Active not recruitingOBSERVATIONAL

A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A

This study aims to understand the long-term safety of a medicine called ADYNOVI/ADYNOVATE for people living with haemophilia A. Haemophilia A is a condition where your blood doesn't clot properly. This medicine helps prevent bleeding. Researchers are monitoring participants who are already using ADYNOVI/ADYNOVATE as part of their regular treatment to see if there are any side effects over time. The study will not change how you use your medicine but will gather information from people in a real-world setting. This helps doctors learn more about how the treatment works for different people over many years.

At a glance

Status

Active not recruiting

Sponsor

Baxalta now part of Shire

Enrolment target

207

Start

09 Jul 2020

Estimated completion

28 Feb 2030

Hemophilia A

What is this study about?

This study is about a medicine called ADYNOVI/ADYNOVATE, which is prescribed to help people with haemophilia A. Haemophilia A is a condition where the blood has trouble clotting, which can lead to bleeding. This medicine helps to replace a missing blood clotting factor, reducing the risk of bleeds. The main goal of this study is to closely observe people who are already taking ADYNOVI/ADYNOVATE to see how it affects them in the long term.

Think of it like this: when a new medicine is first approved, doctors know it's safe and effective for many people. But to truly understand how it works over many years and in different situations, ongoing research is very important. This study is designed to gather more information about ADYNOVI/ADYNOVATE as people use it in their daily lives, outside of strict laboratory settings.

The researchers are particularly interested in finding out if there are any long-term side effects that might appear after using the medicine for a while. This information helps doctors give the best possible advice and care to people with haemophilia A, ensuring treatments are as safe and effective as they can be for everyone.

Key takeaways

This study will monitor the long-term safety of ADYNOVI/ADYNOVATE for haemophilia A.
Participation will not change your current treatment plan.
The study gathers information from people already using the medicine in their daily life.
It aims to identify any long-term side effects.
Your contribution helps improve care for others with haemophilia A.

Who may be eligible?

To join this study, you need to be a person with haemophilia A who is already prescribed and using ADYNOVI/ADYNOVATE to help manage your condition. You will need to agree to take part by signing a consent form yourself, or have a parent or legal guardian sign for you if you're under 16.

Before joining, a special blood test called a factor VIII (FVIII) inhibitor test needs to show a negative result. This is a common test for people with haemophilia A. Importantly, your decision to start using ADYNOVI/ADYNOVATE must have been made with your doctor before you even thought about joining this study. The study doesn't influence your treatment choices.

You cannot join if you've already been part of this exact study before. Also, you can't join if you know you are very allergic to ADYNOVI/ADYNOVATE or similar medicines. If you are unable to understand the study information or cooperate with the study requirements, you also wouldn't be able to 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.

Do I have haemophilia A?
Am I currently prescribed and using ADYNOVI/ADYNOVATE?
Has my doctor carried out a Factor VIII inhibitor test recently that was negative?
Did I decide to start ADYNOVI/ADYNOVATE with my doctor before hearing about this study?
Am I comfortable signing a consent form (or having my parent/guardian sign)?
Do I understand what the study is about and can I cooperate with providing information?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll be giving permission for researchers to collect information about your health and how you're using ADYNOVI/ADYNOVATE. This will likely involve sharing information from your regular doctor visits and possibly some extra questions about your health and medicine use. You will continue to receive your ADYNOVI/ADYNOVATE treatment as prescribed by your own doctor, just as you would normally. This study doesn't involve giving you new medicine or changing your current treatment plan. The study will continue as long as researchers need to collect long-term information, which could be several years.

Potential risks and benefits

The main benefit of taking part in this study is helping doctors and researchers learn more about the long-term effects and safety of ADYNOVI/ADYNOVATE. This information can improve care for everyone with haemophilia A in the future. As you are already using the medication, there are no new risks from the medication itself since the study doesn't change your treatment. The main 'risk' is the time it takes to provide information to the study. You are always free to leave the study at any time, for any reason, without it affecting your medical care.

Locations (36)

UF Health Shands Hospital
Verified postcode
Gainesville, United States
SHAT of Oncohaematology Diseases
Verified postcode
Sofia, Bulgaria
Clinical Hospital Sveti Duh
Verified postcode
Zagreb, Croatia
University hospital centre Zagreb
Verified postcode
Zagreb, Croatia
University hospital centre Zagreb
Verified postcode
Zagreb, Croatia
Fakultni nemocnice v Motole
Verified postcode
Prague, Czechia
Krajska zdravotni a.s. - Masarykova nemocnice v Usti nad Labem o.z.
Verified postcode
Ústí nad Labem, Czechia
SRH Kurpfalzkrankenhaus Heidelberg GmbH
Verified postcode
Heidelberg, Germany
Vivantes Klinikum im Friedrichshain
Verified postcode
Berlin, Germany
Universitaetsklinikum Bonn AoeR
Verified postcode
Bonn, Germany
Werlhof-Institut GmbH
Verified postcode
Hanover, Germany
Heim Pal Orszagos Gyermekgyogyaszati Intezet
Verified postcode
Budapest, Hungary

Common questions

What is haemophilia A?

Haemophilia A is a genetic condition where your blood doesn't clot properly due to a lack of a specific protein called Factor VIII. This can lead to longer bleeding after injuries or even spontaneous bleeds.

What is ADYNOVI/ADYNOVATE?

ADYNOVI/ADYNOVATE is a medicine used to replace the missing Factor VIII in people with haemophilia A, helping their blood clot and preventing bleeding episodes.

Will my treatment change if I join this study?

No, your treatment with ADYNOVI/ADYNOVATE will continue exactly as your own doctor has prescribed it. This study is just observing how you use it in real life.

Do I have to take new medicine for this study?

No, you do not take any new or different medicine for this study. The study is for people already using ADYNOVI/ADYNOVATE.

How long will I be in the study?

The study is looking at long-term effects, so participation could last for several years, following you through your usual medical care.