A Non-randomized, Open-label Study To Evaluate The Pharmacokinetics, Safety And Efficacy Of Refacto Af In Previously Treated Pediatric Subjects Less Than Twelve Years Of Age With Severe Hemophilia A (Fviii:c <1%).

NCT00914459 Phase 4 COMPLETED Results posted

The study will be investigating pharmacokinetics, safety and efficacy in patients less than 12 years of age with severe hemophilia A that have been previously treated with Factor VIII products ( including blood products).

Details

Lead sponsor	Pfizer
Phase	Phase 4
Status	COMPLETED
Enrolment	37
Start date	2009-12
Completion	2016-04

Conditions

• Hemophilia A

Interventions

• Moroctocog alfa ( AF-CC)
• Laboratory tests

Primary outcomes

• Percentage of Participants With Clinically Significant Factor VIII Inhibitor Development — Baseline up to Month 24
  Clinically significant factor VIII (FVIII) inhibitors were defined as a central laboratory confirmed positive inhibitor of greater than or equal to (\>=) 0.6 Bethesda units (BU) using the Nijmegen modification of the Bethesda assay present at 2 consecutive blood draws within a 6-week interval and one of the following within 4 weeks before the initial or within 4 weeks following the second positive FVIII inhibitor sample collection: 1) the need for the participant to administer alternative hemostatic products in order to achieve sufficient efficacy, 2) \>=2 events indicating a decrease in the efficacy of the study treatment. Percentage of participants who developed clinically significant Factor VIII inhibitor after study drug administration were reported.
• Incremental Recovery — Days 1, 15, 50, Months 6, 18 and Final visit (up to Month 24)
  Incremental recovery was the increase in circulating FVIII activity for every international unit (IU) of ReFacto AF administered per kilogram of body weight. It was measured in international units per deciliter (IU/dL) per international units per kilogram (IU/kg).
• Terminal Elimination Half Life of ReFacto AF (t1/2) — Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1
  T1/2 was the time for the plasma concentration of drug to decrease by one-half of its original concentration.
• Clearance (CL) — Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1
  Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood.

Countries

Finland, Georgia, Italy, Romania, Serbia, Spain, Sweden, Turkey (Türkiye), Ukraine