Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients with Mild or Moderate Hemophilia A without FVIII Inhibitors

This research study is testing a new medicine called Hemlibra in adults who have mild or moderate haemophilia A. The main goal is to understand how safe this medicine is and if it helps prevent or reduce bleeding. Researchers will also look at how the body processes Hemlibra and how it might affect people's daily lives and overall health. This is a large-scale study, meaning it’s a later stage of research designed to confirm earlier findings and gather more information before it can be widely used. It's important for those with haemophilia A to know about new treatment options that could potentially improve their quality of life.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

F. Hoffmann-La Roche AG

Enrolment target

Start

30 Jul 2024

Mild or moderate hemophilia A without FVIII inhibitors

What is this study about?

This study is about a medicine called Hemlibra, which is being tested for people who have a condition called haemophilia A. Haemophilia A is a bleeding disorder where the blood doesn't clot properly. The study is specifically for adults with a mild or moderate form of haemophilia A who don't have certain 'inhibitors' that can make treatments less effective.

Researchers want to find out several things. Firstly, they want to understand how safe Hemlibra is. This means they will carefully watch for any side effects and how serious they might be. Secondly, they want to see how well Hemlibra works at preventing or reducing bleeding episodes, which are a common problem for people with haemophilia. They'll also be looking at how the body uses and processes the medicine, and how it affects everyday life and general well-being.

This is a 'Phase III' study, which is a big, important step in testing new medicines. It's designed to confirm the results seen in earlier, smaller studies and gather a lot of information on safety and how well it works in a larger number of people. The aim is to see if Hemlibra could be a good new option for managing haemophilia A and improving the lives of those living with the condition.

Key takeaways

This study is testing a new medicine, Hemlibra, for adults with mild or moderate haemophilia A.
It aims to understand the safety and effectiveness of Hemlibra in preventing bleeding.
The study will closely monitor for any side effects and impacts on quality of life.
Participants must be 18 years or older and not have FVIII inhibitors.
It's a Phase III study, an important step before a new medicine can be widely available.

Who may be eligible?

To be considered for this study, you need to be an adult, 18 years old or older. The study is open to both men and women.

Crucially, you must have been diagnosed with mild or moderate haemophilia A. This study is specifically for people who do not have what are known as FVIII inhibitors. These inhibitors can sometimes develop in people with haemophilia and make certain treatments less effective, so this study is not for them.

If you meet these basic requirements, the research team will do further checks to make sure the study is a good fit for you and that you can safely take part.

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 18 years old or older?
Do you have a diagnosis of mild or moderate haemophilia A?
Have you been told you do NOT have FVIII inhibitors?
Are you able to attend regular clinic visits for assessments?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be given the study medicine, Hemlibra, as an injection. There are two different strengths of the injection. You'll have regular visits to the clinic where the research team will monitor your health very closely.

During these visits, they will ask you about any bleeds you've had, examine you physically, check your vital signs like blood pressure and heart rate, and perform ECGs (heart tests). They'll also take blood samples to check how your body is reacting to the medicine and if there are any changes. You'll be asked to complete questionnaires about your quality of life, joint health, and how you feel about the medicine. Women taking part might also be asked about their menstrual cycles. The full duration of your participation, including follow-up, will be explained by the study team.

Potential risks and benefits

Taking part in a study like this might offer the potential benefit of accessing an investigational new treatment that could help manage your haemophilia. However, it's important to remember that all medicines, including those in studies, can have side effects. Researchers will be carefully monitoring for any issues like allergic reactions, skin reactions at the injection site, or clotting problems, which are a focus of this study. You will be told about all known risks before you agree to participate, and you always have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

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

—
Unverified
Germany
—
Unverified
France
—
Unverified
Belgium

Common questions

What is haemophilia A?

Haemophilia A is a genetic bleeding disorder which means your blood doesn't clot properly. This can lead to longer bleeding after an injury or surgery, and sometimes internal bleeding.

What are FVIII inhibitors?

FVIII inhibitors are antibodies that some people with haemophilia A develop. These antibodies can stop standard haemophilia treatments from working effectively. This study is for people who do NOT have these inhibitors.

What does 'Phase III' study mean?

'Phase III' means this is a later stage of research. It involves a larger number of people and aims to confirm the safety and effectiveness of the new medicine before it can be considered for wider use.

What is Hemlibra?

Hemlibra is the name of the medicine being tested in this study. It's a type of treatment designed to help the blood clot in people with haemophilia A.

Will I know if I'm getting the actual study medicine?

This study is 'open-label', which means both you and your study doctor will know that you are receiving the study medicine, Hemlibra. There is no placebo group in this particular study.