Active not recruitingPHASE1, PHASE2INTERVENTIONAL

Gene Therapy for Haemophilia A.

This study is exploring a new gene therapy treatment for people with severe Haemophilia A. Haemophilia A is a condition where your blood doesn't clot properly, usually requiring frequent injections to prevent bleeding. This new gene therapy, given as a single injection, aims to teach your body to make its own clotting factor, called Factor VIII, which is missing in Haemophilia A. Researchers hope this could be a long-lasting solution, reducing the need for regular treatments. This study is in its early stages (Phase 1/2), focusing on whether the treatment is safe and how well it works at different doses for adult males.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

University College, London

Enrolment target

Start

14 Jun 2017

Estimated completion

01 Dec 2029

Hemophilia A

What is this study about?

Haemophilia A is a lifelong condition where your blood doesn't clot as it should. This is because your body doesn't produce enough of a special protein called Factor VIII. People with severe Haemophilia A often need regular injections, two or three times a week, of this clotting factor to prevent dangerous bleeding. While effective, these treatments are time-consuming and can be expensive.

This study is looking at a new and exciting approach: gene therapy. Instead of regular injections, gene therapy aims to fix the underlying problem. It works by delivering a new instruction manual to your body's cells, teaching them how to make their own Factor VIII. This would ideally mean that a single treatment could provide a long-lasting solution, reducing your reliance on frequent injections.

The specific gene therapy being tested uses a harmless virus (a type called AAV) to carry the new Factor VIII instruction into your body. This type of gene therapy has shown promising results in other bleeding disorders, with patients producing their own clotting factor for many years. This study will carefully check if this new gene therapy is safe and effective for adults with severe Haemophilia A, starting with low doses and gradually increasing them while closely monitoring participants.

Key takeaways

A new gene therapy for severe Haemophilia A is being tested.
It aims to help your body make its own clotting factor (Factor VIII).
The treatment is given as a single injection.
The study will check for safety and how well it works over 5 years.
It's for adult men with severe Haemophilia A only.

Who may be eligible?

This study is looking for adult men aged 18 or older who have been diagnosed with severe Haemophilia A. This means your natural Factor VIII levels are very low (less than 1% of normal). You should also have had significant bleeding problems in the past, or require regular treatment to prevent bleeding.

To be considered, you must have received Factor VIII treatment at least 50 times in your life. You also need to be able to fully understand the study and agree to participate, including a 5-year follow-up period. Because this treatment involves genetic material, you'll also need to agree to use barrier contraception until certain tests show the treatment is no longer detectable in semen.

You won't be able to join if you have developed antibodies (called 'inhibitors') against Factor VIII, or if you have had them in the past. Other reasons for not being able to join include taking other experimental treatments for haemophilia recently, having active Hepatitis B or C infection, HIV, significant liver problems, or uncontrolled conditions like glaucoma, diabetes, or high blood pressure. Your doctor will also need to confirm that you don't have any other serious health conditions that would make taking part unsafe.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you an adult man (18 or older)?
Do you have a confirmed diagnosis of severe Haemophilia A (very low Factor VIII levels)?
Have you had at least 50 Factor VIII treatments in your life?
Do you currently have ongoing bleeding issues or need regular treatment for bleeding?
Do you *not* have Factor VIII inhibitors (antibodies) now or in the past?
Do you *not* have active Hepatitis B or C, HIV, or significant liver problems?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive the gene therapy as a single injection. After this, you will have regular check-ups and assessments over a period of 5 years. These check-ups will involve blood tests to monitor your Factor VIII levels and overall health, and to look for any side effects. You will also have appointments to discuss your progress and any bleeding episodes. Throughout the 5 years, the medical team will be closely monitoring your health and how well the treatment is working. It's a long-term commitment to ensure your safety and to understand the full effects of the gene therapy.

Potential risks and benefits

This study offers the potential benefit of reducing or even eliminating the need for frequent Factor VIII injections, potentially improving your quality of life. However, as with any new treatment, there are potential risks. The gene therapy might cause side effects, and its long-term effects are still being studied. The research team will carefully monitor you for any problems. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

St. Luke'S Regional Medical Center, Ltd
Verified postcode
Boise, United States
University of Kentucky
Verified postcode
Lexington, United States
St Jude's Children's Research Hospital
Verified postcode
Memphis, United States
Royal Free Hospital
Verified postcode
London, United Kingdom

Common questions

What is gene therapy?

Gene therapy aims to fix health problems by giving your body new instructions to make a missing or faulty protein, in this case, Factor VIII, which helps your blood clot.

How is the gene therapy given?

The gene therapy is given as a single injection directly into your body.

How long will I be involved in the study?

If you join, you will be followed up for 5 years after receiving the gene therapy to check on your health and how the treatment is working.

Will this cure my Haemophilia A?

The hope is that this gene therapy could provide a long-lasting solution by allowing your body to produce its own Factor VIII, potentially reducing or eliminating the need for current treatments. More research is needed to fully understand its long-term effects.

What happens if I change my mind during the study?

You are free to leave the study at any time, for any reason, without it affecting your ongoing medical care.