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Giroctocogene Fitelparvovec

Giroctocogene fitelparvovec is an innovative gene therapy being studied in clinical trials for the treatment of moderately severe to severe hemophilia A. This article discusses the ongoing research, potential benefits, and important considerations for patients participating in these trials. The focus is on a Phase 3 clinical study evaluating the efficacy and safety of this promising treatment approach for adult men with hemophilia A.

Table of Contents

What is GIROCTOCOGENE FITELPARVOVEC?

GIROCTOCOGENE FITELPARVOVEC is an innovative gene therapy being developed to treat hemophilia A, a rare bleeding disorder. This medication is also known by other names, including PF-07055480 and SB-525[1]. It belongs to a class of treatments called Advanced Therapy Investigational Medicinal Products (ATIMPs), which use cutting-edge genetic techniques to address diseases[1].

How Does It Work?

GIROCTOCOGENE FITELPARVOVEC works by using a modified virus to deliver a functioning copy of the Factor VIII gene to the patient’s cells. Specifically, it consists of:

  • A recombinant adeno-associated virus serotype 6 (rAAV6) capsid: This is the outer shell of the virus that has been modified to safely deliver genetic material.
  • A single-stranded DNA genome: This contains a specially designed version of the Factor VIII gene.
  • A codon-optimized, B-domain-deleted human Factor VIII transgene: This is the functional gene that will help produce the missing clotting factor in hemophilia A patients[1].

The therapy is administered as a single infusion, with the goal of providing long-lasting production of Factor VIII in the patient’s body[1].

What Condition Does It Treat?

GIROCTOCOGENE FITELPARVOVEC is designed to treat moderately severe to severe hemophilia A. Hemophilia A is a genetic disorder characterized by a deficiency in Factor VIII, a crucial protein for blood clotting. People with this condition experience prolonged bleeding after injuries and may have spontaneous bleeding episodes, especially in joints and muscles[1].

Clinical Trial Details

A Phase 3 clinical trial (study number C3731003) is currently underway to evaluate the efficacy and safety of GIROCTOCOGENE FITELPARVOVEC. Key details of this trial include:

  • It’s an open-label, single-arm study, meaning all participants receive the treatment and know they are receiving it.
  • The study aims to assess the therapy in adult male participants (18 to 65 years old) with moderately severe to severe hemophilia A (Factor VIII activity ≤1%).
  • Participants will receive a single infusion of the gene therapy.
  • The study will follow participants for up to 5 years after the infusion to assess long-term effects[1].

Eligibility Criteria

To participate in the clinical trial, patients must meet certain criteria. Some key inclusion criteria are:

  • Male participants who have been on routine Factor VIII prophylaxis therapy
  • At least 150 documented exposure days to a Factor VIII product
  • Moderately severe to severe hemophilia A (Factor VIII activity ≤1%)
  • Willingness to suspend Factor VIII prophylaxis therapy after receiving the study drug[1]

Some exclusion criteria include:

  • Presence of antibodies against the AAV6 virus used in the therapy
  • History of inhibitors to Factor VIII
  • Significant liver disease or active hepatitis B or C
  • HIV infection with low CD4+ cell count or high viral load[1]

Potential Benefits

The clinical trial aims to demonstrate several potential benefits of GIROCTOCOGENE FITELPARVOVEC, including:

  • Reduced bleeding episodes: The primary goal is to lower the annualized bleeding rate (ABR) compared to previous Factor VIII prophylaxis.
  • Increased Factor VIII activity: The therapy aims to achieve Factor VIII activity levels above 5% at 15 months post-infusion.
  • Reduced need for Factor VIII infusions: Researchers hope to see a significant decrease in the use of exogenous Factor VIII.
  • Improved quality of life: The study will assess changes in joint health and patient-reported outcomes related to quality of life[1].

