Clinical Trial: Pharmacokinetics, Efficacy, and Safety of Human Plasma-Derived Fibrinogen (FIB Grifols) in Patients With Congenital Afibrinogenaemia

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

Official Title: Multicenter, Prospective, Open-Label, Single-Arm Trial to Evaluate the Pharmacokinetics, Efficacy, and Safety of Human Plasma-Derived Fibrinogen (FIB Grifols) in Patients With Congenital This study is a Phase I-II, multi-center, prospective, open-label, single-arm, clinical trial to evaluate PK, efficacy, and safety of human plasma-derived fibrinogen concentrate FIB Grifols in adult and pediatric patients with congenital afibrinogenaemia.

Approximately 10adult subjects (≥ 18 years) with congenital afibrinogenaemia will be administered with a single dose of study drug at 70 mg/kg body weight. and will be followed for PK, efficacy, and safety assessments.

After the safety of fibrinogen concentrate FIB Grifols is assessed in at least 10 adult subjects and no safety issues are raised by the sponsor, the study will start to enroll approximately 10 pediatric subjects who will be dosed with study drug and followed for PK, efficacy, and safety assessments.

All enrolled subjects (both adult and pediatrics) will have documented congenital fibrinogen deficiency manifested as afibrinogenaemia but will not have received any fibrinogen-containing product therapy within the preceding 21 days before the infusion of study drug.

All subjects (both adult and pediatrics) will be infused with the investigational product at 70 mg/kg body weight. PK parameters that will be calculated from plasma fibrinogen levels measured at different time points] include: in vivo recovery [IVR], area under the curve (AUC) calculated as AUC from zero to 14 days (AUC0-14days) and AUC from zero to infinity (AUC0-∞), maximum plasma concentration (Cmax), time to the observed maximum plasma concentration (tmax), half-life (t1/2), mean residence time (MRT), volume of distribution (Vd), and clearance (Cl).

Hemostatic efficacy of the investigational product will be assessed by means of thromboe
Sponsor: Instituto Grifols, S.A.

Current Primary Outcome: Change in MCF measured by rotational thromboelastography (ROTEM). [ Time Frame: Baseline to one hour post-infusion ]

MCF, as a functional parameter of blood's ability to coagulate, provides an indirect measure of hemostatic efficacy of replacement treatment with fibrinogen concentates in patients with fibrinogen deficiency


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Difference (improvement) in Clotting time (CT) [ Time Frame: Baseline to one hour post-infusion ]
  • Difference (improvement) in Clot Formation Time (CFT) [ Time Frame: Baseline to one hour post-infusion ]
  • Difference (improvement) in Alpha angle (α) [ Time Frame: Baseline to one hour post-infusion ]
  • Difference (improvement) in Prothrombin tme (PT) [ Time Frame: Baseline to one hour post-infusion ]
  • Difference (improvement) in Thrombin tme (TT) [ Time Frame: Baseline to one hour post-infusion ]
  • Difference (improvement) in Activated Partial Thromboplastin Time (aPTT) [ Time Frame: Baseline to one hour post-infusion ]


Original Secondary Outcome: Same as current

Information By: Grifols Biologicals Inc.

Dates:
Date Received: October 24, 2014
Date Started: May 2016
Date Completion: January 2018
Last Updated: July 21, 2016
Last Verified: July 2016