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

Launched by NOVO NORDISK A/S · Nov 15, 2021

Keywords

ClinConnect Summary

This clinical trial is studying a new medicine called concizumab to see how well it works for people with Haemophilia A or B, whether or not they have inhibitors (which are substances that make treatment less effective). The goal is to find out if concizumab can help prevent bleeding episodes and is safe for patients to use. Participants will need to give themselves an injection every day using a special pen-injector, and the study will last between 2 to 4 years, depending on when the treatment becomes available for purchase in their country.

To be eligible for this study, participants must have a diagnosis of severe or moderate congenital haemophilia A or B and have a history of treatment for bleeding episodes. For example, boys under 12 with inhibitors or anyone with a history of bleeding treatments may qualify. It's important to note that those with certain other health issues, like severe allergic reactions to the study medicine or specific clotting disorders, cannot participate. Participants can expect regular check-ups and support throughout the study as researchers gather information on the effectiveness and safety of concizumab.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Informed consent/assent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
• • Diagnosis of congenital severe haemophilia A (FVIII below 1%) or moderate/severe congenital haemophilia B (FIX (coagulation factor IX) below or equal to 2%), or congenital haemophilia with inhibitors.
• • For arm 1 only: Male aged below 12 years of age at the time of signing informed consent.
• • For arm 1 only: Patients with inhibitors (haemophilia A with inhibitors or haemophilia B with inhibitors)
• • 1. Patients with HAwI (haemophilia A with inhibitors) with historical medical records of a total of at least 26 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia \<1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available).
• • 2. Patients with HBwI (haemophilia B with inhibitors) with historical medical records of a total of at least 26 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia \<1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available).
• • 3. Patients with HBwI regardless of the regimen and duration of previous haemophilia treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products)
• • For arm 1 only: Patients without inhibitors (haemophilia A or haemophilia B)
• • 1. Patients with historical medical records of at least 52 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products; Surgery related PPX or short-term PPX (e.g., in relation to a severe bleed) is not allowed) during the last year prior to enrolment and with at least 3 documented treated bleeds (For participants less than (\<) 2 years of age there is no limitation for number of documented treated bleeds in the medical history) during this period
• • 2. Patients with historical medical records of a total of at least 26 weeks of PPX (prophylaxis) treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia \<1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available)
• • For arm 2 only: Male patients (regardless of age) previously treated with concizumab via compassionate use.
• Exclusion Criteria:
• • Known or suspected hypersensitivity to study intervention or related products.
• • Known inherited or acquired coagulation disorder other than congenital haemophilia.
• • Ongoing or planned Immune Tolerance Induction treatment.
• • History of thromboembolic disease (aIncludes arterial and venous thrombosis including myocardial infarction, pulmonary embolism, cerebral infarction/thrombosis, deep vein thrombosis, other clinically significant thromboembolic events and peripheral artery occlusion.). Current clinical signs of or treatment for thromboembolic disease. Patients who in the judgement of the investigator are considered at high risk of thromboembolic events (Thromboembolic risk factors could include, but are not limited to, hypercholesterolemia, diabetes mellitus, hypertension, obesity, smoking, family history of thromboembolic events, arteriosclerosis, other conditions associated with increased risk of thromboembolic events).