Active not recruitingPHASE3INTERVENTIONAL

A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors

This research study is looking at a new medicine called concizumab. It's for people who have haemophilia A or B. Some people with haemophilia also develop something called 'inhibitors', which makes treatment more complicated, and this study includes them too. The main goals are to find out how well concizumab prevents bleeding episodes and to make sure it's safe for people to use. If you were to join, you would need to inject the study medicine daily yourself, using a special pen, just under your skin. The study is expected to last for at least two to four years, depending on when the medicine might become available in your country.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Novo Nordisk A/S

Enrolment target

153

Start

24 Mar 2022

Estimated completion

02 Nov 2029

Haemophilia A and B With and Without Inhibitors

What is this study about?

This study is about a new medicine called concizumab, which is being tested for people who have haemophilia A or B. Haemophilia is a condition where your blood doesn't clot properly, leading to longer bleeding and sometimes internal bleeding. People with haemophilia can sometimes develop 'inhibitors', which are antibodies that fight against their usual treatment, making it less effective. This study is important because it includes people with and without these inhibitors, aiming to find new ways to manage their condition.

The main purpose of this study is to see if concizumab can help stop bleeding episodes in a safe way. If successful, this could offer a new and potentially easier treatment option for many people with haemophilia. The medicine is given as a daily injection just under the skin, which is a different way of taking some haemophilia medicines.

This is a 'Phase 3' study, which means the medicine has already been through earlier tests and shown some promise. This phase is usually the final step before a medicine might be approved for wider use. It helps researchers understand how it works over a longer period and in a larger group of people, making sure it's both effective and safe for future patients.

Key takeaways

This study is testing a new haemophilia medicine called concizumab.
It's for people with haemophilia A or B, including those with 'inhibitors'.
The main goals are to prevent bleeds and ensure the medicine is safe.
Concizumab is given as a daily injection under the skin.
Participation could last from two to four years.
You can leave the study at any time if you wish.

Who may be eligible?

To be able to join this study, you would first need to give your permission by signing a consent form. This makes sure you understand what the study involves before you start any checks.

The study is looking for people who have been diagnosed with a severe type of haemophilia A, or a moderate to severe type of haemophilia B. It also includes people who have haemophilia and have developed inhibitors.

There are specific rules about past treatments, especially for children under 12 and those with inhibitors. For some, especially those with inhibitors, you would need to have a history of treating bleeds with specific medicines for at least 26 weeks in the past year.

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 a diagnosis of severe haemophilia A or moderate/severe haemophilia B?
Have you developed 'inhibitors' as part of your haemophilia?
Are you, or the person you care for, male and under 12 years old for some parts of the study?
Have you signed a consent form and understood what the study involves?
For some with inhibitors, have you had specific 'on-demand' treatments for bleeding in the past year?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would first have some checks to make sure the study is right for you. If you qualify, you would then start taking the study medicine, concizumab. This involves giving yourself an injection just under your skin every day using a special pen.

The study will involve regular visits to the clinic so the research team can monitor your health, how the medicine is working, and check for any side effects. You'll also likely have blood tests and other assessments during these visits.

The total time you spend in the study could range from about two years up to four years. The exact length depends on whether the medicine becomes available to buy in your country, as the study aims to follow participants until then.

Potential risks and benefits

Like all medicines, concizumab may have potential benefits and potential risks. The main potential benefit is that it might help prevent bleeding episodes more effectively or offer a more convenient treatment option. However, there could be side effects from taking the new medicine, and daily injections might cause discomfort or skin reactions. The research team will carefully monitor you for any problems. It's very important to remember that you are free to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (91)

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

Rady Childrens Hosp San Diego
Verified postcode
San Diego, United States
University of Colorado Hospital
Verified postcode
Aurora, United States
Arnold Palmer Children's Hospital
Verified postcode
Orlando, United States
Nemours Child Orlando Hem/Onc.
Verified postcode
Orlando, United States
Augusta Univ/Childrens Hosp-GA
Verified postcode
Augusta, United States
Memorial Health University Medical Center
Verified postcode
Savannah, United States
Childrens Hospital of Chicago
Verified postcode
Chicago, United States
Indiana Hemophilia-Thromb Ctr
Verified postcode
Indianapolis, United States
Children's Hosp-New Orleans
Verified postcode
New Orleans, United States
Ochsner Clinic Foundation
Verified postcode
New Orleans, United States
The Children's Mercy Hospital
Verified postcode
Kansas City, United States
Children's Nebraska
Verified postcode
Omaha, United States

Common questions

What is haemophilia?

Haemophilia is a rare inherited condition where your blood doesn't clot properly, which can lead to longer bleeding after an injury or even bleeding inside the body.

What are 'inhibitors' in haemophilia?

Inhibitors are antibodies that your body can sometimes make against the medicine used to treat haemophilia. This makes the standard treatment less effective.

How is the study medicine given?

The study medicine, concizumab, is given as a daily injection just under your skin, using a special pen.

How long will the study last?

The study will last for at least two years, and could be as long as four years, depending on how quickly the medicine becomes available where you live.

Can I stop being part of the study once I start?

Yes, you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.