Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A

Octapharma66 enrolled

Overview

To compare the number of breakthrough bleeds under tailored prophylaxis with Human cell line recombinant factor FVIII (Human-cl rhFVIII) with the historical bleeding rate from patients who received Human-cl rhFVIII as on demand treatment.

There were 3 phases in this study: (1) An initial pharmacokinetic (PK) assessment in which participants received a single infusion of 60±5 IU/kg of Human-cl rhFVIII; blood samples were collected for 72 hours following the infusion. (2) Prophylactic Treatment-Phase I during which participants received infusions of 30-40 IU/kg of human-cl rhFVIII every other day or 3x/week for 1-3 months. (3) Prophylactic Treatment-Phase II during which the dose and dosing interval were determined individually from data gathered in the initial PK assessment. The maximum dosing interval with a dose of ≤ 60-80 IU/kg that maintains a trough level of ≥ 0.01 IU/mL was determined. Participants were treated for 6 months.

Study Type

INTERVENTIONAL

Allocation

Purpose

PREVENTION

Masking

NONE

Enrollment

Conditions

Severe Haemophilia A

Interventions

Human-cl rhFVIIIBIOLOGICAL

Human-cl rhFVIII was provided as a freeze-dried concentrate to be reconstituted in water for injection.

Eligibility

Sex: MALEMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Severe haemophilia A (FVIII:C \< 1%) according to medical history. * Male patients ≥ 18 years old. * Previous treatment with a FVIII concentrate (regular prophylaxis with good compliance or on-demand treatment) for at least 150 exposure days (EDs). * Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start. * Immunocompetence (CD4+ count \> 200/microliter). * HIV-negative, if positive, viral load \< 200 particles/microliter or \< 400,000 copies/mL. * Freely given written informed consent Exclusion Criteria: * Any coagulation disorder other than haemophilia A. * Present or past FVIII inhibitor activity (\> 0.6 Bethesda Unit \[BU\]) * Severe liver or kidney disease.

Locations (20)

Medical University Vienna

Vienna, Austria

Outcomes

Primary Outcomes

Annualized Number of Bleeding Episodes (BE) in Phase II

The annualized number of total BEs was calculated for each participant as follows: d\*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as any BE whether treated or not during Phase II of the study; BEs related to surgery were not included. This study was considered as showing efficacy if the annualized number of BEs was reduced by 50% compared to the number of BEs observed in study GENA-01 where patient where severe Hemophilia A patients were treated on-demand (NCT00989196).

Time frame: Beginning to the end of Phase II (6 months)

Secondary Outcomes

Annualized Number of Spontaneous Bleeding Episodes (BE) in Phase II

The annualized number of spontaneous BEs was calculated for each participant as follows: d\*y/t, where y = the number of spontaneous BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A spontaneous bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery and BEs due to trauma or due to other causes were not included.

Time frame: Beginning to the end of Phase II (6 months)

Annualized Number of Bleeding Episodes (BE) in Phase II in Participants With ≤ 2 Treatments/Week

The annualized number of BEs was calculated for each participant as follows: d\*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery were not included.

Time frame: Beginning to the end of Phase II (6 months)

Median Dosing Interval During Individually Tailored Prophylaxis

The median time between 2 prophylactic doses of Human-cl rhFVIII in the prophylactic treatment phase II were determined per patient

Data from ClinicalTrials.gov

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