Clinical trial NCT01575756: Pharmacokinetic, Efficacy, and Safety Study of Octafibrin Compared to Haemocomplettan/Riastap
Official title: A Prospective, Controlled, Randomised, Crossover Study Investigating the Pharmacokinetic Properties, Surrogate Efficacy and Safety of Octafibrin Compared to Haemocomplettan® P/RiaSTAPTM in Patients With Congenital Fibrinogen Deficiency
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Congenital Fibrinogen Deficiency
Afibrinogenemia
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2012-04-11
Enrollment: 22
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to investigate pharmacokinetic properties, surrogate efficacy and safety of Octafibrin compared to Haemocomplettan® P/RiaSTAPTM in patients with congenital fibrinogen deficiency


Eligibility criteria: Inclusion Criteria:

* Age ≥ 12 years.
* Documented congenital fibrinogen deficiency (afibrinogenemia).

Exclusion Criteria:

* Life expectancy \> 6 month.
* Bleeding disorder other than congenital fibrinogen deficiency.
* Presence or history of hypersensitivity to study medication.
* Presence or history of deep vein thrombosis or pulmonary embolism within 1 year prior to enrollment.
* Presence or history of arterial thrombosis with 1 year prior to enrollment.
* Hypersensitivity to human plasma products.
* Acute bleeding.
* Pregnant or currently breast-feeding women.
* 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 enrollment.
* Human immunodeficiency virus (HIV) positive with a viral load \> 200 particles/µl or \> 400000 copies/mL.
* End-stage liver disease.
* History of oesophageal varicose bleeding.

Primary outcomes:
measure: Ratio of Octafibrin/FIBRYGA® to Haemocomplettan® P/RiaSTAP(TM) for Fibrinogen Activity Normalized Area Under the Curve Unstandardized
timeFrame: Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
measure: Comparison of Maximum Clot Firmness Between Octafibrin/FIBRYGA® and Haemocomplettan® P/RiaSTAP(TM) at 1 hr Post Infusion
timeFrame: 1 hour post-treatment
Secondary outcomes:
measure: Fibrinogen Activity Normalized Area Under the Curve Unstandardized
timeFrame: Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
measure: Fibrinogen Activity Normalized Area Under the Curve Standardized
timeFrame: Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
measure: Maximum Plasma Concentration Normalized (Cmaxnorm)
timeFrame: Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
measure: Maximum Plasma Concentration (Cmax) Unstandardized
timeFrame: Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
measure: Maximum Plasma Concentration (Cmax) Standardized
timeFrame: Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
measure: Incremental in Vivo Recovery
timeFrame: Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
measure: Classical in Vivo Recovery
timeFrame: Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
measure: Time to Reach Maximum Plasma Concentration (Tmax)
timeFrame: Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
measure: Terminal Half-life (t½)
timeFrame: Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
measure: Mean Residence Time (MRT)
timeFrame: Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
measure: Clearance
timeFrame: Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
measure: Volume of Distribution at Steady State (Vss)
timeFrame: Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
Other outcomes:
Locations:
city: Aurora
country: United States
facility: University of Colorado Hemophilia & Thrombosis Center
state: Colorado
city: New Hyde Park
country: United States
facility: Cohen Children's Medical Center of New York
state: New York
city: Sofia
country: Bulgaria
facility: Specialized Hospital for Active Treatment "Joan Pavel"
city: Bangalore
country: India
facility: Department of Hematology St. John's Medical College Hospital
city: Prune
country: India
facility: Sahyadri Speciality Hospital
city: Vellore
country: India
facility: Department of Hematology Christian Medical College
city: Shiraz
country: Iran, Islamic Republic of
facility: Nemazee Hospital Shiraz University of Medical Sciences
city: Tehran
country: Iran, Islamic Republic of
facility: Tehran University of Medical Sciences
city: Zurich
country: Switzerland
facility: Department of Hematology University Hospital
city: London
country: United Kingdom
facility: The Centre for Haemostatis and Thrombosis
Study officials:
affiliation: Octapharma
name: Sigurd Knaub, PhD
role: STUDY_DIRECTOR
References: