Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII

Summary

The purpose of this research study is to determine the effectiveness, safety, and pharmacokinetics (PK) of moroctocog alfa (AF-CC) in previously treated subjects, who are younger than 6 years of age, with severe or moderately severe hemophilia A.

Timeline

Start

2007-12

Primary completion

2018-04

Completion

2018-04

Outcome

Met primary endpoint

registry MEAN Moroctocog Alfa (AF-CC), On Demand Cohort: RP Therapy 25 IU/kg: 1.5 (Standard Deviation 2.2) Bleeds per year NCT00543439 ↗

registry p = 0.0020; Ratio of arithmetic means 0.03; Paired t-test NCT00543439 ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Moroctocog alfa	Protein / enzyme biologic	25 iu/kg	—
Subject	Moroctocog alfa	Protein / enzyme biologic	45 iu/kg	—

Indications