemophilia A gene[7].

Impact on Quality of Life

One of the key areas being studied is how efmoroctocog alfa affects the quality of life of people with hemophilia A. Researchers are using various questionnaires and assessments to measure this, including:

  • Haemophilia-specific Quality of Life Questionnaires: These are tailored to capture the unique challenges faced by people with hemophilia[1].
  • Bleeding Rates: The number and severity of bleeding episodes can significantly impact quality of life[1].
  • Joint Health: Researchers are monitoring joint health using tools like the Hemophilia Joint Health Score, as joint damage from repeated bleeds is a major concern for people with hemophilia[1].
  • Physical Activity: Some studies are looking at how treatment with efmoroctocog alfa affects patients’ ability to engage in physical activities[4].

Safety Considerations

While efmoroctocog alfa is generally well-tolerated, there are some important safety considerations:

  • Inhibitor Development: Some people may develop antibodies (called inhibitors) against factor VIII products, including efmoroctocog alfa. This can make the treatment less effective. Your doctor will monitor you for this[5].
  • Allergic Reactions: As with any biological product, there’s a potential for allergic reactions, including severe ones like anaphylaxis. It’s important to be aware of the signs of an allergic reaction and seek immediate medical attention if they occur[7].
  • Regular Monitoring: Your doctor will likely want to monitor your factor VIII levels regularly to ensure the treatment is working effectively[1].

Remember, while this information provides a general overview, your healthcare provider is the best source of information about whether efmoroctocog alfa is right for you, how it should be used, and what to expect from treatment.

Aspect Details
Drug Name Efmoroctocog Alfa (Eloctate)
Type Extended half-life recombinant factor VIII product
Condition Treated Hemophilia A
Administration Intravenous infusion
Key Clinical Trials NCT04728217, NCT02976753, NCT02196207, NCT04303572, NCT04690322, NCT04303559, NCT03272568
Primary Outcomes Studied Quality of life, annualized bleeding rate, factor consumption, inhibitor formation/eradication, joint health
Comparators in Trials Standard half-life factor VIII products, Emicizumab
Special Populations Previously untreated patients, patients with inhibitors, hemophilia carriers with heavy menstrual bleeding

FAQ

  • What is Efmoroctocog Alfa? Efmoroctocog Alfa, also known as Eloctate, is an extended half-life factor VIII product used to treat hemophilia A. It's designed to stay in the body longer than standard factor VIII products, potentially requiring less frequent dosing.
  • How is Efmoroctocog Alfa being studied in clinical trials? Efmoroctocog Alfa is being studied in various trials to assess its effectiveness in preventing bleeding episodes, improving quality of life, comparing it to other treatments like Emicizumab, and evaluating its potential in preventing or eradicating inhibitor formation in hemophilia A patients.
  • What are some potential benefits of Efmoroctocog Alfa? Potential benefits include longer-lasting protection against bleeding episodes, improved quality of life due to less frequent dosing, and possible effectiveness in preventing or treating inhibitor formation in hemophilia A patients.
  • Are there any side effects associated with Efmoroctocog Alfa? As with any medication, there can be side effects. In clinical trials, researchers monitor for potential hypersensitivity reactions, including anaphylaxis. Patients and caregivers are trained to recognize signs of these reactions and seek immediate care if they occur.
  • How is Efmoroctocog Alfa administered? Efmoroctocog Alfa is typically administered through intravenous infusion. The dosing schedule and amount can vary depending on the specific trial and the patient's needs, but it's generally given less frequently than standard factor VIII products due to its extended half-life.

Ongoing Clinical Trials on Efmoroctocog Alfa

  • Study on the Safety and Effectiveness of Emicizumab for Patients with Type 3 Von Willebrand Disease

    Recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium France Germany Italy The Netherlands Poland +2

  • Study on Joint Health in Hemophilia A and B Patients Using Efmoroctocog Alfa or Eftrenonacog Alfa

    Not yet recruiting

    3 1 1 1
    Investigated drugs:
    Bulgaria Croatia Czechia France Hungary Ireland +4

Glossary

  • Hemophilia A: A genetic disorder caused by missing or defective factor VIII, a clotting protein. People with hemophilia A can experience prolonged bleeding after injuries, surgeries, or spontaneously.
  • Factor VIII: A protein in the blood that helps with clotting. In hemophilia A, this protein is missing or doesn't work properly.
  • Extended Half-Life (EHL): Refers to clotting factor products designed to stay in the body longer than standard products, potentially allowing for less frequent dosing.
  • Prophylaxis: Regular treatment to prevent bleeding episodes in people with hemophilia.
  • Inhibitors: Antibodies that the body produces against factor VIII, making treatment less effective or ineffective.
  • Immune Tolerance Induction (ITI): A treatment approach to eliminate inhibitors in hemophilia patients.
  • Annualized Bleeding Rate (ABR): The number of bleeding episodes a person with hemophilia experiences in a year.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Quality of Life (QoL): A measure of an individual's overall well-being and ability to function in daily life.
  • Recombinant Factor: Clotting factors produced in a laboratory using genetic engineering techniques, rather than derived from human blood.

References

  1. https://clinicaltrials.gov/study/NCT04728217
  2. https://clinicaltrials.gov/study/NCT02976753
  3. https://clinicaltrials.gov/study/NCT02196207
  4. https://clinicaltrials.gov/study/NCT04690322
  5. https://clinicaltrials.gov/study/NCT04303559
  6. https://clinicaltrials.gov/study/NCT04303572
  7. https://clinicaltrials.gov/study/NCT03272568