NCT02065882

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

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 Fibrinogen Deficiency

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 67 (actual)
• Sponsor: Biotest · Industry
• Sex: All
• Age: 75 Years
• Healthy volunteers: Not accepted

Summary

This was a prospective, open-label, multicenter, phase I/III study investigating the 14-day single-dose pharmacokinetic and pharmacodynamic 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.

Detailed description

The study was divided into two parts (Part I and Part II). Part I focused on the primary endpoint of the study, 14-day single-dose pharmacokinetics (PK) and 14-day single-dose pharmacodynamics (PD), as well as the assessment of maximum clot firmness (MCF) as a surrogate parameter for efficacy. Part I of the study was followed by Part II, which evaluated the efficacy and safety of single and repetitive administration of BT524 in patients undergoing on-demand prophylaxis (ODP) and/or on-demand treatment (ODT) for various bleeding events \[e.g., elective surgery, spontaneous or post-traumatic major bleeding\]. All patients who participated in the PK assessment (Part I) without severe post-administration complications ideally continued treatment with BT524 for ODP and/or ODT in Part II.

Conditions

• Congenital Afibrinogenemia
• Congenital Hypofibrinogenemia

Interventions

Type	Name	Description
DRUG	BT524 (Part I)	Single intravenous infusion of 70 mg BT524 per kg body weight.
DRUG	BT524 (Part II)	Single or repetitive intravenous infusion(s) of BT524, depending on the severity of the disorder, location and extent of the bleeding and patient's clinical condition. Dosage based on individual body weight and fibrinogen level.

Timeline

• Start date: 2013-03-01
• Primary completion: 2020-05-18
• Completion: 2020-11-18
• First posted: 2014-02-19
• Last updated: 2025-07-03
• Results posted: 2025-07-03

Locations

6 sites across 5 countries: Bulgaria, Egypt, Germany, Lebanon, Tunisia