Patients: 150 male, previously treated patients aged 12–65 years with severe hemophilia A (FVIII activity ≤1%) and without inhibitors to FVIII (<0.6 BU). Prior to this trial, all patients had at least 150 exposure days to any FVIII product. Exposure day is defined as every day where the patient received at least one dose of FVIII.

Study design: Multicenter, multinational, open-label, and non-controlled. Patients infused Novoeight^® prophylactically, either 20–40 IU/kg every second day or 20–50 IU/kg three times weekly. Dose for all patients started at 20 IU/kg and was adjusted as necessary at the investigator’s discretion. For breakthrough bleeds, dosage was based on achieving a plasma FVIII activity level of 0.50 IU/mL, with treatment occurring as soon as bleeds were identified. For the 150 patients, mean exposure was 85 exposure days (based on a range of 11 to 172 exposure days).

Primary endpoint: The incidence rate of FVIII inhibitors, measured by positive results for inhibitors (≥0.6 BU) in two consecutive test samples analyzed at a central laboratory.

Patients: Previously treated pediatric patients (0–11 years), adolescents (12-17 years) and adults (18-65 years) with severe hemophilia A (FVIII activity ≤1%) and without inhibitors. Patients had completed earlier phase 1 pharmacokinetic trials (guardian^™1 or guardian^™3) and chose to continue taking Novoeight^® as prophylaxis (n=207) and/or on-demand treatment (n=19).^1,2,6

Study design: Non‐randomized, open‐label, multicenter, multinational, single‐arm, safety and efficacy extension trial. Patients infused Novoeight^® prophylactically, with standard dosing (25–50 IU/kg every second day or 20–60 IU/kg three times weekly) or less-frequent dosing (40-60 IU/kg twice weekly or once every third day). The recommended dose level for on-demand treatment was 20-50 IU/kg.⁶

Primary endpoint: The incidence rate of FVIII inhibitors (≥0.6 BU).⁶

Secondary endpoint: Frequency of adverse events and serious adverse events.⁶

Patients: Male, previously treated patients aged 0–11 years (n=63) with severe hemophilia A (FVIII activity ≤1%) and without inhibitors. Prior to this trial, all patients had at least 50 exposure days to any FVIII product.

Study design: Multinational, open-label, and uncontrolled. Patients infused Novoeight^® prophylactically, either 25–50 IU/kg every second day or 20–60 IU/kg three times weekly. For breakthrough bleeds, dosage was based on achieving a plasma FVIII activity level of 0.50 IU/mL, with treatment occurring as soon as bleeds were identified. For the 63 patients, mean exposure was 60 exposure days (based on a range of 20 to 104 exposure days).

Primary endpoint: The incidence rate of FVIII inhibitors, measured by positive results for inhibitors (≥0.6 BU) in two consecutive test samples analyzed at a central laboratory.

Patients: Male patients participating in guardian^™1 (adults and adolescents), guardian^™3 (children) or guardian^™2 (safety and efficacy extension) trials. Data shown is from guardian^™1 and guardian^™3 only. All patients had severe hemophilia A (FVIII ≤ 1%) without inhibitors and were previously treated with at least 150 exposure days (adolescents and adults) or at least 50 exposure days (children) to any FVIII product.

Study design: Thirty-three male patients (7 children, 1 adolescent and 25 adults) underwent a total of 15 major and 26 minor surgical procedures. Patients received Novoeight^® as bolus injections or continuous infusion, at dosage determined by the investigator, including a loading dose immediately prior to surgery.

Endpoints: The safety and efficacy of Novoeight^® as perioperative treatment was assessed as part of the secondary objectives of the guardian^™ trial program. Efficacy assessment included hemostatic response, which was evaluated at the end of the surgical procedure. A hemostatic response rated as “excellent” or “good” was considered treatment success.

Patients: Previously untreated children (n=59) below 6 years of age with severe hemophilia A (factor VIII activity ≤1%) and no history of FVIII inhibitors.

Study design: This trial evaluated the safety and efficacy of Novoeight^® in routine prophylaxis and the on-demand treatment and control of breakthrough bleeds. Subjects received Novoeight^® at standard recommended dose levels. Breakthrough bleeds were treated at the investigator’s discretion, aiming for a FVIII activity level above 0.5 IU/mL.

Primary endpoint: Incidence rate of FVIII inhibitors. 

Efficacy endpoints: Hemostatic effect during treatment of bleeding episodes, number of infusions needed to stop a bleeding episode, and annualized bleeding rate (ABR).