Active not recruitingPHASE3INTERVENTIONAL

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

This research study is testing a new gene therapy called PF-06838435 for adult men who have moderately severe to severe haemophilia B. Haemophilia B is a condition where your blood doesn't clot properly, leading to bleeding. The gene therapy aims to help your body make the missing Factor IX, a protein needed for clotting. Researchers want to compare how well this new treatment reduces bleeding compared to standard care, which involves regular injections of Factor IX. They will also be closely watching for any side effects to make sure the treatment is safe. Participants will receive a single treatment and be followed for six years.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Pfizer

Enrolment target

Start

29 Jul 2019

Estimated completion

25 Feb 2031

Hemophilia B

What is this study about?

This study is investigating a new type of treatment called gene therapy for a blood clotting condition called haemophilia B. If you have haemophilia B, your body doesn't produce enough of a special protein called Factor IX, which is essential for your blood to clot properly. This means you might experience more bleeding than usual, either inside your body or externally.

The gene therapy being tested, called PF-06838435, works by giving your body instructions to make the missing Factor IX. The idea is that with these new instructions, your cells can start producing enough Factor IX on their own, potentially reducing your bleeding episodes. This treatment is given just once, as a single injection into a vein (intravenously).

Researchers want to see if this single gene therapy treatment can significantly lower the number of bleeding episodes you have each year compared to your current routine treatment (regular Factor IX injections). They will also be carefully monitoring everyone in the study to make sure the gene therapy is safe and to track any side effects. Your participation would help us understand if this new approach could be a better long-term option for managing haemophilia B.

Key takeaways

This study trials a single-dose gene therapy for moderately severe to severe haemophilia B.
It aims to reduce bleeding better than current regular Factor IX treatment.
Participation involves a single treatment and 6 years of follow-up.
Key eligibility includes being an adult male (18-65) and having used Factor IX prophylaxis.
The main goal is to assess how well the treatment reduces bleeding and its safety.

Who may be eligible?

To be considered for this study, you must be an adult male between 18 and 65 years old with moderately severe to severe haemophilia B. This means your body produces 2% or less of the normal amount of Factor IX. You would also need to have been on regular Factor IX treatment for at least six months just before joining this study.

There are also some important reasons why you might not be able to join. For example, if your body has previously developed resistance or a bad reaction to Factor IX, or if you have certain other serious health problems like liver disease or chronic infections, you wouldn't be eligible. Also, if you've already had gene therapy in another study, or if you have specific antibodies related to the gene therapy, you wouldn't be able to take part.

The research team will carry out several tests and checks to make sure the study is suitable and safe for you. This includes checking your blood, ensuring your liver and kidneys are working well, and that you do not have certain medical conditions that might interfere with the study or make it unsafe for you.

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 male between 18 and 65 years old?
Do you have moderately severe to severe haemophilia B (Factor IX activity 2% or less)?
Have you been on regular Factor IX treatment for at least 6 months recently?
Do you have any known severe allergies to Factor IX products?
Do you have any serious chronic infections or uncontrolled liver or kidney problems?

Answer every question to see your result.

What does participation involve?

If you join this study, you will receive the gene therapy as a single injection into a vein. Before this, you would have already completed at least six months of your usual Factor IX treatment in a related study. After receiving the gene therapy, you would typically stop your regular Factor IX treatments. The study team will then closely follow your health and progress for six years. This will involve regular visits to the clinic for medical checks, blood tests, and to record any bleeding episodes you might have. You will also need to use contraception during the study until the drug is completely out of your system.

Potential risks and benefits

Participating in this study might offer the potential benefit of reducing your bleeding episodes and possibly needing fewer regular Factor IX injections. However, as with any new treatment, there are potential risks and side effects that the study team will discuss with you. These could include reactions to the gene therapy or other unexpected health issues. You will be closely monitored for any problems. Remember, your participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (67)

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

UCSF IDS Pharmacy
Verified postcode
San Francisco, United States
University of California, San Francisco - Clinical Research Center
Verified postcode
San Francisco, United States
University of California, San Francisco - Outpatient Hematology Clinic
Verified postcode
San Francisco, United States
The Regents of the University of California, San Francisco campus
Verified postcode
San Francisco, United States
Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus
Verified postcode
Aurora, United States
Regents of the University of Colorado University of Colorado Denver, Office of Grants and Contracts
Verified postcode
Aurora, United States
Indiana Hemophilia & Thrombosis Center, Inc.
Verified postcode
Indianapolis, United States
Indiana Hemophilia and Thrombosis Center, Inc
Verified postcode
Indianapolis, United States
Innovative Hematology, Inc.
Verified postcode
Indianapolis, United States
St. Vincent Hospital & Health Care Center, Inc.
Verified postcode
Indianapolis, United States
Madison Radiological Group
Verified postcode
Madison, United States
Mississippi Center for Advanced Medicine
Verified postcode
Madison, United States

Common questions

What is haemophilia B?

Haemophilia B is a genetic condition where your blood doesn't clot properly because you don't have enough Factor IX, a protein needed for clotting.

What is gene therapy?

Gene therapy aims to fix a problem at its source by delivering new genetic material to your cells, in this case, to help your body make the missing Factor IX.

How is the treatment given?

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

How long will I be in the study?

You will be followed by the study team for six years after receiving the gene therapy.

Can I stop my regular Factor IX treatment?

Typically, you would stop your routine Factor IX treatment after receiving the gene therapy, but this will be managed by the study doctors.