A controlled, randomised, study investigating the pharmacokineticproperties (to see how active the study drug is in your blood and how long it takes for the study drug to get out of your blood) , surrogate efficacy and safety of Octafibrin compared to Haemocomplettan® P/ RiaSTAPTM in subjects with congenital fibrinogen deficiency
- Conditions
- MedDRA version: 14.1Level: LLTClassification code 10052651Term: AfibrinogenaemiaSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Congenital fibrinogen deficiency (afibrinogenaemia) defined as plasma fibrinogen activity and antigen at screening below detection limit (i.e. <20mg/dl)
- Registration Number
- EUCTR2011-002403-15-DE
- Lead Sponsor
- OCTAPHARMA AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 22
•Subject age =12 years
•Documented congenital fibrinogen deficiency (afibrinogenaemia)
Plasma fibrinogen activity and antigen at screening bellow detection limit (i.e. <20mg/dl)
•Informed consent signed by subject or legal guardian
Are the trial subjects under 18? yes
Number of subjects for this age range: 2
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 2
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2
•Life expectancy <6 months
•Bleeding disorder other than congenital fibrinogen deficiency
•Dysfibrinogenemia
•Treatment with:
Any fibrinogen concentrate or other fibrinogen-containing blood product in the 2 weeks prior to enrolment
•Presence or history of:
Hypersensitivity to study medication
Deep vein thrombosis or pulmonary embolism within 1 year prior to enrolment
Arterial thrombosis within 1 year prior to enrolment
Hypersensitivity to human plasma proteins
•Acute bleeding
•History of oesophageal varicose bleeding
•End-stage liver disease (i.e. Child-Pugh-score B or C)
•Planned major surgery with a need for blood transfusion during the PK blood-sampling period of this study
•Pregnancy, or the intention to become pregnant during the study
•Currently breast-feeding, or with the intention of breast-feeding during the study
•HIV positive with a viral load >200 particles/µl ~ >400000 copies/ml
•Polytrauma 1 year prior to enrolment
• Blood or plasma donation in the 3 months prior to enrolment
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: •To determine the single dose pharmacokinetics of Octafibrin and Haemocomplettan® P/RiaSTAPTM in subjects with congenital fibrinogen deficiency<br>•To determine maximum clot strength (maximum clot firmness [MCF]) as a surrogate marker for haemostatic efficacy before and after administration of Octafibrin and Haemocomplettan® P/ RiaSTAPTM in subjects with congenital fibrinogen deficiency<br>;Secondary Objective: To assess the safety of Octafibrin in subjects with congenital fibrinogen deficiency;Primary end point(s): A comparison of the AUC between Octafibrin and Haemocomplettan® P/RiaSTAPTM<br>Surrogate endpoint for haemostatic efficacy<br>Comparison of MCF between Octafibrin and Haemocomplettan® P/RiaSTAPTM at 1 hour post-infusion<br>;Timepoint(s) of evaluation of this end point: At baseline, 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216 and 312 hours post-infusion.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): •To compare the in vivo recovery between Octafibrin and Haemocomplettan® P/ RiaSTAPTM<br>•To compare the pharmacokinetics between Octafibrin and Haemocomplettan® P/ RiaSTAPTM<br>•To document the safety of Octafibrin<br>;Timepoint(s) of evaluation of this end point: At baseline, 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216 and 312 hours post-infusion.