Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

ATLAS-OLE: An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients with Hemophilia A or B, with or without Inhibitory Antibodies to Factor VIII or IX

This research study, called ATLAS-OLE, is looking at a new treatment called fitusiran for people living with haemophilia A or B. Haemophilia is a condition where your blood doesn't clot properly, leading to bleeding problems. This study is trying to understand how safe fitusiran is and how well it works over a long period. Researchers will be carefully checking if participants experience any side effects. They will also count how often people have bleeding episodes, including joint bleeds, and look at how the treatment might improve their quality of life, especially their physical health. The study is particularly interested in whether fitusiran helps whether or not your body has developed 'inhibitors' to other treatments.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Genzyme Corp.

Enrolment target

Start

17 May 2024

Hemophilia A or Hemophilia B

What is this study about?

Imagine your body has a special team of workers called clotting factors. When you get a cut, these workers rush to the spot to form a plug and stop the bleeding. If you have haemophilia, you're missing some of these important workers, so your blood doesn't clot as it should. This can lead to unexpected and sometimes serious bleeding, even from small bumps or without any clear injury.

This study, ATLAS-OLE, is a long-term project looking at a new medicine called fitusiran. It's designed to help people with haemophilia A or B, whether their bodies have developed an immune response (called 'inhibitors') to other common treatments or not. Inhibitors can make regular haemophilia treatments less effective, so new options are very important. The main goal of the study is to see how safe this new medicine is when taken over a long time. Researchers will keep a close eye on any new health problems or side effects people might have while on the treatment.

Beyond safety, the study also wants to see how well fitusiran works. They will be carefully counting how many bleeding episodes people have each year, especially spontaneous bleeds (those without a clear cause) and bleeds in the joints. Painful joint bleeds are a common and serious problem for people with haemophilia. The study also aims to understand how fitusiran might improve a person's physical health and overall quality of life, using a special questionnaire designed for people with haemophilia. This type of study is called a Phase 3 study, which means it’s one of the last steps before a medicine might be made available to everyone.

Key takeaways

This study is testing a new long-term treatment called fitusiran for haemophilia A or B.
It aims to understand how safe the new treatment is and how well it reduces bleeding.
The study includes people with or without 'inhibitors' to other haemophilia medicines.
Researchers will also look at how the treatment affects quality of life, especially physical health.
Only men aged 18 and over with haemophilia A or B can participate.
This is a Phase 3 study, meaning it's an important step towards a new treatment being available.

Who may be eligible?

This study is looking for specific people to take part, to make sure the results are clear and safe. To be considered for this study, you must be at least 18 years old. There is no upper age limit, meaning older adults can also participate.

Only men are being invited to take part in this particular study. This is because haemophilia A and B almost exclusively affect males. If you are a man aged 18 or older with a confirmed diagnosis of haemophilia A or B, you might be eligible to 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?
Are you 18 years old or older?
Have you been diagnosed with haemophilia A?
OR have you been diagnosed with haemophilia B?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll be taking a new medicine called fitusiran. You'll have regular visits to the clinic so doctors can check your health, see how you're reacting to the medicine, and manage any side effects. These visits will involve blood tests to check how the medicine is working and to monitor your general health. You'll also likely be asked to fill out questionnaires about your bleeding episodes and how you're feeling, including your quality of life. The study is designed to be long-term, meaning you'll be involved for an extended period, potentially several years, to understand the long-lasting effects of the medicine. You will receive the study medication, and all study-related medical care will be provided at no cost, though travel costs might not always be covered.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. You might benefit from a new treatment that could reduce your bleeding episodes or improve your quality of life, especially if current treatments aren't working well for you. However, fitusiran is a new medicine, and there's a chance you could experience side effects that are not yet fully known. Also, it might not work for everyone. Before joining, you will be given all the detailed information about known risks, and your study team will monitor you closely for any new issues. Remember, you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (5)

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

—
Unverified
Ireland
—
Unverified
Denmark
—
Unverified
Italy
—
Unverified
France
—
Unverified
Hungary

Common questions

What is haemophilia?

Haemophilia is a genetic condition where your blood doesn't clot properly, leading to longer bleeding after injuries or even spontaneous bleeding.

What are 'inhibitors' mentioned in the study?

Inhibitors are antibodies your body can develop against treatments for haemophilia, making those treatments less effective.

What is an 'annualized bleeding rate'?

This is a way doctors count how many bleeding episodes a person has on average over a year. A lower number is better.

What does 'Phase 3 study' mean?

It means the medicine has been tested in earlier stages and is now being widely tested in people to confirm its safety and effectiveness before possibly becoming available to everyone.

Will I get paid to take part?

Clinical studies generally don't pay you to participate, but often cover study-related medical costs. Check with the study team about travel compensation.