NCT01863758

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

Prospective, Open-label, Multicenter Phase 3b Study to Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Previously Treated Adult Patients With Severe Haemophilia A

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 66 (actual)
• Sponsor: Octapharma · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Severe Haemophilia A

Interventions

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

Timeline

• Start date: 2013-08-01
• Primary completion: 2015-01-01
• Completion: 2015-01-01
• First posted: 2013-05-29
• Last updated: 2018-01-30
• Results posted: 2018-01-30

Locations

20 sites across 8 countries: Austria, Bulgaria, Germany, Hungary, Poland, Romania, Slovakia, United Kingdom