Prospective,open-label,uncontrolled,phase III study to assess the efficacy, safety and pharmacokinetics of Octafibrin for on-demand treatment of acute bleeding and to prevent bleeding during and after surgery in paediatric subjects with congenital fibrinogen deficiency
Goal:To demonstrate the efficacy and safety of Octafibrin for on-demand treatment of acute bleeding episodes (spontaneous or after trauma) .
Study design:multinational, multicentre. prospective,open-label,uncontrolled,Phase III study.
Number of subjects:3 subjects between 6-12 years old with congenital afibrinogenemia
Inclusion:
age 6-12; documented diagnosis of congenital fibrinogen deficiency; Historical plasma fibrinogen activity of <50ml/dl; expected to have an acute bleeding episode(spontaneous or after trauma) or planing to undergo elective surgery; Informed consent signed by the subject's legal guardian
Exclusion:
Life expectancy<6 months; bleeding disorder other than congenital fibrinogen dificiency; prophylactic treatment with a fibrinogen concentrate; any fibrinogen concentrate or other fibrinogen-containing blood product within 2 weeks prior to start of treatment for the PK phase,a bleeding episode, or surgery; any coagulation drug within 1 week prior to start of PK phase or treatment for the bleeding episode or surgery, or as a planned or expected medication during the time period from Day 1 until 24 hours presence or history of hypersensitivity to study medication,DVT or pulmonary embolism, arterial thrombosis within 1 year prior to start drug infusion,hypersensitivity to human plasma protein,Oesophageal varicose bleeding
intervention: infusion of Octafibrin and monitoring
Duration:30 days after infusion
primary endpoint:the overall clinical assessment of the haemostatic efficacy of octafibrin in treating the first documented bleeding episode of each patient
General information
Acronym
IRCT registration information
IRCT registration number:IRCT2014112320051N3
Registration date:2015-09-01, 1394/06/10
Registration timing:prospective
Last update:
Update count:0
Registration date
2015-09-01, 1394/06/10
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 AG
Expected recruitment start date
2015-09-23, 1394/07/01
Expected recruitment end date
2015-12-22, 1394/10/01
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, safety and pharmacokinetics of Octafibrin for on-demand treatment of acute bleeding and to prevent bleeding during and after surgery in paediatric subjects with congenital fibrinogen deficiency
Public title
The effect of haemostatic factor,Octafibrin in congenital afibrinogenemia
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion:
age 6-12; documented diagnosis of congenital fibrinogen deficiency; Historical plasma fibrinogen activity of <50ml/dl; expected to have an acute bleeding episode(spontaneous or after trauma) or planing to undergo elective surgery; Informed consent signed by the subject's legal guardian
Exclusion:
Life expectancy<6 months; bleeding disorder other than congenital fibrinogen dificiency; prophylactic treatment with a fibrinogen concentrate; any fibrinogen concentrate or other fibrinogen-containing blood product within 2 weeks prior to start of treatment for the PK phase,a bleeding episode, or surgery; any coagulation drug within 1 week prior to start of PK phase or treatment for the bleeding episode or surgery, or as a planned or expected medication during the time period from Day 1 until 24 hourspresence or history of hypersensitivity to study medication,DVT or pulmonary embolism, arterial thrombosis within 1 year prior to start drug infusion,hypersensitivity to human plasma protein,Oesophageal varicose bleeding
Age
From 6 years old to 12 years old
Gender
Both
Phase
3
Groups that have been masked
No information
Sample size
Target sample size:
3
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
EudraCT
Secondary trial Id
2014-005115-16
Registration date
2017-11-21, 1396/08/30
Ethics committees
1
Ethics committee
Name of ethics committee
Ethic Committee of Shiraz University of Medical Sciences
Street address
Next to Helal Ahmar-Zand Avenue-Shiraz
City
Shiraz
Postal code
Approval date
2015-08-05, 1394/05/14
Ethics committee reference number
IR.SUMS.REC.1394.76
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
overall clinical assessment of the haemostatic efficacy of Octafibrin
Timepoint
24 hours
Method of measurement
4-point haemostatic efficacy scale
Secondary outcomes
1
Description
‘clot strength’ or ‘clot firmness’
Timepoint
1 hour
Method of measurement
blood sampling
Intervention groups
1
Description
Infusion of Octafibrin
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Seyedo Shohada Isfahan Hospital
Full name of responsible person
Dr. Mehran Karimi
Street address
27, jahannema bldg, 10th alley
City
Shiraz
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Octapharma
Full name of responsible person
Bruce A. Schwartz
Street address
USA, Inc. | 121 River Street, Suite 1201 | Hoboken, New Jersey 07030 |
City
Hoboken
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Octapharma
Proportion provided by this source
100
Public or private sector
empty
Domestic or foreign origin
empty
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
empty
Person responsible for general inquiries
Contact
Name of organization / entity
Shiraz University of Medical Sciences, Hematology Research Center
Full name of responsible person
Mehran Karimi
Position
Sub-special in pediatrics hematology and oncology
Other areas of specialty/work
Street address
Mohammad Rasoolallah research Tower, Khalili Street
City
Shiraz
Postal code
Iran
Phone
+98 71 3628 1563
Fax
Email
karimim@sums.ac.ir
Web page address
Person responsible for scientific inquiries
Contact
Name of organization / entity
Shiraz University of Medical Sciences
Full name of responsible person
Mehran Karimi
Position
Professor of Pediatric Hematology-Oncology Hematology Research Center
Other areas of specialty/work
Street address
Hematology Research Center, Nemazee Hospital, Zand street, Shiraz
City
Shiraz
Postal code
Phone
00
Fax
Email
karimim@sums.ac.ir
Web page address
Person responsible for updating data
Contact
Name of organization / entity
Shiraz university of Medical Sciences, Hematology Research Center
Full name of responsible person
Zohreh Zahedi
Position
BS, Research
Other areas of specialty/work
Street address
Mohammad rasoollalah research Tower, Khalili Street
City
Shiraz
Postal code
Phone
00
Fax
Email
Web page address
Sharing plan
Deidentified Individual Participant Data Set (IPD)