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Damoctocog Alfa Pegol

Damoctocog Alfa Pegol, also known as Jivi, is an extended half-life recombinant Factor VIII product used in the treatment of Hemophilia A. This article explores various clinical trials investigating the long-term safety, effectiveness, and impact on patients’ quality of life when using Jivi for prophylaxis and treatment of bleeding in Hemophilia A patients. These trials aim to provide valuable insights into real-world usage, patient outcomes, and potential benefits of this innovative therapy.

Table of Contents

What is Damoctocog Alfa Pegol?

Damoctocog alfa pegol, also known by its brand name Jivi or BAY94-9027, is a medication used to treat and prevent bleeding in people with hemophilia A. Hemophilia A is a genetic bleeding disorder caused by a lack of a protein in the blood called “clotting factor VIII” (FVIII)[1].

Jivi is an extended half-life (EHL) product, which means it stays in the body longer than standard factor VIII products. This allows for less frequent injections, potentially making treatment more convenient for patients[1].

How Does It Work?

Damoctocog alfa pegol works by replacing the missing or defective factor VIII in people with hemophilia A. It is a recombinant factor VIII product, which means it is made in a laboratory using genetic engineering techniques. The medication is specifically designed to last longer in the body, reducing the frequency of required injections[2].

The “pegol” part of the name refers to a polyethylene glycol (PEG) molecule attached to the factor VIII protein. This PEG molecule helps the medication stay in the body longer, allowing for extended protection against bleeding[2].

Treatment and Prevention of Bleeding

Damoctocog alfa pegol is used for both the treatment of active bleeding episodes and the prevention of bleeding (prophylaxis) in people with hemophilia A. It can be used in patients aged 12 years and older[2].

The medication helps in:

  • Stopping active bleeding episodes
  • Preventing spontaneous bleeding
  • Reducing the frequency of bleeding into joints (joint bleeds)
  • Managing bleeding during surgical procedures

Dosing and Administration

Damoctocog alfa pegol is given as an injection into a vein (intravenous injection). The dosing schedule can be tailored to individual patient needs, which is one of the advantages of this medication[2].

Typical dosing schedules may include:

  • Every 5 days
  • Every 7 days
  • Twice per week

The exact dose and frequency will be determined by your doctor based on your individual needs and response to treatment[3].

Safety and Effectiveness

Several studies are ongoing to evaluate the long-term safety and effectiveness of damoctocog alfa pegol. These studies are looking at various aspects, including:

  • The occurrence of side effects (adverse events)
  • The development of inhibitors (antibodies that can make the treatment less effective)
  • The medication’s ability to prevent bleeding episodes
  • Its impact on joint health

One of the main safety concerns being monitored is the development of factor VIII inhibitors. These are antibodies that the body might produce against the treatment, potentially making it less effective. Regular testing for inhibitors is part of the ongoing safety monitoring[3].

Ongoing Research

Several clinical trials are currently underway to gather more information about damoctocog alfa pegol. These studies aim to:

  • Evaluate the long-term safety of the medication[4]
  • Assess its effectiveness in preventing bleeding episodes[2]
  • Understand how it affects joint health over time[1]
  • Measure its impact on patients’ physical activity levels[5]

These studies will provide valuable information about the long-term use of damoctocog alfa pegol in real-world settings.

Impact on Quality of Life

Researchers are also interested in understanding how damoctocog alfa pegol affects patients’ quality of life. This includes looking at:

  • Patient satisfaction with the treatment
  • Impact on daily activities and work productivity
  • Changes in physical activity levels
  • Overall health-related quality of life

These aspects are being measured using various questionnaires and tools, including the Hemophilia Treatment Satisfaction Questionnaire, the Hemophilia Quality of Life Measure, and devices that track physical activity[5].

By offering less frequent dosing and potentially improved protection against bleeding, damoctocog alfa pegol aims to make life easier for people living with hemophilia A. However, as with any medication, it’s important to discuss the potential benefits and risks with your healthcare provider to determine if it’s the right choice for you.

