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Fidanacogene Elaparvovec

Fidanacogene Elaparvovec is an innovative gene therapy being studied in clinical trials for the treatment of moderate to severe hemophilia B. This therapy aims to provide a long-lasting solution for patients by introducing a modified gene that helps produce the clotting factor IX, which is deficient in people with hemophilia B. The ongoing clinical trials are evaluating the safety and effectiveness of this potential breakthrough treatment.

Table of Contents

What is Fidanacogene Elaparvovec?

Fidanacogene elaparvovec, also known by its product code PF-06838435, is an innovative gene therapy being developed to treat moderate to severe hemophilia B[1]. This therapy is classified as an advanced therapy medicinal product, specifically a gene transfer product[1].

How Does It Work?

Fidanacogene elaparvovec uses a specially designed virus (called a viral vector) to deliver a functional copy of the Factor IX (FIX) gene to patients’ cells. This gene has been modified to produce a highly active variant of FIX known as Padua[1]. The therapy is administered as a single intravenous infusion, with the goal of providing long-term production of the FIX protein in the patient’s body[1].

Target Condition: Hemophilia B

Hemophilia B is a rare genetic bleeding disorder caused by a deficiency in Factor IX, a crucial protein for blood clotting. Patients with moderate to severe hemophilia B (defined as having 2% or less of normal FIX activity) experience frequent spontaneous bleeding episodes, which can lead to joint damage and other serious complications[1].

Clinical Trial Details

A Phase 3 clinical trial, known as BENEGENE-2, is currently evaluating the efficacy and safety of fidanacogene elaparvovec[1]. Key aspects of the trial include:

  • It’s an open-label, single-arm study, meaning all participants receive the therapy.
  • The main goal is to demonstrate the effectiveness of a single infusion in adult males with moderate to severe hemophilia B.
  • The study will last for 6 years after the infusion to assess long-term effects and durability of the treatment.

Eligibility Criteria

The trial has specific criteria for participants, including[1]:

  • Male patients aged 18-65 years
  • Diagnosed with moderate to severe hemophilia B (FIX activity ≤2%)
  • Previous experience with FIX therapy (at least 50 exposure days)
  • No history of FIX inhibitors
  • Acceptable liver function and no significant liver disease

Potential Benefits

The potential benefits being investigated include[1]:

  • Reduced bleeding episodes
  • Decreased need for regular FIX replacement therapy
  • Improved quality of life
  • Better joint health
  • Long-lasting effects from a single treatment

Safety Considerations

As with any new therapy, safety is a crucial aspect being monitored in the trial. Some key safety considerations include[1]:

  • Potential immune responses to the therapy
  • Liver function changes
  • Risk of developing inhibitors to FIX
  • Possibility of thrombotic events (blood clots)

Future Prospects

If successful, fidanacogene elaparvovec could represent a significant advancement in the treatment of hemophilia B. It has the potential to provide long-term relief from symptoms with a single treatment, potentially transforming the lives of patients who currently require regular infusions of FIX[1].

As the clinical trial progresses, more information will become available about the effectiveness and safety of this promising gene therapy. Patients with hemophilia B and their healthcare providers should stay informed about the latest developments in this field.

Aspect Details
Study Type Phase 3, open-label, single-arm study
Participants Adult males (18-65 years) with moderate to severe hemophilia B (FIX:C ≤2%)
Intervention Single infusion of Fidanacogene Elaparvovec (PF-06838435)
Primary Endpoint Non-inferiority on annualized bleeding rate (ABR) from Week 12 to Month 15 compared to usual care
Secondary Endpoints Factor IX activity levels, annualized infusion rate, quality of life measures, safety assessments
Follow-up Duration Up to 6 years post-infusion
Key Exclusion Criteria History of FIX inhibitors, significant liver disease, active hepatitis B or C, planned surgeries

FAQ

  • What is Fidanacogene Elaparvovec? Fidanacogene Elaparvovec is a gene therapy designed to treat hemophilia B. It uses a modified virus to deliver a functional copy of the factor IX gene to the patient's liver cells, potentially allowing them to produce the clotting factor they lack.
  • Who is eligible for the clinical trial? The trial is open to adult males (18-65 years old) with moderate to severe hemophilia B, defined as having factor IX activity levels of 2% or less. Participants must have previous experience with factor IX therapy and agree to certain study requirements.
  • How is the therapy administered? Fidanacogene Elaparvovec is given as a single intravenous infusion. After receiving the therapy, participants are asked to stop their regular factor IX prophylaxis treatment.
  • What are the main goals of the clinical trial? The primary goal is to demonstrate the effectiveness of Fidanacogene Elaparvovec in reducing bleeding rates compared to regular factor IX prophylaxis. Secondary goals include assessing the therapy's impact on factor IX levels, quality of life, and long-term safety.
  • How long does the clinical trial last? The study follows participants for up to 6 years after receiving the gene therapy to assess its long-term effectiveness and safety.

Ongoing Clinical Trials on Fidanacogene Elaparvovec

  • A study on the long-term safety and effectiveness of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B

    Not yet recruiting

    3 1 1
    Investigated drugs:
    France Germany Greece Italy Spain Sweden

  • Study on the Efficacy and Safety of Fidanacogene Elaparvovec Gene Therapy in Adult Males with Moderate to Severe Hemophilia B

    Not recruiting

    3 1 1
    Investigated drugs:
    Belgium France Germany Greece Italy Spain +1

Glossary

  • Hemophilia B: A genetic bleeding disorder caused by a deficiency in clotting factor IX, which can lead to prolonged bleeding after injuries or spontaneous bleeding in severe cases.
  • Factor IX (FIX): A protein in the blood that is essential for proper blood clotting. People with hemophilia B have low levels or absent factor IX.
  • Gene therapy: A treatment approach that involves introducing genetic material into a person's cells to treat or prevent disease.
  • Prophylaxis: Regular treatment given to prevent bleeding episodes in people with hemophilia.
  • Annualized Bleeding Rate (ABR): A measure used to assess the effectiveness of hemophilia treatments by calculating the number of bleeding episodes a patient experiences in a year.
  • Vector: In gene therapy, a vector is a vehicle (often a modified virus) used to deliver genetic material into cells.
  • Transgene: A gene that is transferred from one organism to another, in this case, the modified factor IX gene introduced by the therapy.
  • Infusion: The process of administering a substance directly into the bloodstream through a vein.

References

  1. http://clinicaltrials.eu/trial/study-on-the-efficacy-and-safety-of-fidanacogene-elaparvovec-gene-therapy-in-adult-males-with-moderate-to-severe-hemophilia-b/