Comparing 3 approaches for haemophilia a

Exclusion

• −Participant who has discontinued or been withdrawn from studies NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728.
• −Participation (i.e., signed informed consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more →) in any interventionalInterventional studyA study where participants are given something to see what happens.Read more →, clinical study (except from study NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728) of an approved or non-approved investigational medicinal product.
• −Full-term pregnancy (gestational age ≥37 weeks).
• −Known or suspected hypersensitivity to trial product or related products.
• −Any disorder, except for conditions associated with haemophilia A, which in the investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →'s opinion might jeopardise participant's safety or compliance with the protocolProtocolThe detailed plan a study must follow.Read more →.
• −Previous participation in this study. Participation is defined as signed informed consent.
• −Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation.
• −Inclusion criteriaInclusion criteriaThe things you must have or be for a study to consider you.Read more →
• −Informed consent 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.
• −Male or female with diagnosis of congenital severe haemophilia A (endogenous FVIII activity less than (\<) 1 percentage \[%\]) with or without FVIII inhibitors based on medical records.
• −Aged \<1 year at the time of signing informed consent.
• −Body weight greater than or equal to (≥) 3.2 kilograms at the time of signing informed consent.
• −previously untreated patients (PUPs) or minimally treated patients (MTPs) (i.e., up to 5 days of exposure to haemophilia-related treatment such as plasma-derived FVIII, recombinant FVIII, fresh frozen plasma, cryoprecipitate, or whole blood products).
• −Participant's parent(s)/LAR(s) willingness and ability to comply with scheduled visits and ArmArmOne of the groups in a study, each receiving something different.Read more → 3 (infant) procedures, including the completion of diary and patient reported outcome (PRO) questionnaire.
• −Participants \<3 months of age must show no signs of active intracranial haemorrhage at screeningScreeningThe checks done before joining, to see whether a study fits.Read more →. This is confirmed by cranial ultrasound performed according to local practice and regardless of delivery method.
• −Receipt of vitamin K prophylaxis (as per local standard practice).
• −Availability of historical results in medical records for:
• −activated partial thromboplastin time (aPTT)
• −FVIII levels.
• −Availability of historical results in medical records or pre-dose sample taken for:
• −fibrinogen
• −haematology parameters
• −biochemistry parameters (aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT), bilirubin and creatinine).
• −Exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more →
• −Previous participation in study 4532. Participation is defined as signed informed consent.
• −Participation (i.e., signed informed consent) in any interventional clinical study with receipt of the last dose within 6 months (or 5 half-lives of the investigational medicinal product, whichever is shorter) before planned enrolmentEnrolmentThe number of participants a study plans to include, or has included.Read more →.
• −Exposure to non-factor haemostatic products for bleeding prophylaxis within 6 months (or 5 half-lives of the medicinal product, whichever is shorter) before planned enrolment.
• −Known congenital or acquired coagulation disorders other than haemophilia A.
• −Other conditions (e.g., autoimmune disease) or laboratory abnormality that may increase the risk of bleeding or thrombosis, as evaluated by the investigator. Any disorder, except for conditions associated with haemophilia A, that in the investigator's opinion might jeopardise the participant's safety or compliance with the protocol.
• −Lack of adequate parental/legally acceptable representative (LAR) support to enter accurately and timely information regarding treatment and bleeding episodes into an (electronic) diary.
• −Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease.
• −Immune tolerance induction planned to take place after treatment initiation.
• −Hepatic dysfunction defined as AST and/or ALT greater than (\>) 3 times the upper limit of normal (ULN) combined with total bilirubin \>1.5 times the ULN.
• −Serum creatinine above 1.5 times the ULN.
• −Female who is pregnant, breast-feeding or intends to become pregnant.
• −Female of child-bearing potential and not using a highly effective contraceptive method (highly effective contraceptive measures or as required by local regulation or practice).
• −Any planned major surgery, during part 1 of the study.
• −Any planned major surgery, during part 1 of Arm 3 (infant). For definition of major surgery.