Aspect Details
Drug Name Damoctocog Alfa Pegol (Jivi, BAY94-9027)
Condition Treated Hemophilia A
Trial Objectives Evaluate long-term safety, efficacy, impact on joint health, physical activity levels, and patient satisfaction
Key Outcome Measures Annualized Bleeding Rate (ABR), HEAD-US score, HJHS, inhibitor development, adverse events
Study Designs Observational studies, post-marketing investigations
Patient Population Previously treated patients with Hemophilia A, including adults and children
Treatment Duration Ranging from 1 to 4 years, depending on the specific trial
Unique Features Extended half-life, potential for less frequent dosing, real-world effectiveness assessment

FAQ

  • What is Damoctocog Alfa Pegol (Jivi)? Damoctocog Alfa Pegol, marketed as Jivi, is an extended half-life recombinant Factor VIII product used to treat and prevent bleeding in patients with Hemophilia A. It is designed to stay longer in the body, potentially reducing the frequency of injections compared to standard Factor VIII products.
  • What are the main objectives of the clinical trials involving Jivi? The clinical trials aim to evaluate the long-term safety of Jivi, its effectiveness in preventing bleeding, its impact on joint health, changes in physical activity levels of patients, and overall patient satisfaction with the treatment. Some trials also focus on real-world usage patterns and outcomes.
  • How is joint health being assessed in these trials? Joint health is being assessed using various methods, including ultrasound examinations, the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) score, and the Hemophilia Joint Health Score (HJHS). These measures help researchers understand how Jivi affects joint status over time.
  • Are there any specific safety concerns being monitored in these trials? Yes, the trials are monitoring several safety aspects, including the development of inhibitors (antibodies that can reduce the effectiveness of the treatment), hypersensitivity reactions, and the potential impact on renal and hepatic function. They are also tracking any adverse events that may occur during treatment.
  • How do these trials measure the effectiveness of Jivi? The effectiveness of Jivi is being measured through various outcomes, including the annualized bleeding rate (ABR), the number of infusions needed to control bleeding, changes in joint health scores, and patient-reported outcomes on quality of life and treatment satisfaction. Some trials are also looking at physical activity levels as a measure of treatment effectiveness.

Ongoing Clinical Trials on Damoctocog Alfa Pegol

  • Safety Study of Damoctocog Alfa Pegol for Children Aged 7 to 11 with Severe Hemophilia A

    Not recruiting

    1 1 1 1
    Investigated drugs:
    Italy Norway

  • Study on Fitusiran for Preventing Bleeding in Males Aged 12 and Older with Severe Hemophilia A or B, with or without Inhibitors

    Not recruiting

    1 1 1 1
    Investigated drugs:
    France Germany Greece Italy Poland Spain

Glossary

  • Hemophilia A: A genetic bleeding disorder caused by a deficiency in clotting Factor VIII, leading to prolonged bleeding and potential joint damage.
  • Factor VIII (FVIII): A protein in the blood that is essential for normal blood clotting. People with Hemophilia A have low levels or dysfunctional FVIII.
  • Prophylaxis: Regular treatment to prevent bleeding episodes in people with hemophilia.
  • Annualized Bleeding Rate (ABR): The number of bleeding episodes a patient experiences over a year, used to measure the effectiveness of hemophilia treatments.
  • HEAD-US score: Hemophilia Early Arthropathy Detection with Ultrasound score, used to assess joint health in hemophilia patients using ultrasound imaging.
  • HJHS: Hemophilia Joint Health Score, a measure used to assess joint health and function in hemophilia patients.
  • Inhibitor: Antibodies that the body may develop against Factor VIII treatment, reducing its effectiveness.
  • Extended Half-Life (EHL): Refers to clotting factor products engineered to remain active in the body for a longer time, potentially allowing for less frequent dosing.
  • PEG: Polyethylene Glycol, a molecule attached to Damoctocog Alfa Pegol to extend its half-life in the body.
  • Recombinant: Refers to proteins produced using genetic engineering techniques, as opposed to being derived from human or animal blood products.

References

  1. https://clinicaltrials.gov/study/NCT05643560
  2. https://clinicaltrials.gov/study/NCT03932201
  3. https://clinicaltrials.gov/study/NCT04085458
  4. https://clinicaltrials.gov/study/NCT04461639
  5. https://clinicaltrials.gov/study/NCT04091386