A controlled, randomised, study investigating the pharmacokinetic properties (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

• Register: EUCTR
• Last refreshed on: 23 December 2013
• Main ID: EUCTR2011-002403-15-IT
• Date of registration: 15/04/2013
• Prospective Registration: Yes
• Primary sponsor: OCTAPHARMA AG
• Public title: A controlled, randomised, study investigating the pharmacokinetic properties (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
• Scientific title: A prospective, controlled, randomised, cross-over study investigating the pharmacokinetic properties, surrogate efficacy and safety of Octafibrin compared to Haemocomplettan® P/ RiaSTAPTM in subjects with congenital fibrinogen deficiency
• Date of first enrolment: 14/05/2013
• Target sample size: 18
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-002403-15
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: no Cross over: yes Other: no If controlled, specify comparator, Other Medicinial Product: yes Placebo: no Other: no Number of treatment arms in the trial: 2

Countries of recruitment

Bulgaria, Germany, Iran, Islamic Republic of, Italy, Spain, Turkey, United Kingdom, United States

Contacts

• Name: PROJECT MONITORING
• Affiliation: BIOIKOS FARMA S.R.L.

• Name: PROJECT MONITORING
• Affiliation: BIOIKOS FARMA S.R.L.

Key inclusion & exclusion criteria

Inclusion criteria:
• 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: 6
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 6
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 6

Exclusion criteria:
• 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 an 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
? Suspicion of an anti-fibrinogen inhibitor as indicated by previous in-vivo recovery, if available ? Blood or plasma donation in the 3 months prior to enrolment



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

congenital fibrinogen deficiency

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

Product Name: octafibrin
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: HUMAN FIBRINOGEN
CAS Number: 9001-32-5
Other descriptive name: HUMAN FIBRINOGEN
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 70-

Trade Name: Haemocomplettan® P
Product Name: Haemocomplettan® P
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: HUMAN FIBRINOGEN
CAS Number: 9001-32-5
Other descriptive name: HUMAN FIBRINOGEN
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 70-

Primary Outcome(s)

Main Objective: • To determine the single dose pharmacokinetics of Octafibrin and Haemocomplettan® P/RiaSTAPTM in subjects with congenital fibrinogen deficiency
• 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

Timepoint(s) of evaluation of this end point: at baseline, 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216 and 312 hours postinfusion.

Primary end point(s): A comparison of the AUC between Octafibrin and Haemocomplettan®
P/RiaSTAPTM
Surrogate endpoint for haemostatic efficacy
Comparison of MCF between Octafibrin and Haemocomplettan®
P/RiaSTAPTM at 1 hour post-infusion

Secondary Objective: To assess the safety of Octafibrin in subjects with congenital fibrinogen deficiency

Secondary Outcome(s)

Secondary end point(s): • To compare the in vivo recovery between Octafibrin and
Haemocomplettan® P/ RiaSTAPTM
• To compare the pharmacokinetics between Octafibrin and
Haemocomplettan® P/ RiaSTAPTM
• To document the safety of Octafibrin

Timepoint(s) of evaluation of this end point: at baseline, 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216 and 312 hours postinfusion.

Secondary ID(s)

2011-002403-15-GB

Forma-01

Source(s) of Monetary Support

OCTAPHARM