RecruitingOBSERVATIONAL

Treatment of Hemophilia A Patients With FVIII Inhibitors

This study is for men with Haemophilia A who have developed 'inhibitors' – meaning their bodies stop responding to standard treatments. It's a long-term observational study, not a test of a new drug, that aims to understand current approaches to managing this condition across different hospitals. Researchers want to see how various treatments, including existing ones and a newer treatment called emicizumab, are used. They'll also look at a treatment strategy called 'Immune Tolerance Induction' (ITI), which aims to remove inhibitors. The study will track patients for up to five years to gather information on how well these treatments work and if they are safe in the real world.

At a glance

Status

Recruiting

Sponsor

Emory University

Enrolment target

120

Start

17 Mar 2020

Estimated completion

01 Jun 2029

Hemophilia A

What is this study about?

Imagine you have Haemophilia A, a condition where your blood doesn't clot properly because you're missing a special protein called Factor VIII. Usually, doctors can give you this missing protein to help your blood clot. However, sometimes, a person's body can develop 'inhibitors.' This means their immune system sees the Factor VIII treatment as something foreign and tries to fight it off, making the medicine stop working. This can lead to more bleeds and serious problems.

This study aims to understand how doctors currently manage Haemophilia A in men who have developed these inhibitors. It's like watching and learning from what's already happening in different clinics around the world, rather than testing a brand new medicine. The researchers are particularly interested in a treatment approach called 'Immune Tolerance Induction' (ITI), which involves giving Factor VIII over a long period to try and train the body to accept it. They also want to see how well treatments like emicizumab, which is a newer option, are being used alongside Factor VIII.

By observing how people are treated and how they respond over a period of up to five years, the study hopes to gather important information. This information will help doctors understand which treatment approaches are most effective and safe for men with Haemophilia A and inhibitors, ultimately leading to better care in the future.

Key takeaways

The study observes how Haemophilia A with inhibitors is currently treated.
It focuses on men whose Factor VIII treatment no longer works effectively.
No new medicines are given; your current treatment will be recorded.
Information will be collected for up to five years during your regular clinic visits.
The goal is to help doctors understand the best ways to manage this complex condition.

Who may be eligible?

This study is looking for men who have Haemophilia A and have developed 'inhibitors' to their usual treatment for blood clotting. These inhibitors mean their body no longer fully responds to Factor VIII medicine. You might be eligible if you've had inhibitors before, even if you've tried reducing them with a treatment called Immune Tolerance Induction (ITI) in the past and it didn't work.

The study is specifically interested in people who are currently undergoing ITI treatment using certain Factor VIII products (Nuwiq, Octanate, or Wilate). You could also be eligible if you're on these ITI treatments and also receiving other medicines like emicizumab or bypassing agents (aPCC or rFVIIa) to help manage bleeds.

To join, you or your parent/guardian must be able to understand the study information and agree to take part by signing a consent form. However, you can't join if you have any other bleeding disorder besides Haemophilia A, or if your medical records for a past period aren't detailed enough about your treatments and bleeds.

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 diagnosed with Haemophilia A?
Have you developed 'inhibitors' that stop your Factor VIII medicine from working well?
Are you currently having treatment to try and get rid of the inhibitors, or using certain medicines like emicizumab?
Are you able to understand the study information and sign a consent form (or your parent/guardian can)?
Do you only have Haemophilia A, with no other bleeding disorders?

Answer every question to see your result.

What does participation involve?

If you join this study, you won't be given new or different medicines just for the study. Instead, the doctors will continue with your usual Haemophilia A treatment as they normally would. The study will simply observe and record information about the treatments you are already receiving. This includes details about your medication, any bleeding episodes you might have, and how well your treatments are working.

You will be placed into one of three groups based on the treatments your doctor chooses for you. You might switch groups if your treatment plan changes during the study. Researchers will follow your progress and collect information during your regular clinic visits. You will be observed for a maximum of five years. There are no extra tests or appointments required specifically for the study; all information will be gathered from your routine care.

Potential risks and benefits

There are no direct medical benefits from taking part in this study, as it doesn't involve new experimental treatments. However, the information gathered from your experience, alongside many others, will help doctors better understand the best ways to manage Haemophilia A with inhibitors, which could benefit others in the future. As the study only observes your current medical care, there are no extra risks beyond those associated with your existing treatments. You can choose to stop participating in the study at any time, for any reason, without it affecting your medical care.

Locations (2)

Arthur M. Blank Hospital
Verified postcode
Atlanta, United States· Recruiting
HZRM Hämophilie-Zentrum Rhein Main
Verified postcode
Mörfelden-Walldorf, Germany· Recruiting

Common questions

What are 'inhibitors' in Haemophilia A?

Inhibitors are like antibodies your body makes that stop your usual Haemophilia A medicine (Factor VIII) from working properly.

Will I get new medicine in this study?

No, this study only observes the treatments your doctor has already chosen for you; you won't receive any new study medicines.

How long will I be in the study?

You will be observed for up to a maximum of five years.

Is this a test of a new drug?

No, it's an observational study to learn how existing treatments for Haemophilia A with inhibitors are currently used and how well they work.

Can I leave the study if I change my mind?

Yes, you can withdraw from the study at any time without affecting your medical care.

How to find out more

Robert Sidonio, MD, MSc

robert.sidonio.jr@emory.edu 404-785-1637 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.