Not recruitingRare disease

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

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying hemophilia A, a genetic disorder that affects the blood's ability to clot properly, leading to excessive bleeding. The treatment being tested is called giroctocogene fitelparvovec, also known by its code name PF-07055480. This treatment is a type of gene therapy, which involves using a specially designed virus to deliver a healthy copy of the gene responsible for producing Factor VIII, a protein that helps blood clot. The goal of the study is to evaluate how effective and safe this gene therapy is for adult males with moderately severe to severe hemophilia A.

Participants in the study will receive a single infusion of the gene therapy. After the infusion, they will be monitored over a period of time to see how their condition changes. The study will look at various factors, such as the rate of bleeding episodes and the level of Factor VIII in the blood, to determine the treatment's effectiveness. The study will also assess any side effects or adverse reactions to the treatment.

The trial is designed to provide valuable information about the potential benefits and risks of using gene therapy to treat hemophilia A. By participating in this study, researchers hope to find a new way to manage this condition and improve the quality of life for those affected by it. The study will continue for several years to gather comprehensive data on the long-term effects of the treatment.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial infusion
Receive a single infusion of PF-07055480, a gene therapy solution administered through an intravenous line. This is a one-time treatment designed to introduce a functional copy of the gene responsible for producing Factor VIII, which is deficient in individuals with hemophilia A.
Step 2
Post-infusion monitoring
Undergo regular monitoring to assess the effectiveness and safety of the treatment. This includes measuring the Factor VIII activity level and tracking any bleeding episodes.
Monitoring will continue from Week 12 through at least 15 months after the infusion. The goal is to evaluate changes in bleeding rates and Factor VIII levels compared to previous treatments.
Step 3
Evaluation of bleeding rates
The annualized bleeding rate (ABR) will be assessed, including both spontaneous and traumatic bleeding events. This will be compared to the bleeding rates experienced prior to the infusion.
The evaluation will focus on the total number of bleeding events, their causes, and locations, such as joints or soft tissue.
Step 4
Assessment of joint health
Joint health will be evaluated using the Hemophilia Joint Health Score (HJHS) to determine any changes from baseline.
This assessment aims to identify improvements or changes in joint condition following the gene therapy infusion.
Step 5
Quality of life and activity assessment
Complete questionnaires to assess changes in quality of life and daily activities. These include the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) and the Haemophilia Activities List (HAL).
These assessments will help determine the impact of the treatment on daily living and overall well-being.
Step 6
Long-term follow-up
Participate in long-term follow-up visits to monitor the sustained effects of the gene therapy. This includes ongoing evaluation of Factor VIII levels, bleeding rates, and any potential adverse events.
The study is expected to continue until October 2028, providing comprehensive data on the long-term efficacy and safety of the treatment.

Who can join the trial?

6 criteria

Participants must be males.
Participants should be between 18 and 65 years old.
Participants must have been part of a previous study where they received regular treatment with a product called Factor VIII, which helps with blood clotting.
Participants need to have had at least 150 days of using the Factor VIII product.
Participants must have a condition called moderately severe to severe hemophilia A, which means their Factor VIII activity is very low (1% or less).
Participants must stop their regular Factor VIII treatment after receiving the study drug.

Who cannot join the trial?

4 criteria

Participants who are not male cannot join the study.
Participants who are younger than 18 years old or older than 64 years old cannot join the study.
Participants who do not have **hemophilia A** cannot join the study. Hemophilia A is a condition where the blood does not clot properly, leading to excessive bleeding.
Participants with a **Factor VIII (FVIII) level** greater than 1% cannot join the study. Factor VIII is a protein that helps blood to clot, and a level greater than 1% means the condition is not severe enough for this study.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Hippokration Hospital
Athens, Greece
Verified
Hopital Necker Enfants Malades
Paris, France
Responsive
Vivantes Netzwerk fuer Gesundheit GmbH
Berlin, Germany

Trial locations

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Investigated drugs

PF-07055480 is a gene therapy being studied for its effectiveness and safety in treating adult males with moderately severe to severe hemophilia A. This therapy involves a single infusion designed to deliver a functional copy of the human Factor VIII gene using a modified virus. The goal is to help the body produce its own Factor VIII, which is a protein necessary for blood clotting, potentially reducing or eliminating the need for regular Factor VIII replacement therapy.

Giroctocogene Fitelparvovec

What is already known about the treatment

PF-07055480 – This medication is administered as a single infusion and is currently being studied in clinical trials for its effectiveness in treating hemophilia A, a condition characterized by a deficiency in clotting factor VIII. The medication is a gene therapy that uses a recombinant AAV2/6 vector to deliver a functional copy of the human factor VIII gene to the patient's cells, aiming to restore normal clotting function. It is classified as a gene therapy product and represents a novel approach in the treatment of hemophilia A, with ongoing research to establish its safety and efficacy.

Investigated diseases

Hemophilia A – Hemophilia A is a genetic disorder characterized by a deficiency in clotting factor VIII, which is essential for blood clotting. This condition leads to prolonged bleeding after injuries, surgeries, or even spontaneously without any apparent cause. Individuals with hemophilia A may experience frequent bleeding episodes, particularly into joints and muscles, which can cause pain and swelling. Over time, repeated bleeding into joints can lead to joint damage and reduced mobility. The severity of the condition varies, with some individuals experiencing mild symptoms and others having more severe bleeding episodes. Hemophilia A is typically diagnosed in childhood, but symptoms can appear at any age.