Active not recruitingPHASE2INTERVENTIONAL

Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

This study is for men aged 18 to 65 with haemophilia B. It’s testing a new treatment called PF-06838435. For people who have already had this treatment in a previous study, researchers are checking its long-term safety and how well it continues to work. There’s also a new part of the study for people who haven't had the treatment before. This new part is looking at a higher dose of PF-06838435 to see if it's safe and how the body handles it. The goal is to understand if this treatment can help people with haemophilia B live healthier lives by providing long-lasting benefits.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Pfizer

Enrolment target

Start

22 Jun 2017

Estimated completion

25 Oct 2029

Hemophilia B

What is this study about?

This study is called a 'clinical trial' and it's looking into a new treatment for a condition called haemophilia B. Haemophilia B is a bleeding disorder where your blood doesn't clot properly. The new treatment being tested is called PF-06838435. This is a type of gene therapy, which aims to give your body the instructions it needs to make the clotting factor it's missing.

There are two main parts to this study. The first part is for people who have already received this new treatment in an earlier study. Here, the doctors want to keep a close eye on them over a long time to make sure the treatment continues to be safe and effective. They'll be checking to see if the treatment keeps working as it should and if there are any long-term effects, good or bad.

Now, there's also a new part of the study (called a 'dose-escalation sub-study') for people who haven't received PF-06838435 before. In this part, researchers are giving a slightly higher dose of the treatment than was used previously. The main goal here is to see if this higher dose is safe and well-tolerated by the body. All participants will be carefully monitored for their safety and how well the treatment works over time. The information gathered from this study will help doctors understand more about this potential new medicine and how it could help people with haemophilia B in the future.

Key takeaways

New gene therapy (PF-06838435) for haemophilia B is being tested.
This study focuses on long-term safety and a higher dose.
Only men aged 18-65 with haemophilia B are currently eligible.
Close medical monitoring for a long time is required.
Potential for long-lasting treatment, but risks are possible.

Who may be eligible?

This study is currently only accepting new participants for the part that explores a higher dose of the treatment. To join this part, you need to be a male between 18 and 65 years old and have a confirmed diagnosis of haemophilia B. You should also have experience with treatment for haemophilia B, having used factor IX products for at least 50 days in the past.

There are also some important things that would mean you can't join the study. For example, if you have active hepatitis B or C, or significant liver problems, you wouldn't be able to take part. Also, if you've had certain reactions to medicines in the past, or if you've previously had gene therapy, you wouldn't be eligible. The study team needs to make sure it's safe for everyone involved, so they have strict rules about who can join.

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 a man between 18 and 65 years old?
Do you have a confirmed diagnosis of haemophilia B?
Have you used Factor IX products for treatment in the past (at least 50 days)?
Do you have any active liver conditions like active Hepatitis B or C?
Have you ever had gene therapy or another clinical study in the last 3 months?
Are you willing to use contraception and follow specific guidelines after treatment?

Answer every question to see your result.

What does participation involve?

If you join the study, you'll receive a single dose of the study treatment through an intravenous (IV) drip, which means it will be given directly into a vein. You'll have regular visits to the clinic so the study team can monitor your health, take blood samples, and check how the treatment is affecting you. These visits will help them understand if the treatment is safe and working as expected.

The study involves a long-term follow-up, which means you'll be monitored for an extended period after receiving the treatment. This is important to track the lasting effects of the gene therapy. Throughout the study, you'll need to follow specific instructions, including using reliable contraception or avoiding sex, and not donating sperm until tests show the treatment is no longer present in your semen. The exact number of visits and the full duration of your participation will be explained in detail by the study team.

Potential risks and benefits

Participating in this study might offer potential benefits, such as receiving a new and potentially long-lasting treatment for haemophilia B that could reduce bleeding episodes. However, as with any new medicine, there are potential risks and side effects that are not yet fully known, especially with a higher dose. The study team will explain all known and potential risks to you. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (25)

UC Davis Comprehensive Cancer Center
Verified postcode
Sacramento, United States
UC Davis Ellison Ambulatory Care Clinic
Verified postcode
Sacramento, United States
UC Davis Medical Center department of Radiology
Verified postcode
Sacramento, United States
UC Davis Medical Center
Verified postcode
Sacramento, United States
UC Davis Midtown Cancer Center
Verified postcode
Sacramento, United States
UC DavisHealth Main Hospital
Verified postcode
Sacramento, United States
LA Center for Bleeding and Clotting Disorders - Metairie
Verified postcode
Metairie, United States
Tulane Lakeside Hospital
Verified postcode
Metairie, United States
LA Center for Bleeding and Clotting Disorders
Verified postcode
New Orleans, United States
Tulane University Clinical Translational Unit
Verified postcode
New Orleans, United States
Tulane University Hospitals and Clinic
Verified postcode
New Orleans, United States
Tulane University School of Medicine
Verified postcode
New Orleans, United States

Common questions

What is haemophilia B?

Haemophilia B is a condition where your blood doesn't clot properly, leading to bleeding that can be hard to stop.

What is PF-06838435?

It's a new treatment being tested in this study, designed to help your body make the clotting factor it needs.

Who can join this study?

Currently, only men aged 18-65 with haemophilia B, who meet specific health requirements, can join the part of the study exploring a higher dose.

Will I have to pay to be in the study?

No, participation in a clinical trial, including the study treatment and related visits, is typically free of charge.

Can I leave the study at any time?

Yes, you have the right to leave the study at any point, and it won't affect your regular medical care.