Active not recruitingOBSERVATIONAL

UPLC-MS/MS Monitoring of Emicizumab Therapy

This study is for people with severe haemophilia A, a condition where blood doesn't clot properly. The research focuses on a treatment called emicizumab, which helps prevent bleeding. We know that the amount of this medicine in a person's body can vary depending on things like age or body size. Sometimes, a person's body can even develop a resistance to the medicine. Doctors want to find out if regularly checking the level of emicizumab in the blood can help predict whether someone might still be at risk of bleeding. This could help doctors tailor treatment better to each patient.

At a glance

Status

Active not recruiting

Sponsor

University Hospital, Lille

Enrolment target

100

Start

13 Apr 2022

Estimated completion

01 Nov 2026

Hemophilia A

What is this study about?

This study is looking into a treatment called emicizumab, which is a medicine used for people with severe haemophilia A. Haemophilia A is a condition that affects how blood clots, leading to bleeding problems. Emicizumab helps the blood clot more effectively, reducing the risk of bleeds. The medicine stays in the body for quite a long time, about 28 days, before it's naturally cleared out.

We know that the amount of emicizumab in a person's body can be different from one person to another. This can depend on factors like their age, their weight, or how they are taking the medicine. Sometimes, a person's body might even develop an 'antidrug antibody', which basically means their immune system starts to fight against the medicine, making it less effective. Currently, it's not clear whether checking the exact level of emicizumab in a person's blood can help predict if they are still at risk of bleeding.

Because standard blood clotting tests aren't very useful for checking emicizumab levels, this study will use a special lab test (called UPLC-MS/MS) to measure the medicine in the blood. The main aim is to see if these measured levels can help doctors understand and predict a patient's risk of bleeding, allowing them to adjust treatment to keep people as safe as possible.

Key takeaways

This study looks at a bleeding prevention medicine called emicizumab for severe haemophilia A.
It aims to see if checking medicine levels in your blood can predict bleeding risk.
Both children and adults with severe haemophilia A (who are already on or starting emicizumab) can take part.
The findings could help doctors adjust treatment to better protect you from bleeds.
Taking part involves giving blood samples for specific tests.

Who may be eligible?

This study is looking for both children and adults who have been diagnosed with severe haemophilia A. This means your blood doesn't clot well because you have very low levels of a clotting factor called FVIII (less than 1%). You might have developed resistance to some treatments (called 'inhibitors') or not; either way, you could still be eligible if your doctor recommends emicizumab for you.

However, some people won't be able to join. You can't take part if you refuse to give your permission or if your haemophilia A is 'acquired' (meaning it started later in life rather than being something you were born with). You'll also be excluded if you have a different type of bleeding disorder or if you weigh less than 10 kilogrammes (about 22 pounds) – this is typically for very young children.

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.

Do you have severe haemophilia A?
Is your doctor recommending or have you started emicizumab treatment?
Are you able to give your informed consent (or your parent/guardian for a child)?
Are you an adult or a child weighing 10 kg (about 22 pounds) or more?
Do you have any other bleeding disorders or acquired haemophilia A? (If yes, you may not be eligible.)

Answer every question to see your result.

What does participation involve?

The information provided doesn't give specific details about what taking part in this study would involve, such as how many visits, what tests would be done, or for how long. Generally, studies like this might involve regular blood tests to measure the emicizumab levels. You would continue receiving your emicizumab treatment as prescribed by your doctor. The study would then look at your bleeding history in relation to your medicine levels. More detailed information would be provided by the study team if you are interested in taking part.

Potential risks and benefits

The potential benefit of this study is that it might help doctors to better understand how emicizumab works in individual patients, potentially leading to more personalised and effective treatment to reduce bleeding risks. The main risk would likely involve the discomfort and small risk associated with blood draws. There's also the possibility that your body might develop antibodies against the medicine, which is a known risk of emicizumab. You are free to withdraw from the study at any time without giving a reason, and this will not affect your future medical care.

Locations (2)

CHU de Caen
Verified postcode
Caen, France
Institut Coeur-Poumon, Pôle d'Hématologie-Transfusion, CHU
Verified postcode
Lille, France

Common questions

What is haemophilia A?

Haemophilia A is a rare inherited bleeding disorder where blood doesn't clot properly, leading to longer bleeding episodes and bruising.

What is emicizumab?

Emicizumab is a medicine given by injection that helps to prevent bleeding in people with severe haemophilia A by allowing their blood to clot more effectively.

Why is this study important?

This study aims to find out if checking the level of emicizumab in your blood can help doctors predict your risk of bleeding, which could lead to better-tailored treatment.

Will I keep taking my emicizumab?

Yes, if you join the study, you will continue to receive your emicizumab treatment as prescribed by your doctor.

Are there any side effects from emicizumab?

Like all medicines, emicizumab can have side effects. Your doctor can discuss these with you, but they are not the main focus of this study.