“Prospective, open-label, uncontrolled, Phase III study to assess the efficacy and safety of Octafibrin for on-demand treatment of acute bleeding and to prevent bleeding during and after surgery in subjects with congenital fibrinogen deficiency”
Objectives:
To demonstrate the efficacy and safety of Octafibrin for on-demand treatment of acute bleeding episodes and to prevent bleeding (spontaneous or after trauma) during and after surgery in subjects with congenital fibrinogen deficiency”
Inclusion criteria:
Aged ≥12 years ; Documented diagnosis of congenital fibrinogen deficiency, expected to require on-demand treatment for bleeding or surgical prophylaxis :Fibrinogen deficiency manifested as afibrinogenaemia or severe hypofibrinogenaemia; Expected to have an acute bleeding episode (spontaneous or after trauma) or planning to undergo elective surgery.
Exclusion criteria: Life expectancy <6 months ;Other Congenital Bleeding disorders ; Prophylactic treatment with a fibrinogen concentrate ;Treatment with any fibrinogen concentrate or other fibrinogen-containing blood product within 2 weeks prior to start of treatment for the bleeding episode or surgery.
Treatment with any coagulation-active drug (within 1 week prior to 24 hours after start of treatment
Population: 2-4 patient more than 12 ages with congenital Afibrinogenemia
Design:Prospective ; open-label, uncontrolled, Phase III
Intervention: assessment of efficacy and safety of Octafibrin and till 30 days after injection
Primary endpoint:
The primary endpoint is the overall clinical assessment of the haemostatic efficacy of Octafibrinin treating the first documented bleeding episode of each patient
General information
Acronym
IRCT registration information
IRCT registration number:IRCT2014112420051N4
Registration date:2015-09-24, 1394/07/02
Registration timing:registered_while_recruiting
Last update:
Update count:0
Registration date
2015-09-24, 1394/07/02
Registrant information
Name
Mehran Karimi
Name of organization / entity
Hematology Research Center, Shiraz University of Medical Sciences
Country
Iran (Islamic Republic of)
Phone
+98 71 3612 5617
Email address
karimim@sums.ac.ir
Recruitment status
Recruitment complete
Funding source
Octapharma Company
Expected recruitment start date
2015-09-20, 1394/06/29
Expected recruitment end date
2015-12-20, 1394/09/29
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
“Prospective, open-label, uncontrolled, Phase III study to assess the efficacy and safety of Octafibrin for on-demand treatment of acute bleeding and to prevent bleeding during and after surgery in subjects with congenital fibrinogen deficiency”
Public title
The assessment of the efficacy and safety of Fibrinogen ( Octafibrin) in subjects with congenital fibrinogen deficiency”
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Aged ≥12 years ; Documented diagnosis of congenital fibrinogen deficiency, expected to require on-demand treatment for bleeding or surgical prophylaxis :Fibrinogen deficiency manifested as afibrinogenaemia or severe hypofibrinogenaemia; Expected to have an acute bleeding episode (spontaneous or after trauma) or planning to undergo elective surgery.
Exclusion criteria: Life expectancy <6 months ;Other Congenital Bleeding disorders ; Prophylactic treatment with a fibrinogen concentrate ;Treatment with any fibrinogen concentrate or other fibrinogen-containing blood product within 2 weeks prior to start of treatment for the bleeding episode or surgery.
Treatment with any coagulation-active drug (within 1 week prior to 24 hours after start of treatment
Age
From 12 years old
Gender
Both
Phase
3
Groups that have been masked
No information
Sample size
Target sample size:
24
Randomization (investigator's opinion)
N/A
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Single
Other design features
Secondary Ids
1
Registry name
Efficacy and Safety Study of Octafibrin for On-demand Treatment of Acute Bleeding and Prevention Bleeding During and After Surgery
Secondary trial Id
NCT02267226
Registration date
empty
Ethics committees
1
Ethics committee
Name of ethics committee
Shiraz University of Medical Science
Street address
inside HELAL AHMAR Building; Zand Avenue
City
SHIRAZ
Postal code
Approval date
2015-08-02, 1394/05/11
Ethics committee reference number
IR.SUMS.REC.1394.76
2
Ethics committee
Name of ethics committee
Added at 2017-11-04: Ethics committee of Isfahan University of Medical Sciences
Street address
Added at 2017-11-04:
City
Added at 2017-11-04: Isfahan
Postal code
Added at 2017-11-04:
Approval date
2016-11-25, 1395/09/05
Ethics committee reference number
Added at 2017-11-04: p/12/4/M/277
Health conditions studied
1
Description of health condition studied
congenital Afibrinogenemia
ICD-10 code
D68.2
ICD-10 code description
Hereditary deficiency of other clotting factors
Primary outcomes
1
Description
the overall clinical assessment of the haemo¬static efficacy of Octafibrin in treating the first documented bleeding episode of each patient
Timepoint
24 hrs after up to 30 days
Method of measurement
the assessment made by the investigator on the 4-point rating scale
Secondary outcomes
1
Description
Clot strength (MCF)
Timepoint
at pre infusion and at 1 hour after first infusion up to 30 days
Method of measurement
changes of MCF from pre infusion will be summarised using descriptive statistics and displayed graphically؛ a correlation analysis between haemostatic efficacy assessment
Intervention groups
1
Description
the administration of Octafibrin for bleeding
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Shiraz University of Medical Science
Full name of responsible person
MEHRAN KARIMI
Street address
City
SHIRAZ
2
Recruitment center
Name of recruitment center
Added at 2016-11-27: Seyed al shohada Hospital
Full name of responsible person
Added at 2016-11-27: Dr. Hamid Hoodfar
Street address
Added at 2016-11-27: Motahari st
City
Added at 2016-11-27: Isfahan
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Octapharma AG
Full name of responsible person
Flora Peyvandi
Street address
A. Bianchi Bonomi Hemophilia and Thrombosis Center, University of Milan, Department of Medicine and Medical Specialties, IRCCS Maggiore Hospital, Mangiagalli and Regina Elena Foundation, Via Pace 9 20122
City
Milan
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?