Clinical Trial: Study to Assess the Efficacy, Safety and Pharmacokinetic of Octafibrin in Paediatric Subjects With Fibrinogen Deficiency

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

Official Title: Prospective, Open-label, Uncontrolled, Phase III Study to Assess the Efficacy, Safety and Pharmacokinetic of Octafibrin for On-demand Treatment of Acute Bleeding and to Prevent Bleeding During and Aft

Brief Summary:

This study will assess the efficacy of Octafibrin, a fibrinogen concentrate in in the on-demand treatment of spontaneous or traumatic bleeding episodes in paediatric patients less than 12 years of age.The planned study duration is up to 5 years. The study will be considered completed when a minimum of 6 subjects (i.e., at least 3 subjects aged between 0 and <6 years and 3 subjects aged between 6 and <12 years) have at least one documented bleeding episode and when in total a minimum of 2 surgical procedures have been performed.

All patients will undergo a pharmacokinetic (PK) study after screening. This will have a duration of 14 days, after which a patient can be treated for a bleeding episode or planned surgical procedure when they occur.


Detailed Summary:
Sponsor: Octapharma

Current Primary Outcome: Assess efficacy of Octafibrin for on-demand treatment of acute bleeding episodes (spontaneous or traumatic) [ Time Frame: 5 years ]

The efficacy will be assessed by the investigator using a 4-point ordinary efficacy scale (excellent, good, moderate, none)


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • terminal half-life of Octafibrin [ Time Frame: 5 years ]
    Determine pharmacokinetics of Octafibrin: Determine the terminal half-life of Octafibrin in paediatric subjects
  • area under the curve of Octafibrin [ Time Frame: 5 years ]
    Determine pharmacokinetics of Octafibrin: Determine the area under the curve of Octafibrin in paediatric subjects
  • Investigate the association between clinical hemostatic efficacy and MCF (maximum clot firmness) via thromboelastometry (ROTEM) [ Time Frame: 5 years ]
    To investigate an association between the overall clinical assessment of haemostatic efficacy and the surrogate endpoint 'clot strength' or 'clot firmness' (referred to as 'maximum clot firmness' [MCF] in this protocol) via thromboelastometry (ROTEM). Therefore, MCF as surrogate efficacy parameter will be determined before and after the first infusion of IMP for treatment of a bleeding episode.
  • Determine peak target plasma fibrinogen levels in minor and major bleedings [ Time Frame: 5 years ]
    To achieve a peak target plasma fibrinogen level of 100 mg/dL in minor bleeds and 150 mg/dL for major bleeds 1 hour post-infusion.
  • Determine response to Octafibrin based on incremental in vivo recovery [ Time Frame: 5 years ]
    To determine the response to Octafibrin based on incremental in vivo recovery (IVR) by measuring the Fibrinogen plasma level pre- and 1 hour and 3 hours post infusion.
  • Assess efficacy of Octafibrin in preventing bleeding during and after surgery [ Time Frame: 5 years ]
    The efficacy of Octafibrin in preventing bleeding during and after surgery will be assess using a 4-point hemostatic efficacy scale (excellent, good, moderate, none).
  • Number of subjects with (serious) adverse events [ Time Frame: 5 years ]
    Assess the safety of Octafibrin in subject with fibrinogen deficiency. To assess the safety of Octafibrin in subjects with congenital fibrinogen deficiency. Number of subjects with (serious) adverse events, including immunogenicity, thromboembolic complications, and early signs of allergic or hypersensitivity reactions.


Original Secondary Outcome: Same as current

Information By: Octapharma

Dates:
Date Received: March 25, 2015
Date Started: December 2015
Date Completion: October 2020
Last Updated: May 4, 2016
Last Verified: May 2016