Concizumab for Hemophilia
(explorer8 Trial)

Recruiting in Palo Alto (17 mi)

+151 other locations

Age: Any Age

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Novo Nordisk A/S

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 4 Jurisdictions

Trial Summary

What is the purpose of this trial?This trial tests concizumab, a new daily injectable medicine, in people with haemophilia A or B without inhibitors. The goal is to see if it can safely prevent bleeding episodes. Participants will either start the new medicine immediately or after a period of time, depending on their current treatment plan. Concizumab has shown significant reduction in bleeding rates in previous trials.

Eligibility Criteria

This trial is for males aged 12 or older with severe haemophilia A (FVIII below 1%) or B (FIX equal to or below 2%), without inhibitors. Participants must consent and be able to inject themselves daily with the study drug, concizumab. They cannot join if they have other coagulation disorders, a history of blood clots, are at high risk for clotting events, or are allergic to ingredients in the trial product.

Inclusion Criteria

Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.

Congenital severe haemophilia A (FVIII below 1%) or B (FIX equal to or below 2%).

I am a male aged 12 or older.

Exclusion Criteria

You have had an allergic reaction or are at risk for an allergic reaction to the trial product or related products.

I have a blood clotting disorder that is not congenital haemophilia.

I have a history of blood clots or am currently being treated for them.

+1 more

Participant Groups

The effectiveness of concizumab in preventing bleeds in people with haemophilia A or B is being tested. Some participants will start on concizumab immediately while others will begin after six months; assignment is random. Those already on prophylaxis treatment will receive it from the start. The medicine is self-administered daily via a pen-injector.

4Treatment groups

Experimental Treatment

Group I: Arm 4: Concizumab prophylaxisExperimental Treatment1 Intervention

Arm 4 will include patients previously on prophylaxis with factor products with a minimum of 24 weeks observation in NN7415-4322 (explorer 6) (at least 30 HA and 30 HB patients). In addition, arm 4 will also include: 1) Patients who were randomised to arms 1 and 2 before the treatment pause. 2) HA patients who were in NN7415-4255 (explorer 5) at the time of the treatment pause, and who have now completed explorer 5. 3) On demand patients included after arms 1 and 2 are closed.

Group II: Arm 3: Concizumab prophylaxisExperimental Treatment1 Intervention

The HA patients enrolled into the concizumab phase 2 trial NN7415-4255 (explorer 5) will be offered enrolment into this arm.

Group III: Arm 2: Concizumab prophylaxisExperimental Treatment1 Intervention

HA and HB patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis.

Group IV: Arm 1: No prophylaxis (PPX)Experimental Treatment1 Intervention

Haemophilia A (HA) and haemophilia B (HB) patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis. In the extension phase, this group will receive treatment with concizumab.

Concizumab is already approved in European Union, United States, Canada, Australia for the following indications:

🇪🇺 Approved in European Union as Alhemo for: