Not recruitingRare disease

Title: Study of gene therapy Etranacogene Dezaparvovec (Hemgenix) in adult patients with moderate to severe Hemophilia B

Verified siteRegistered drugNo placebo

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What is this trial about?

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

This clinical trial studies a gene therapy treatment for people with Hemophilia B, a rare inherited blood clotting disorder. The study uses etranacogene dezaparvovec (also known as Hemgenix), which is given as a single intravenous infusion. This medication contains a modified virus that carries a working copy of the blood clotting factor gene that is defective in people with Hemophilia B.

The purpose of this study is to determine if this one-time gene therapy treatment works as well as the standard treatment of regular preventive blood clotting factor injections. The treatment involves receiving a single dose of the gene therapy through a vein, and then patients are monitored for 18 months to see how well it works.

The gene therapy works by using a harmless virus called adeno-associated virus to deliver a working copy of the Factor IX gene to the liver. This gene helps the body produce the blood clotting protein that is missing in people with Hemophilia B. The study will measure how well this treatment prevents bleeding episodes compared to regular preventive treatment with clotting factor injections.

The research process

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

Step 1
Initial treatment phase
You will receive a single intravenous infusion of Hemgenix (etranacogene dezaparvovec).
The dose will be 2 x 10^13 genome copies per kilogram of body weight.
After the infusion, you will need to use a condom during sexual intercourse until laboratory tests confirm that the treatment is no longer present in semen samples. This requires three consecutive negative test results.
Step 2
First 6 months monitoring
Your factor IX activity levels will be measured periodically.
Any bleeding events will be recorded.
Regular blood tests will monitor various health parameters.
Abdominal ultrasound examinations will be performed.
Step 3
Months 6-18 evaluation period
This is the main evaluation period lasting 52 weeks.
Your bleeding rate will be monitored and compared to your previous treatment.
Regular measurements of your factor IX activity will continue at months 6, 12, and 18.
You will complete quality of life questionnaires about your physical activity and general health.
Blood tests will continue to monitor your health status, including liver function and immune response.
Step 4
Extended follow-up
The study continues until June 2025.
Regular monitoring of your health status will continue.
Any side effects or complications will be recorded.
Your bleeding episodes and factor IX activity levels will continue to be tracked.

Who can join the trial?

9 criteria

Must be male
Must be 18 years or older
Must have congenital hemophilia B with severe or moderately severe factor IX deficiency (2% or less of normal blood clotting factor IX levels)
Must be currently receiving regular preventive treatment with factor IX protein (prophylaxis) on a fixed schedule (such as twice weekly or every two weeks)
Must have received more than 150 previous treatments with factor IX protein
Must have been on stable preventive treatment for at least 2 months before screening
Must be able to complete a patient diary accurately and on time, as determined by the study doctor
Must agree to use condom during sexual intercourse until the study medication (AAV5) is no longer present in semen, confirmed by three negative laboratory tests in a row (this applies even if surgically sterilized)
Must be able to provide informed consent after receiving verbal and written information about the trial

Who cannot join the trial?

9 criteria

Male patients who are younger than 18 years old or older than 65 years old cannot participate
Female patients cannot participate in this trial
Patients who do not have confirmed diagnosis of Haemophilia B are not eligible
Patients who belong to vulnerable populations (such as people who cannot give informed consent) are not eligible
Patients who cannot follow the 18-month study duration requirement cannot participate
Patients who cannot comply with regular factor IX (a blood clotting protein) prophylaxis treatment during the initial phase cannot participate
Patients who cannot undergo regular blood testing and medical assessments are not eligible
Patients who cannot provide informed consent in writing are not eligible
Patients who are currently participating in other clinical trials cannot take part in this study

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Verified sites

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Cliniques Universitaires Saint-Luc
Responsive
Woluwe-Saint-Lambert, Belgium
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Responsive
Rotterdam, The Netherlands
Katholieke Universiteit te Leuven
Responsive
Leuven, Belgium

Trial locations

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Investigated drugs

Based on the provided trial data: AAV5-hFIXco-Padua (also known as AMT-061 or CSL222) is a gene therapy treatment delivered through a virus vector. It is designed to help patients with hemophilia B by delivering a modified version of the Factor IX gene to their cells. This therapy aims to enable the body to produce its own clotting factor IX, which is essential for blood clotting. It is administered as a single dose and could potentially reduce or eliminate the need for regular factor IX replacement therapy. Factor IX is a standard replacement therapy used to prevent and treat bleeding in people with hemophilia B. It is typically administered regularly as a preventive treatment (prophylaxis) to maintain adequate clotting factor levels in the blood. This is the traditional treatment approach that serves as the comparison for the gene therapy in this trial.

Etranacogene Dezaparvovec

What is already known about the treatment

CSL222 (AMT-061) – A gene therapy medication administered as a single intravenous dose, containing a modified adeno-associated viral vector (AAV5) that carries the Padua variant of the human factor IX gene. This investigational treatment is designed for adults with severe or moderately severe Hemophilia B, aiming to provide long-term production of clotting factor IX in the body. The medication works at the molecular level by delivering functional copies of the modified factor IX gene to liver cells, enabling them to produce the necessary clotting protein. Currently undergoing Phase III clinical trials, this therapy represents an innovative approach in the field of genetic treatments for bleeding disorders, potentially offering an alternative to regular factor IX replacement therapy.

Investigated diseases

Hemophilia B - A rare inherited blood clotting disorder caused by a deficiency in blood clotting factor IX. The condition primarily affects males and is characterized by prolonged bleeding after injuries or surgeries. People with hemophilia B may experience spontaneous bleeding into joints and muscles, which can occur without any apparent injury. The severity of the condition varies among individuals, depending on how much factor IX their body produces. The condition is present from birth and continues throughout life. People with this disorder may develop recurring joint bleeding that can lead to joint problems over time.