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Eftrenonacog Alfa

This article discusses an 18-month clinical trial investigating the effectiveness of Eftrenonacog Alfa (ALPROLIX) in improving joint health for patients with hemophilia A and B. The study aims to assess joint outcomes using ultrasound technology, clinical evaluations, and patient-reported measures. This low-intervention trial provides valuable insights into the long-term effects of ALPROLIX on joint health and quality of life for individuals with hemophilia.

Table of Contents

What is Eftrenonacog Alfa?

Eftrenonacog alfa is a medication used to treat hemophilia B, a rare bleeding disorder. It is also known by several other names, including:

  • Recombinant fusion protein consisting of human coagulation factor IX attached to the Fc domain of human IgG1
  • BIIB-029
  • rFIXFc
  • Coagulation factor IX (recombinant), Fc fusion protein
The brand name for this medication is Alprolix[1].

Medical Conditions Treated

Eftrenonacog alfa is specifically designed to treat hemophilia B, also known as factor IX deficiency. Hemophilia B is a genetic disorder that affects the blood’s ability to clot properly. People with this condition lack sufficient levels of clotting factor IX, which can lead to prolonged bleeding after injuries, surgeries, or even spontaneously[1].

How It Works

Eftrenonacog alfa works by replacing the missing or deficient factor IX in patients with hemophilia B. It is a recombinant fusion protein, which means it’s artificially created in a laboratory. The medication combines factor IX with a part of an antibody (the Fc domain of human IgG1). This fusion allows the medication to last longer in the body, potentially reducing the frequency of injections needed[1].

Administration and Dosage

Eftrenonacog alfa is administered as an injection. It comes in various strengths, including 250 IU, 500 IU, 1000 IU, 2000 IU, and 3000 IU, all provided as a powder and solvent for solution for injection[1]. The specific dosage depends on the patient’s condition, weight, and doctor’s recommendation. The maximum daily dose is typically 100 IU/kg (international units per kilogram of body weight)[1].

Clinical Study Overview

A clinical study called JOIN-us is currently underway to assess the effectiveness of eftrenonacog alfa in treating hemophilia B. This 18-month study aims to evaluate joint health in patients using this medication for prophylaxis (preventive treatment)[1].

The main objective of the study is to assess overall joint status using ultrasound in patients treated with eftrenonacog alfa over the 18-month period. Secondary objectives include evaluating specific aspects of joint health, such as:

  • Synovium (joint lining) thickness
  • Cartilage condition
  • Bone damage
  • Clinical joint status using a standardized score (Hemophilia Joint Health Score)
  • Presence and development of target joints (joints that bleed frequently)
  • Bleeding episodes
  • Quality of life
[1]

Benefits and Effectiveness

The study aims to measure the effectiveness of eftrenonacog alfa through several key outcomes:

  • Changes in joint health as measured by ultrasound and clinical examination
  • Reduction in the number of bleeding episodes, particularly in joints
  • Improvement in physical function and reduction in pain
  • Overall improvement in quality of life
These outcomes will be assessed at various time points throughout the 18-month study period[1].

Patient Eligibility

To be eligible for treatment with eftrenonacog alfa, patients typically need to meet certain criteria:

  • Diagnosed with hemophilia B
  • At least 6 months of documented pre-study treatment data
  • Previous treatment with any marketed recombinant or plasma-derived factor IX concentrate for at least 6 months
  • No presence of factor IX inhibitors (antibodies that can interfere with treatment)
  • Not currently enrolled in other interventional clinical studies
It’s important to note that the final decision on eligibility should always be made by a healthcare professional[1].

Quality of Life Impact

The JOIN-us study also aims to evaluate the impact of eftrenonacog alfa on patients’ quality of life. This includes assessing:

  • Physical function and activity levels
  • Pain intensity and interference with daily life
  • Overall health-related quality of life
These aspects are measured using standardized questionnaires at the beginning and end of the study period[1].

Aspect Details
Study Type 18-month low-intervention prospective, multicentre study
Participants Patients with hemophilia A or B on prophylaxis with Eftrenonacog Alfa (ALPROLIX)
Primary Objective Evaluate overall joint status using ultrasound (US)
Secondary Objectives Assess specific joint changes, clinical joint status, target joints, bleeding episodes, quality of life
Assessment Methods HEAD-US, HJHS, bleeding episode tracking, PROMIS, IPAQ-SF
Drug Information ALPROLIX (Eftrenonacog Alfa), available in 250, 500, 1000, 2000, and 3000 IU strengths
Administration Injection, maximum daily dose 100 IU/kg
Study Duration 18 months with assessments at baseline, 6, 12, and 18 months

FAQ

  • What is the main goal of this clinical trial? The main goal is to evaluate overall joint status in hemophilia A and B patients treated with Eftrenonacog Alfa (ALPROLIX) over an 18-month period using ultrasound technology.
  • Who can participate in this clinical trial? Participants must have a diagnosis of hemophilia A or B, at least 6 months of documented pre-study treatment data, and be starting or already on prophylactic treatment with ALPROLIX.
  • What methods are used to assess joint health in this study? The study uses ultrasound imaging (HEAD-US), clinical evaluations (Hemophilia Joint Health Score), and patient-reported outcomes to assess joint health comprehensively.
  • How long does the clinical trial last? The clinical trial lasts for 18 months, with assessments conducted at baseline, 6 months, 12 months, and 18 months.
  • What aspects of patient well-being are measured in this study? The study measures joint health, bleeding episodes, quality of life, physical function, pain intensity and interference, and physical activity levels using various assessment tools.

Ongoing Clinical Trials on Eftrenonacog Alfa

  • 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

  • Eftrenonacog Alfa: A recombinant fusion protein consisting of human coagulation factor IX attached to the Fc domain of human IgG1, used to treat hemophilia B.
  • Hemophilia: A genetic disorder that impairs the body's ability to form blood clots, leading to prolonged bleeding and easy bruising.
  • Prophylaxis: Regular treatment to prevent bleeding episodes in people with hemophilia.
  • HEAD-US: Haemophilia Early Arthropathy Detection with Ultrasound, a method used to detect early joint changes in hemophilia patients.
  • HJHS: Hemophilia Joint Health Score, a clinical assessment tool used to evaluate joint health in hemophilia patients.
  • Target joint: A joint that has had repeated bleeding episodes, often resulting in chronic damage.
  • Annualized bleeding rate (ABR): The number of bleeding episodes a patient experiences over a year.
  • PROMIS: Patient-Reported Outcomes Measurement Information System, a set of measures used to assess physical, mental, and social health.
  • IPAQ-SF: International Physical Activity Questionnaire – Short Form, used to assess physical activity levels.
  • Recombinant factor: A clotting factor protein produced through genetic engineering, rather than derived from human blood.

References

  1. http://clinicaltrials.eu/trial/study-on-joint-health-in-hemophilia-a-and-b-patients-using-efmoroctocog-alfa-or-eftrenonacog-alfa/