A Two Part Randomized Cross-Over Trial to Evaluate the Pharmacokinetics, Efficacy, and Safety Profile of Plasma Protein-Free Recombinant FVIII Formulated With Sucrose (BAY81-8973) in Previously Treated Subjects With Severe Hemophilia A Under Prophylaxis Therapy (Leopold I)

NCT01029340 Phase 3 COMPLETED Results posted

The study will assess the pharmacokinetics (part A) safety, tolerability, and efficacy of prophylaxis treatment (2 to 3 times a week) (part B) with BAY81-8973 over a one year period (split into two six month treatment periods). The study will compare 2 different methods (assays) for measuring the amount of study drug, the chromogenic substrate assay per European Pharmacopeia (CS/EP) with the classical assay (Chromogenic Substrate Adjusted, CS/ADJ). During one six month period patients will receive the study drug where the dose has been measured using the" (CS/EP) and during the other six months period the dose will be measured based on the Chromogenic Substrate Adjusted assay CS/ADJ)

Details

Lead sponsor	Bayer
Phase	Phase 3
Status	COMPLETED
Enrolment	74
Start date	2009-12
Completion	2013-03

Conditions

• Blood Coagulation Disorders
• Hemophilia A

Interventions

• Recombinant Factor VIII (BAY81-8973)
• Recombinant Factor VIII (Kogenate FS, BAY14-2222)
• Recombinant Factor VIII (BAY81-8973)
• Recombinant Factor VIII (BAY81-8973)

Primary outcomes

• Part A - Area Under the Drug Concentration-time Curve (AUC) — Samples taken at pre-injection, and at 0.25, 0.5, 1, 3, 6, 8, 24, 30 and 48 hours post injection. AUC calculated from time of injection to infinity.
  To examine the Pharmacokinetic (PK) characteristics of BAY 81-8973 and ensure that the new drug is similar to Kogenate FS. All results are based on the chromogenic assay.
• Part A - Half-life (t 1/2) — Samples taken at pre-injection, and at 0.25, 0.5, 1, 3, 6, 8, 24, 30 and 48 hours post injection.
  To examine the PK characteristics of BAY81-8973 and ensure that the new drug is similar to Kogenate FS. All results are based on the chromogenic assay.
• Part B - Annualized Number of Total Bleeds — 12 months after randomization
  The annualized number of bleeds experienced by participants

Countries

United States, Argentina, Austria, Croatia, Denmark, Germany, Hong Kong, India, Indonesia, Israel, Italy, Norway, Pakistan, Poland, Serbia, South Africa, Spain, Sweden, Taiwan, Thailand, Turkey (Türkiye), United Kingdom