Safety Considerations

As with any new therapy, safety is a crucial aspect of the clinical trial. The study will closely monitor:

  • Adverse events and their severity
  • Events of special interest, such as hypersensitivity reactions or thrombotic events
  • Immune responses to the therapy, including antibodies against the AAV6 virus and T-cell responses
  • Development of inhibitors to Factor VIII[1]

It’s important to note that while GIROCTOCOGENE FITELPARVOVEC shows promise, it is still an investigational therapy. Patients interested in this treatment should discuss it with their healthcare providers and consider participating in clinical trials if eligible.

Aspect Details
Study Type Phase 3, Open-Label, Single-Arm
Participants Adult males (18-65 years) with moderately severe to severe hemophilia A
Treatment Single infusion of giroctocogene fitelparvovec (PF-07055480)
Primary Endpoint Total annualized bleeding rate (ABR) from Week 12 through 15 months post-infusion
Key Secondary Endpoints Factor VIII activity levels, exogenous Factor VIII use, quality of life measures
Safety Assessments Adverse events, immunogenicity, long-term safety up to 5 years
Exclusion Criteria History of Factor VIII inhibitors, significant liver disease, active hepatitis B or C
Study Duration 5 years post-infusion

FAQ

  • What is giroctocogene fitelparvovec? Giroctocogene fitelparvovec is a gene therapy that uses a modified virus (AAV6) to deliver a functional Factor VIII gene to patients with hemophilia A. It aims to help the body produce its own Factor VIII protein, which is essential for blood clotting.
  • Who is eligible for the clinical trial? The trial is open to adult males (18-65 years old) with moderately severe to severe hemophilia A (Factor VIII activity ≤1%) who have been on routine Factor VIII prophylaxis therapy and have at least 150 documented exposure days to a Factor VIII product.
  • What is the main goal of the clinical trial? The primary objective is to evaluate the efficacy of a single infusion of giroctocogene fitelparvovec in reducing the annualized bleeding rate (ABR) compared to the participants' previous Factor VIII prophylaxis regimen.
  • How long does the clinical trial last? The study duration is 5 years after the infusion of giroctocogene fitelparvovec. This allows researchers to assess the long-term efficacy and safety of the treatment.
  • What are some of the key outcomes being measured in the trial? The trial is measuring several outcomes, including Factor VIII activity levels, reduction in exogenous Factor VIII use, changes in bleeding events, quality of life improvements, and safety parameters such as adverse events and immune responses to the treatment.

Ongoing Clinical Trials on Giroctocogene Fitelparvovec

  • 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

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

  • Study on the Safety and Effectiveness of Giroctocogene Fitelparvovec for Adult Men with Moderate to Severe Hemophilia A

    Not recruiting

    1 1 1
    Investigated drugs:
    France Germany Greece Spain Sweden

Glossary

  • Hemophilia A: A genetic bleeding disorder caused by a deficiency in blood clotting Factor VIII, leading to prolonged bleeding and easy bruising.
  • Factor VIII (FVIII): A protein essential for blood clotting. In hemophilia A, this protein is missing or doesn't work properly.
  • Gene therapy: A treatment approach that involves introducing genetic material into a person's cells to compensate for abnormal genes or to make a beneficial protein.
  • AAV6: Adeno-associated virus serotype 6, a type of virus used as a vector to deliver the therapeutic gene in this treatment.
  • Annualized Bleeding Rate (ABR): The number of bleeding episodes a person experiences in a year, used to measure the effectiveness of hemophilia treatments.
  • Prophylaxis: Regular treatment to prevent bleeding episodes in people with hemophilia.
  • Exogenous Factor VIII: Factor VIII protein given as a medication, as opposed to the body producing it naturally.
  • Immunogenicity: The ability of a substance to provoke an immune response in the body.
  • Exposure days: The number of days a person with hemophilia has received factor replacement therapy.
  • Patient-reported outcomes (PROs): Health outcome measures reported directly by patients, such as quality of life or symptom severity.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-giroctocogene-fitelparvovec-for-adult-men-with-moderate-to-severe-hemophilia-a/