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Long-Term Safety Study of Valoctocogene Roxaparvovec for Patients with 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 focuses on individuals with Hemophilia A, a genetic disorder that affects the blood's ability to clot properly, leading to excessive bleeding. The study involves a treatment called BMN 270, also known as valoctocogene roxaparvovec, which is a gene therapy designed to help the body produce more of a protein called Factor VIII, essential for blood clotting. This treatment is administered as a solution for infusion, meaning it is given directly into the bloodstream through a vein.

The purpose of this study is to evaluate the long-term safety of BMN 270 in participants who have previously received this treatment in earlier clinical trials. Participants will be monitored over an extended period to observe any potential side effects or health changes. The study will look for specific events such as liver problems, blood clots, the development of inhibitors to Factor VIII, and any transmission of the treatment to others. Additionally, the study will assess changes in bleeding rates, Factor VIII activity levels, and the use of other medications for managing Hemophilia A.

Participants will also provide feedback on their overall health and quality of life throughout the study. This feedback will help researchers understand the impact of BMN 270 on daily living and any changes in the severity of symptoms. The study aims to gather comprehensive data to ensure the treatment's safety and effectiveness for individuals with Hemophilia A over the long term.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment

    Participation begins after completing a primary treatment study or being enrolled in one at the time of its closure. Eligibility requires the ability to provide informed consent.

  2. Step 2

    Treatment administration

    The study involves the use of valoctocogene roxaparvovec, administered as a solution for infusion through intravenous use. This is a gene therapy treatment aimed at addressing hemophilia A.

  3. Step 3

    Monitoring and follow-up

    The primary objective is to evaluate the long-term safety of the treatment. Monitoring includes checking for adverse reactions, serious events, and specific concerns such as liver issues, blood clots, and the development of inhibitors against the treatment.

    Secondary assessments involve tracking changes in bleeding rates, factor VIII activity, and the use of additional medications. Quality of life and overall impressions of severity and change are also evaluated at specific intervals, such as Week 52.

  4. Step 4

    Study duration

    The study is expected to continue until November 13, 2036, with recruitment starting on June 17, 2024.

Who can join the trial?

4 criteria

  • Participants must have completed their primary treatment study or be currently enrolled in it at the time of closure by the Sponsor. This means they should have been part of specific studies related to their condition.
  • Participants can join the study even if they have restarted their regular treatment for **Hemophilia A**. This includes treatments like **FVIII prophylaxis**, which is a preventive treatment to reduce bleeding episodes.
  • Participants must be able to give signed informed consent. This means they need to understand the study details and agree to follow the rules and requirements outlined in the consent form.
  • Only male participants are eligible for this study.

Who cannot join the trial?

3 criteria

  • Patients who do not have Hemophilia A cannot participate. Hemophilia A is a condition where blood doesn't clot properly.
  • Only male patients are allowed to participate. Female patients cannot join the study.
  • Patients who are considered part of a vulnerable population are not eligible. A vulnerable population includes groups who might be at higher risk of harm or exploitation.
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Investigated drugs

BMN 270 is a gene therapy being studied for its potential to treat severe Hemophilia A. It uses an adeno-associated virus vector to deliver a copy of the human Factor VIII gene to the patient's cells. This therapy aims to help the body produce its own Factor VIII, which is a protein necessary for blood clotting. The trial is focused on evaluating the long-term safety of this treatment in patients who have previously received it.

What is already known about the treatment

BMN 270 – This medication is administered through gene therapy, specifically using an adeno-associated virus vector to deliver the human Factor VIII gene. It is currently under investigation in clinical trials, with a focus on its long-term safety for treating Hemophilia A. The main therapeutic indication is for individuals with severe Hemophilia A, a condition characterized by a deficiency in Factor VIII, which is crucial for blood clotting. At the molecular level, BMN 270 works by introducing a functional copy of the Factor VIII gene into the patient's liver cells, enabling the production of the missing protein. It is classified under gene therapy products, representing a novel approach to treating genetic disorders.

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 nosebleeds, easy bruising, and joint bleeds, which can cause pain and swelling. Over time, repeated joint bleeds can lead to joint damage and mobility issues. The severity of symptoms can vary, with some individuals experiencing mild symptoms and others having more severe bleeding episodes. Hemophilia A is typically diagnosed in childhood, often after a significant bleeding event.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-507749-27-00Protocol code270-401 (GENEr8-LTE)Estimated enrolment200 patientsSponsorBiomarin Pharmaceutical Inc.

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