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Peboctocogene Camaparvovec

This article explores the use of Peboctocogene Camaparvovec (BAY 2599023/DTX201) in clinical trials for treating severe hemophilia A. This innovative gene therapy aims to deliver a healthy version of the defective Factor VIII gene into liver cells using a modified virus. The trials focus on safety, tolerability, and effectiveness in adult patients with severe hemophilia A who have previously been treated with Factor VIII products.

Table of Contents

What is Peboctocogene Camaparvovec?

Peboctocogene Camaparvovec, also known as BAY 2599023 or DTX201, is an innovative gene therapy being developed to treat severe hemophilia A. Hemophilia A is a genetic disorder that affects blood clotting, leading to prolonged bleeding and easy bruising.[1]

This therapy is classified as an advanced therapy medicinal product (ATMP), specifically a gene therapy product. It uses a modified virus to deliver a healthy version of the defective Factor VIII gene into the patient’s liver cells.[1]

How Does It Work?

Peboctocogene Camaparvovec works by using a specially engineered virus called adeno-associated virus serotype hu37 (AAVhu37). This virus acts as a “shuttle” to carry a healthy copy of the Factor VIII gene into the nucleus of liver cells. The gene delivered is a slightly modified version called B-domain deleted human Factor VIII (BDD-hFVIII).[1]

Once inside the liver cells, this gene allows the body to produce the missing Factor VIII protein, potentially reducing or eliminating the need for regular Factor VIII injections in patients with severe hemophilia A.[1]

Target Patient Group

This therapy is being developed for adult patients with severe hemophilia A. Severe hemophilia A is defined as having less than 1% of normal Factor VIII activity in the blood.[1]

Clinical Trial Details

A Phase 1/2 clinical trial is currently underway to test the safety and effectiveness of Peboctocogene Camaparvovec. Key details of the trial include:

  • It’s an open-label study, meaning both researchers and participants know which treatment is being given.
  • The study aims to find the right dose of the therapy.
  • Participants receive a single intravenous (IV) dose of the treatment.
  • The study is looking at different dose levels to see which one works best.[1]

Potential Benefits

The main goals of this therapy are:

  1. To achieve sustained expression of Factor VIII above 5% of normal levels at 6 and 12 months after treatment. This level could significantly reduce bleeding episodes in patients with severe hemophilia A.
  2. To potentially reduce or eliminate the need for regular Factor VIII injections, which are currently the standard treatment for severe hemophilia A.[1]

Safety Considerations

As with any new treatment, safety is a primary concern. The clinical trial is closely monitoring:

  • Adverse events (side effects)
  • Serious adverse events
  • Any special safety concerns related to gene therapy[1]

Eligibility Criteria

To participate in the clinical trial, patients must meet certain criteria. Some key eligibility factors include:

  • Male patients aged 18 or older
  • Diagnosed with severe hemophilia A (Factor VIII activity less than 1%)
  • Previously treated with Factor VIII products for at least 150 exposure days
  • No current evidence of inhibitors to Factor VIII
  • No significant liver disease or active hepatitis B or C
  • No history of certain cancers or autoimmune diseases[1]

It’s important to note that this is not a complete list of eligibility criteria. Patients interested in participating should discuss their eligibility with their healthcare provider.

Peboctocogene Camaparvovec represents a promising advancement in the treatment of severe hemophilia A. While still in clinical trials, this gene therapy approach could potentially offer long-term benefits for patients, reducing the burden of regular treatments and improving quality of life. As with all new treatments, ongoing research will help determine its safety and effectiveness.

Aspect Details
Drug Name Peboctocogene Camaparvovec (BAY 2599023/DTX201)
Type of Therapy Gene therapy using adeno-associated virus (AAV) vector
Target Condition Severe Hemophilia A
Trial Phase Phase 1/2
Main Objective Investigate safety and tolerability of single ascending IV doses
Secondary Objective Identify dose for sustained Factor VIII expression above 5% at 6 and 12 months
Key Eligibility Adult males with severe hemophilia A, previously treated with FVIII products
Administration Single intravenous dose
Primary Endpoint Incidence of adverse events and safety profile
Secondary Endpoint Expression pattern of FVIII activity and proportion of patients achieving target FVIII levels

FAQ

  • What is Peboctocogene Camaparvovec? Peboctocogene Camaparvovec, also known as BAY 2599023 or DTX201, is a gene therapy drug designed to treat severe hemophilia A. It uses a modified virus to deliver a healthy version of the Factor VIII gene into liver cells.
  • Who is eligible for the clinical trial? The trial is open to adult males (18 years or older) with severe hemophilia A who have been previously treated with Factor VIII products for at least 150 exposure days. Participants must either be on prophylaxis treatment or have experienced more than 4 bleeding events in the past year.
  • What is the main objective of the clinical trial? The main objective is to investigate the safety and tolerability of single ascending intravenous doses of Peboctocogene Camaparvovec in adult patients with severe hemophilia A.
  • What are some key exclusion criteria for the trial? Key exclusion criteria include current evidence of inhibitors to Factor VIII, history of certain malignancies, autoimmune diseases, hypersensitivity to Factor VIII products, and active hepatitis B or C infection.
  • How is the drug administered in the trial? Peboctocogene Camaparvovec is administered as a single intravenous (IV) dose. The trial aims to identify the optimal dose that will achieve sustained expression of vector-derived Factor VIII above 5% at 6 and 12 months following administration.

Ongoing Clinical Trials on Peboctocogene Camaparvovec

  • Study on the Safety and Effects of BAY 2599023 (Peboctocogene Camaparvovec) for Adults with Severe Hemophilia A

    Not recruiting

    2 1 1
    Investigated drugs:
    Bulgaria France Germany The Netherlands

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 in the blood that helps in the clotting process. People with hemophilia A have low levels or absence of this protein.
  • Gene therapy: A technique that uses genes to treat or prevent disease by introducing a healthy copy of a gene to replace a faulty one.
  • Adeno-associated virus (AAV): A small virus that is not known to cause disease and is commonly used in gene therapy to deliver genetic material into cells.
  • Prophylaxis: Preventive treatment, in this case, regular infusions of clotting factor to prevent bleeding episodes in hemophilia patients.
  • Inhibitors: Antibodies that the immune system develops against treatment factors, making them less effective or ineffective.
  • Exposure days (ED): The number of days a patient has received factor replacement therapy.
  • Intravenous (IV): Administration of a substance directly into a vein.
  • B-domain deleted human factor VIII: A modified version of the Factor VIII protein with a specific portion (B-domain) removed, which is used in some treatments for hemophilia A.
  • Neutralizing antibody: Antibodies that bind to a specific target and block its biological effect, in this case potentially interfering with the gene therapy.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-bay-2599023-peboctocogene-camaparvovec-for-adults-with-severe-hemophilia-a/