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Valoctocogene Roxaparvovec

This article discusses a clinical trial investigating the long-term safety and effects of Valoctocogene Roxaparvovec, also known as Roctavian or BMN 270, in patients with severe hemophilia A. The study aims to evaluate the drug’s long-term safety, its impact on bleeding rates, and its effects on patients’ quality of life. This follow-up study involves participants who have previously received the drug in other BioMarin clinical trials.

Table of Contents

What is Valoctocogene Roxaparvovec?

Valoctocogene roxaparvovec, also known by its brand name Roctavian, is a groundbreaking gene therapy designed to treat severe hemophilia A[1]. This innovative treatment is also referred to by several other names, including BMN 270, AAV-hFVIII-SQ, and BMN-270[1]. Hemophilia A is a genetic disorder that affects blood clotting, leading to prolonged bleeding and easy bruising.

How Does It Work?

Valoctocogene roxaparvovec uses a modified virus (called an adeno-associated virus) to deliver a functional copy of the blood-clotting factor VIII gene to the patient’s liver cells[1]. This gene therapy aims to enable the body to produce its own factor VIII, potentially reducing or eliminating the need for regular factor VIII replacement therapy.

Clinical Trial Information

A long-term follow-up study (Study 270-401, also known as GENEr8-LTE) is being conducted to evaluate the safety and effectiveness of valoctocogene roxaparvovec in patients with severe hemophilia A who have previously received the treatment in other BioMarin clinical trials[1].

Eligibility Criteria

To participate in this long-term follow-up study, patients must meet certain criteria:

  • Have completed or be currently enrolled in a primary treatment study with valoctocogene roxaparvovec[1]
  • Be capable of giving informed consent[1]
  • Be in generally good health, without any conditions that would prevent full compliance with the study requirements[1]

Study Objectives

The main objectives of this long-term follow-up study include:

  1. Evaluating the long-term safety of valoctocogene roxaparvovec[1]
  2. Assessing the long-term effects of the treatment in hemophilia A patients[1]
  3. Monitoring the use of other hemostatic agents (medications that help control bleeding)[1]
  4. Evaluating the long-term impact on the patients’ quality of life[1]

Safety Monitoring

The study will closely monitor participants for any adverse reactions or events, with special attention to:

  • Hepatotoxicity: Potential liver damage[1]
  • Thromboembolic events: Blood clots that could block blood vessels[1]
  • Development of factor VIII inhibitors: Antibodies that could interfere with the treatment[1]
  • Potential transmission to third parties[1]
  • Theoretical risk of tumorigenesis: The possibility of tumor formation[1]

Potential Benefits

The study will assess several potential benefits of valoctocogene roxaparvovec, including:

  • Changes in the annual bleeding rate[1]
  • Factor VIII activity levels over time[1]
  • Reduction in the use of other hemostatic medications[1]
  • Improvements in the patients’ quality of life[1]

Administration

Valoctocogene roxaparvovec is administered as a single intravenous infusion[1]. The solution for infusion contains 2 × 10^13 vector genomes per milliliter[1]. It’s important to note that this is a one-time treatment, and no additional dosing will occur during the long-term follow-up study[1].

Patients enrolled in this study will be monitored through regular check-ins, which may include phone calls, remote visits, or site visits, conducted quarterly for at least 10 years[1]. This long-term follow-up is crucial to understand the lasting effects and safety of this innovative gene therapy.

Aspect Details
Study Type Long-term follow-up (Phase IV)
Drug Name Valoctocogene Roxaparvovec (Roctavian, BMN 270)
Target Condition Severe Hemophilia A
Main Objective Evaluate long-term safety
Secondary Objectives Assess long-term effects, use of hemostatic agents, impact on quality of life
Study Duration ≥ 10 years
Key Outcomes Adverse events, bleeding rates, Factor VIII activity, use of other medications, quality of life measures
Eligibility Participants from previous BioMarin clinical trials of Valoctocogene Roxaparvovec

FAQ

  • What is Valoctocogene Roxaparvovec (Roctavian)? Valoctocogene Roxaparvovec, also known as Roctavian or BMN 270, is a gene therapy drug designed to treat severe hemophilia A. It uses a modified virus to deliver a functional copy of the blood clotting factor VIII gene to the patient's cells.
  • What is the main objective of this clinical trial? The main objective of this clinical trial is to evaluate the long-term safety of Valoctocogene Roxaparvovec (BMN 270) in patients with severe hemophilia A who have previously received the drug in other BioMarin clinical trials.
  • How long will participants be followed in this study? Participants will be followed for 10 years or more, with data collection occurring quarterly through phone calls, mobile nursing/remote visits, or site visits.
  • What are some of the key outcomes being measured in this study? Key outcomes include the occurrence of adverse drug reactions, changes in annual bleeding rates, Factor VIII activity levels over time, use of other hemostatic medications, and changes in quality of life measures.
  • Who is eligible to participate in this study? Eligible participants are those who have completed or are currently enrolled in a primary treatment study of Valoctocogene Roxaparvovec (BMN 270) conducted by BioMarin. They must be capable of giving informed consent and be in generally good health.

Ongoing Clinical Trials on Valoctocogene Roxaparvovec

  • Long-Term Safety Study of Valoctocogene Roxaparvovec for Patients with Severe Hemophilia A

    Not recruiting

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

Glossary

  • Hemophilia A: A genetic disorder that impairs the body's ability to form blood clots, leading to excessive bleeding. It is caused by a deficiency in clotting factor VIII.
  • Valoctocogene Roxaparvovec (Roctavian): A gene therapy drug designed to treat severe hemophilia A by providing a functional copy of the factor VIII gene.
  • Gene therapy: A technique that uses genes to treat or prevent disease, often by replacing a faulty gene or adding a new gene to help the body fight a disease.
  • Factor VIII: A protein essential for blood clotting. People with hemophilia A have little or no factor VIII, leading to bleeding problems.
  • Annualized Bleeding Rate (ABR): A measure of how often a person with hemophilia experiences bleeding episodes over a year.
  • Prophylaxis: Preventive treatment, often involving regular infusions of clotting factor to prevent bleeding episodes in people with hemophilia.
  • Health-Related Quality of Life (HRQoL): A measure of a person's perceived physical and mental health over time.
  • Adverse drug reaction: An unwanted or harmful reaction experienced following the use of a medication.
  • Hepatotoxicity: Liver damage caused by exposure to drugs or other substances.
  • Thromboembolic events: The formation of blood clots that can potentially block blood vessels, which can be dangerous.

References

  1. http://clinicaltrials.eu/trial/long-term-safety-study-of-valoctocogene-roxaparvovec-for-patients-with-severe-hemophilia-a/