Not recruitingRare disease

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

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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 a condition known as hemophilia B, which is a genetic disorder that affects the blood's ability to clot properly. People with this condition can experience excessive bleeding even from minor injuries. The study is specifically looking at adult males with moderately severe to severe forms of this condition. The treatment being tested is a gene therapy called fidanacogene elaparvovec, also known by its code name PF-06838435. This therapy involves a single infusion of a solution that contains a modified gene designed to help the body produce more of a protein called Factor IX, which is essential for blood clotting.

The purpose of the study is to evaluate how effective and safe this gene therapy is for individuals with hemophilia B. Participants in the study will receive a single infusion of the gene therapy and will be monitored over a period of time to see how their condition changes. The study will look at various factors, such as the number of bleeding episodes participants experience and their overall health, to determine the therapy's effectiveness. Participants will also be observed for any side effects or adverse reactions to the treatment.

The study is designed to provide valuable information about the potential benefits and risks of using fidanacogene elaparvovec as a treatment for hemophilia B. By participating in this research, scientists hope to find a new way to help people with this condition manage their symptoms and improve their quality of life. The study will continue for several years to gather comprehensive data on the long-term effects of the therapy.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Screening and consent
Upon joining the study, participants will undergo a screening process to ensure eligibility. This includes reviewing medical history and conducting laboratory tests to confirm the diagnosis of moderately severe to severe hemophilia B.
Participants must provide informed consent, agreeing to the study's requirements and restrictions.
Step 2
Pre-infusion preparation
Participants will continue their usual FIX prophylaxis replacement therapy for at least 6 months prior to the infusion. This period is used to collect baseline data.
Participants must agree to stop prophylaxis therapy after receiving the study drug, although FIX replacement therapy is allowed as needed.
Step 3
Infusion visit
Participants will receive a single intravenous infusion of the study drug, fidanacogene elaparvovec, which is a solution for infusion.
Participants must refrain from donating sperm and agree to use contraception or abstain from sexual activity until certain conditions are met.
Step 4
Post-infusion monitoring
Participants will be monitored for any adverse events and changes in health status. Regular follow-up visits will be scheduled to assess the efficacy and safety of the treatment.
The study will evaluate the annualized bleeding rate and other health parameters over a period of up to 6 years.
Step 5
Long-term follow-up
Participants will continue to be monitored for up to 6 years to assess the long-term effects of the treatment.
Regular assessments will include laboratory tests and health evaluations to ensure the safety and effectiveness of the gene therapy.

Who can join the trial?

11 criteria

Participants must have completed at least 6 months of data collection while receiving **FIX prophylaxis replacement therapy**. This means they have been regularly treated to prevent bleeding episodes.
Participants must have **moderately severe to severe hemophilia B**, which is a condition where the blood does not clot properly. This is defined as having a **FIX:C level of 2% or less**.
Participants must have previous experience with **FIX therapy**, having used a FIX protein product for at least 50 days. FIX therapy involves using a protein to help the blood clot.
Participants must agree to stop their regular preventive treatment for hemophilia B after receiving the study treatment. They can still use FIX replacement therapy if needed.
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Participants must have acceptable laboratory test results, including:
- **Hemoglobin** of at least 11 g/dL. Hemoglobin is a protein in red blood cells that carries oxygen.
- **Platelets** of at least 100,000 cells/µL. Platelets are cells that help the blood clot.
- **Creatinine** of 2.0 mg/dL or less. Creatinine is a waste product measured to check kidney function.
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  Participants must be **male** and agree to certain requirements regarding sperm donation and sexual activity to prevent the spread of the study treatment. This includes:
  - Not donating sperm.
  - Either abstaining from sexual activity or using a **male condom** during sexual activity.
  - Participants must be able to give signed informed consent, meaning they understand and agree to the study's requirements and restrictions.
  - Participants must be between the ages of **18 and 65 years**.

Who cannot join the trial?

8 criteria

Participants cannot have any other serious medical conditions that might interfere with the study.
Participants should not have a history of allergic reactions to the study medication or similar treatments.
Participants must not have received any other experimental treatments for hemophilia B within a certain period before the study.
Participants should not have any bleeding disorders other than hemophilia B.
Participants must not have any active infections that require treatment.
Participants should not have a history of drug or alcohol abuse that could affect their ability to participate in the study.
Participants must not have any conditions that could affect their ability to understand or comply with the study requirements.
Participants should not be involved in any other clinical trials at the same time.

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Verified sites

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

Verified
Azienda Ospedaliera Universitaria Federico II Di Napoli
Naples, Italy
Responsive
Cliniques Universitaires Saint-Luc
Woluwe-Saint-Lambert, Belgium
Responsive
Vivantes Netzwerk fuer Gesundheit GmbH
Berlin, Germany

Trial locations

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Investigated drugs

PF-06838435 is a gene therapy treatment being studied for its potential to help people with hemophilia B, a condition where the blood doesn't clot properly due to a lack of a specific protein called Factor IX. This therapy involves a one-time infusion that aims to deliver a working copy of the gene responsible for producing Factor IX. By doing this, the body may be able to produce more of this protein on its own, which could reduce bleeding episodes and improve the quality of life for those affected by this condition.

Fidanacogene Elaparvovec

What is already known about the treatment

PF-06838435 – This medication is administered as a single infusion directly into the bloodstream. It is currently being studied in clinical trials for its effectiveness and safety in treating adult males with moderately severe to severe hemophilia B, a condition where blood does not clot properly due to low levels of a specific clotting factor. The medication works by delivering a modified gene that helps the body produce more of the missing clotting factor, thereby improving blood clotting. It is classified as a gene therapy, which is a type of treatment that aims to correct or replace faulty genes in the body.

Investigated diseases

Hemophilia B – Hemophilia B is a genetic bleeding disorder caused by a deficiency of clotting factor IX. This condition leads to prolonged or spontaneous bleeding, especially into joints and muscles. Individuals with severe forms may experience frequent bleeding episodes, while those with moderate forms may bleed less often. Bleeding can occur after injuries, surgeries, or even without any apparent cause. Over time, repeated bleeding into joints can cause damage and lead to chronic joint problems. The severity of the disease is often classified based on the level of factor IX activity in the blood.