Concizumab Compassionate Use Programme for Patients With Congenital Haemophilia

NN7415-4807

The compassionate use programme will give participants concizumab for free, even though it is not yet approved by health authorities. This is because participants need this medicine to treat their haemophilia properly. The programme will check that participants are safe and that the medicine works for them. The programme may last for years. Participants will take one injection under their skin every day. Participants will have 4-5 visits with the study doctor for the first half year. After that they will have 1 visit every half year. At all clinic visits participants will have blood samples taken. Participants will fill in a diary between the visits. A patient is considered to have completed the programme when any of the following criteria occurred first: 1) when the patient is included in a clinical trial with concizumab or 2) up to 6 months after concizumab is commercially available in the patient's country and approved for the patient (The time span of 6 months should provide ample time for the patient to obtain concizumab commercially) or 3) the sponsor decides to discontinue concizumab clinical development for the patient's population.

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Inclusion Criteria: * Informed consent obtained before any programme-related activities. Programme-related activities are any procedures that are carried out as part of the programme. * Patients with congenital haemophilia: 1. severe haemophilia A (coagulation factor VIII (FVIII) less than 1%) or moderate/severe haemophilia B (coagulation factor IX (FIX) less than or equal to 2%) without inhibitors or 2. any haemophilia severity with documented history of inhibitors (more than or equal to 0.6 bethesda unit (BU)) who cannot be treated satisfactorily with authorised and marketed medicines (example: due to inhibitors or allergic reactions to factor-containing products, or due to poor venous access), and who are not able to enrol in clinical trials designed to support the development and registration of concizumab medicines (example: due to inhibitors or allergic reactions to factor-containing products, or due to poor venous access) as per investigator and Novo Nordisk assessment. * The potential benefit for the individual patient justifies the potential risks of treatment. Exclusion Criteria: * Known or suspected hypersensitivity to investigational medicinal product or related products. * Any condition (current or medical history), which in the investigator's or Novo Nordisk's opinion might jeopardise patient's safety or compliance with the protocol.

{'studyType': 'EXPANDED_ACCESS', 'nPtrsToThisExpAccNctId': 3, 'expandedAccessTypes': {'intermediate': True}}

2024-04-26