Research Study to Look at Side Effects During Regular Injection With Factor VIII Medicine Named Turoctocog Alfa for a 8 Weeks Period

• turoctocog alfa (Drug): Patients will receive standard prophylaxis treatment and treatment of bleeding episodes, according to label. Trial product will be administered as intravenous injections (i.v.)

Primary outcomes

• Occurrence of Confirmed FVIII Inhibitor Development (≥ 0.6 BU) [Weeks 0-8]: The number of participants who confirmed the presence of FVIII inhibitor development (≥ 0.6 BU) during 8 weeks of treatment period.

Secondary outcomes

• Incidence of Adverse Drug Reactions (ARs) and Serious Adverse Reactions (SARs) [Weeks 0-12]: Incidence of adverse drug reactions (ARs) and serious adverse reactions (SARs) were calculated as the number of adverse reactions per patient years. All presented ARs and SARs are treatment emergent and related to trial product, which were defined as the events reported after trial product administration until the follow-up, 12 weeks after first treatment.
• Number of Bleeding Episodes With Successful Haemostatic Effect of Turoctocog Alfa [Weeks 0-8]: The haemostatic effect (HE) of turoctocog alfa when used for treatment of bleeding episodes was evaluated during 8 weeks of treatment. Successful haemostatic effect means the haemostatic response when used for treatment of a bleeding episode was either excellent or good. Excellent haemostatic respose: Abrupt pain relief and/or clear improvement in objective signs of bleeding episode within approximately 8 hours after a single injection. Good haemostatic response: Definite pain relief and/or improvement in signs of bleeding episode within approximately 8 hours after an injection, but possibly requiring more than 1 injection for complete resolution.
• Total Annualised Consumption of Turoctocog Alfa [Weeks 0-8]: Total consumption of turoctocog alfa was evaluated during 8 weeks of treatment and it was presented as IU of turoctocog alfa/kg body weight (BW) per year per participant.
• Incidence of Allergic or Infusion Reactions Related to the Trial Product [Weeks 0-12]: Incidence of allergic or infusion reactions related to trial products were calculated as the number of reactions per patient years. Allergic reactions are a class of adverse events related to allergy.

Sex: Male, ages: 12 Years to any

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 - Male, age above or equal to 12 years at the time of signing informed consent - Patients with the diagnosis of congenital moderate or severe Haemophilia A based on medical records. (FVIII below or equal to 5%) - Documented history of at least 150 EDs (exposure days) to FVIII containing products Exclusion Criteria: - Confirmed inhibitors to FVIII (above or equal to 0.6 BU) at screening as assessed by central laboratory - History of FVIII inhibitors - Known or suspected hypersensitivity to trial product(s) or related products - Previous participation in this trial. Participation is defined as signed informed consent - Participation in any clinical trial of an approved or non-approved investigational medicinal product within 1 month before screening (visit 1) - Any disorder, except for conditions associated with haemophilia A, which in the investigator's opinion might jeopardise patient's safety or compliance with the protocol - Immunocompromised patients due to HIV infection (defined as viral load above or equal to 400.000 copies/mL and/or CD4+ lymphocyte count below or equal to 200/μL). HIV status and CD4+ lymphocyte count /viral load results may be obtained at screening or from available medical records; results must be not older than 6 months - Known congenital or acquired coagulation disorders other than haemophilia A - Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation

Participant flow

Outcome results

• Occurrence of Confirmed FVIII Inhibitor Development (≥ 0.6 BU) (Primary), Count of participants, Participants: Adolescents (12 - <18 Years) 0; Adults (≥18 Years) 0
• Incidence of Adverse Drug Reactions (ARs) and Serious Adverse Reactions (SARs) (Secondary), Number, Number of ARs per patient years: Adolescents (12 - <18 Years) 0; Adults (≥18 Years) 0
• Number of Bleeding Episodes With Successful Haemostatic Effect of Turoctocog Alfa (Secondary), Number, Bleeding episodes with successfull HE: Adolescents (12 - <18 Years) 2; Adults (≥18 Years) 38
• Total Annualised Consumption of Turoctocog Alfa (Secondary), Mean, IU/kg BW/year/participant: Adolescents (12 - <18 Years) 7030 +/- 1053; Adults (≥18 Years) 6086 +/- 1735
• Incidence of Allergic or Infusion Reactions Related to the Trial Product (Secondary), Number, Number of reactions per patient years: Adolescents (12 - <18 Years) 0; Adults (≥18 Years) 0

Adverse events

Time frame: Week 0 - 12 (8 weeks of treatment period + 4 weeks of follow-up period).

Other adverse events by term

• Study Protocol and Statistical Analysis Plan (PDF, 2019-08-26)

NIH grants: 2017