Pharmacokinetic, Efficacy, and Safety Study of Octafibrin Compared to Haemocomplettan/Riastap
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
1 other identifier
interventional
22
6 countries
10
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jun 2013
10 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 9, 2012
CompletedFirst Posted
Study publicly available on registry
April 11, 2012
CompletedStudy Start
First participant enrolled
June 1, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 1, 2015
CompletedStudy Completion
Last participant's last visit for all outcomes
January 1, 2015
CompletedResults Posted
Study results publicly available
November 30, 2016
CompletedMarch 9, 2018
March 1, 2018
1.6 years
April 9, 2012
October 5, 2016
March 6, 2018
Conditions
Outcome Measures
Primary Outcomes (2)
Ratio of Octafibrin/FIBRYGA® to Haemocomplettan® P/RiaSTAP(TM) for Fibrinogen Activity Normalized Area Under the Curve Unstandardized
Fibrinogen activity was determined via a validated Clauss assay (fibrinogen activity) and fibrinogen-specific enzyme-linked immunosorbent assay (ie, fibrinogen antigen) using paired antibodies for fibrinogen antigen. All determinations were performed on frozen plasma samples in a central laboratory. The Clauss assay was modified and validated to achieve a limit of quantification of 0.2 g/L. The pharmacokinetic analysis was assessed individually using a non-compartmental model. Plasma levels were measured at Baseline, and at 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment. The mean ratio of normalized area under the curve was calculated as Octafibrin/FIBRYGA® over Haemocomplettan® P/RiaSTAP(TM)
Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
Comparison of Maximum Clot Firmness Between Octafibrin/FIBRYGA® and Haemocomplettan® P/RiaSTAP(TM) at 1 hr Post Infusion
Thromboelastometry (ROTEM®) was used to measure maximum clot firmness. Thromboelastometry is a method for the continuous measurement of clot formation. Maximum clot firmness is a functional parameter that depends on the activation of coagulation, the platelet and fibrinogen content of the blood sample, and the polymerisation and cross-linking of the fibrin network. In order to obtain comparable results from all study centres, maximum clot firmness data were assessed from frozen citrated plasma samples in a central laboratory. As these samples did not contain platelets that would be found in the whole blood assay, the fibrinogen content primarily defined the maximum clot firmness.
1 hour post-treatment
Secondary Outcomes (12)
Fibrinogen Activity Normalized Area Under the Curve Unstandardized
Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
Fibrinogen Activity Normalized Area Under the Curve Standardized
Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
Maximum Plasma Concentration Normalized (Cmaxnorm)
Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
Maximum Plasma Concentration (Cmax) Unstandardized
Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
Maximum Plasma Concentration (Cmax) Standardized
Baseline to 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 216, and 312 hours post-treatment
- +7 more secondary outcomes
Study Arms (2)
Octafibrin followed by Haemocomplettan® P or RiaSTAPTM
EXPERIMENTALParticipants received Octafibrin 70 mg/kg intravenously once followed by Haemocomplettan® P or RiaSTAPTM 70 mg/kg intravenously once 45 days later.
Haemocomplettan® P or RiaSTAPTM followed by Octafibrin
EXPERIMENTALParticipants received Haemocomplettan® P or RiaSTAPTM 70 mg/kg intravenously once followed by Octafibrin 70 mg/kg intravenously once 45 days later.
Interventions
Octafibrin was supplied as a powder for reconstitution with water for injection.
Commercially available Haemocomplettan® P or RiaSTAPTM (same product with different names in different markets) were supplied as powders for reconstitution with water for injection.
Eligibility Criteria
You may qualify if:
- Age ≥ 12 years.
- Documented congenital fibrinogen deficiency (afibrinogenemia).
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Octapharmalead
Study Sites (10)
University of Colorado Hemophilia & Thrombosis Center
Aurora, Colorado, 80045, United States
Cohen Children's Medical Center of New York
New Hyde Park, New York, 11040, United States
Specialized Hospital for Active Treatment "Joan Pavel"
Sofia, Bulgaria
Department of Hematology St. John's Medical College Hospital
Bangalore, India
Sahyadri Speciality Hospital
Prune, India
Department of Hematology Christian Medical College
Vellore, India
Nemazee Hospital Shiraz University of Medical Sciences
Shiraz, Iran
Tehran University of Medical Sciences
Tehran, Iran
Department of Hematology University Hospital
Zurich, Switzerland
The Centre for Haemostatis and Thrombosis
London, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Director of Clinical Operations
- Organization
- Octapharma USA
Study Officials
- STUDY DIRECTOR
Sigurd Knaub, PhD
Octapharma
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 9, 2012
First Posted
April 11, 2012
Study Start
June 1, 2013
Primary Completion
January 1, 2015
Study Completion
January 1, 2015
Last Updated
March 9, 2018
Results First Posted
November 30, 2016
Record last verified: 2018-03