Active not recruitingPHASE3INTERVENTIONAL

Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors

This research study is investigating a new medicine called concizumab for people in the UK who have haemophilia A or B with inhibitors. The main goal is to find out how well concizumab works to prevent bleeding and to make sure it's safe. If you usually only take medicine when you bleed, you'll be put into one of two groups by chance. One group receives concizumab from the start, while the other continues their usual medicine for 6 months before switching to concizumab. If you already take medicine to prevent bleeds or are already using concizumab in another study, you'll receive the study medicine from the beginning. Concizumab is given as a daily injection under the skin, which you can do yourself at home using a special pen.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Novo Nordisk A/S

Enrolment target

134

Start

21 Oct 2019

Estimated completion

21 Feb 2027

Haemophilia A With Inhibitors Haemophilia B With Inhibitors

Results

Results from this study

Posted April 2025

Results have been published for this study.

Primary outcome

Rate of Treated Spontaneous and Traumatic Bleeding Episodes

Rate of treated spontaneous and traumatic bleeding episodes is presented. The observation period used for reporting this endpoint is on-treatment without ancillary therapy excl. data on initial regimen for participants exposed to both regimens (OTwoATexIR). It is defined as the time period where participants are treated by either the new concizumab dosing regimen or the initial concizumab dosing regimen (only included if not exposed to the new concizumab dosing regimen) or are treated by on-demand treatment and additionally have not used factor-containing products not related to treatment of a bleed during any of the cases. The data is reported in the terms of annualised bleeding rate (ABR). Week 0 is defined as time of randomisation to on-demand administration or time of start of the new concizumab dosing regimen.

Full results on the registry

What is this study about?

This study is a research project designed to explore a new medicine called concizumab for individuals in the UK living with haemophilia A or B who have developed 'inhibitors'. Inhibitors are antibodies that your body might produce, which make your usual haemophilia medicines less effective at stopping bleeds.

The main aim of this study is to see if concizumab can effectively prevent bleeding episodes in your body and to check that it is safe to use. By understanding how well it works and if it has any side effects, researchers can determine if concizumab could be an important new treatment option for people like you with haemophilia and inhibitors.

Taking part would involve a commitment over several years, but it could offer a new approach to managing your condition. The study is carefully designed to gather important information, comparing different ways to introduce the new medicine, depending on your current treatment routine.

Key takeaways

New medicine (concizumab) for haemophilia A or B with inhibitors.
Aims to prevent bleeding and check safety.
Concizumab is given as a daily self-injection under the skin.
Study duration potentially up to seven years.
Close monitoring by a medical team.
Participation involves keeping an electronic diary.

Who may be eligible?

This study is looking for men aged 12 years or older. You need to have been diagnosed with haemophilia A or B from birth, and your doctors must have found that you have 'inhibitors' – substances in your blood that can make regular haemophilia medicines less effective.

You would also need to have been taking or needing certain bleeding-control medicines (called 'bypassing agents') in the last six months to be considered for the study. This helps make sure the study includes people who truly need new ways to manage their haemophilia.

However, you can't join if you're allergic to any part of the study medicine, or if you have another bleeding or clotting problem that isn't haemophilia. You also can't take part if you're currently having treatment to get rid of inhibitors, or if you have a history of blood clots or are at high risk of them.

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 male aged 12 or older?
Do you have haemophilia A or B from birth?
Have your doctors told you that you have 'inhibitors'?
Have you needed or used specific bleeding-control medicines (bypassing agents) in the last 6 months?
Do you have any history of blood clots or are at high risk of them?
Are you currently undergoing treatment to remove inhibitors?

Answer every question to see your result.

What does participation involve?

If you join this study, the first thing is to give your informed consent, meaning you'll fully understand what's involved and agree to take part. All participants will receive concizumab via a daily injection under the skin, which you will learn to do yourself at home using a special pre-filled pen.

Your involvement will last for quite a long time, potentially up to seven years. The exact length depends on when you started and when the medicine becomes widely available in your country. For the first 6 to 12 months, you'll have visits with the study team approximately every four weeks. After that, your visits will be about every eight weeks for the rest of the study. You'll also be asked to record information into an electronic diary throughout the study, and some participants might wear an activity tracker.

Potential risks and benefits

There are potential benefits to taking part in this study, such as receiving a new medicine that might help prevent bleeding more effectively. However, like all medicines, concizumab may have side effects, and some are not yet fully known. The study team will monitor you closely for any issues, especially related to blood clots, which is a potential concern for treatments that affect clotting. You are free to withdraw from the study at any time, for any reason, without it affecting your usual healthcare. The study aims to be reassuring and safe, but it's important to understand it's a new treatment being investigated.

Locations (99)

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

Center for Inherited Blood Dis
Verified postcode
Orange, United States
Children's Healthcare Atlanta
Verified postcode
Atlanta, United States
Indiana Hemophilia-Thromb Ctr
Verified postcode
Indianapolis, United States
Washington University School of Medicine_St. Louis
Verified postcode
St Louis, United States
St. Jude Affiliate Clinic at Novant Health Hemby Children's
Verified postcode
Charlotte, United States
TriStar Medical Group Children's Specialist
Verified postcode
Nashville, United States
University of Texas San Antonio
Verified postcode
San Antonio, United States
Haematology and Blood Bank Department
Verified postcode
Algiers, Algeria
CHU Constantine BEN BADIS/ Hematology department
Verified postcode
Constantine, Algeria
The Alfred
Verified postcode
Melbourne, Australia
The Royal Children's Hospital
Verified postcode
Parkville, Australia
Fiona Stanley Hospital - Haemophilia and Haemostasis Centre
Verified postcode
Murdoch, Australia

Common questions

What is haemophilia with inhibitors?

Haemophilia with inhibitors means your body makes special antibodies that stop your usual medication from working well to control bleeding.

What is concizumab?

Concizumab is a new medicine being studied to help prevent bleeding in people with haemophilia and inhibitors.

How will I take the medicine?

You'll give yourself a daily injection under your skin, using a special pen device, from the comfort of your home.

How long will the study last?

The study could last for up to seven years, with regular check-ups and follow-up appointments during that time.

Will I have to travel often?

Initially, you'll have visits about every four weeks, then every eight weeks for the rest of the study, reducing your travel frequency over time.