Clinical Trial: Pharmacokinetic, Efficacy and Safety of BT524 in Patients With Congenital Fibrinogen Deficiency

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Prospective, Open-label, Phase I/III Study Investigating Pharmacokinetic Properties of BT524 and Efficacy and Safety of BT524 in the Treatment and Prophylaxis of Bleeding in Patients With Congenital

Brief Summary: The purpose of the study is to investigate pharmacokinetics, efficacy and safety of BT524 in patients with congenital fibrinogen deficiency.

Detailed Summary: The present study is designed as a prospective, open-label, multicentre, phase I/III study investigating the 14 day single-dose pharmacokinetic properties, efficacy and safety of BT524 following intravenous administration in the treatment or prophylaxis of bleeding in patients with congenital afibrinogenemia or severe congenital hypofibrinogenemia.
Sponsor: Biotest

Current Primary Outcome: Terminal Elimination Half-life (t1/2) for fibrinogen antigen, [ Time Frame: Prior to the initial dose on day 1, at the end of the infusion and 0.5, 1, 2, 4, 8 hours post dose, 24, 48,96, 168, 240h and 336 hours post-dose ]

The primary objective of this study is to investigate the 14 day single-dose pharmacokinetics of BT524 following intravenous (IV) infusion in patients with congenital afibrinogenemia or severe congenital hypofibrinogenemia (part I).


Original Primary Outcome: Same as current

Current Secondary Outcome: Terminal Elimination Half-life (t1/2) for fibrinogen activity [ Time Frame: Prior to the initial dose on day 1, at the end of the infusion and 0.5, 1, 2, 4, 8 hours post dose, 24, 48,96, 168, 240 and 336 hours post-dose ]

Secondary objectives are to investigate the 14 day single-dose pharmacodynamics of BT524, and the surrogate efficacy and safety of BT524 in part I of the study. In addition, the hemostatic efficacy, surrogate efficacy, and safety of single and/or repetitive administrations for on-demand prophylaxis (ODP) and/or on-demand treatment (ODT) of bleeding events will be investigated.


Original Secondary Outcome: Same as current

Information By: Biotest

Dates:
Date Received: February 3, 2014
Date Started: March 2013
Date Completion: October 2018
Last Updated: June 2, 2016
Last Verified: June 2016