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Inhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) when Exposed to plasma-derived von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates

Protocol summary

Summary
Patients enrolled in the trial will be treated for the first 50 EDs or for the first 3 years from enrolment, whichever comes first, with either a single plasma-derived FVIII concentrate containing von Willebrand factor or a single rFVIII concentrate as assigned by the randomisation. Treatment with FVIII products other than that initially prescribed will represent a reason for withdrawal. The therapeutic regimen for on-demand treatment will be at the discretion of the physician, even though a dose of 25-40 IU/Kg is recommended, to repeat until complete resolution of the episode, or 40 IU/Kg at bleeding occurrence, followed by 20 IU/Kg at 24 and 72 hours. The dosage for prophylaxis will be at the discretion of the physician. The suggested regimen for standard prophylaxis is 25-40 IU/Kg, 3-4 times a week. For the sake of the analysis, prophylaxis is defined as at least 2 infusions weekly on non-consecutive days with 20 IU/Kg or more. Any other regular treatment will be defined as modified prophylaxis. Patients will be monitored for inhibitor development by Bethesda assay with Nijmegen modification locally and at the central laboratory in case of treatment failure or every 3 to 4 EDs for the first 20 EDs and every 10 EDs thereafter, or every 3 months, whichever come first, and at any time inhibitor development would be clinically suspected. Patients on prophylaxis will be tested every 2 weeks. In case of suspect inhibitor development, inhibitor levels must be assessed locally (a second aliquot will be stored in the refrigerator) and confirmed both locally and at the central laboratory within 14 days after the first positive test and every month for the following 6 months.

General information

Acronym
SIPPET
IRCT registration information
IRCT registration number: IRCT201104106161N1
Registration date: 2011-04-30, 1390/02/10
Registration timing: registered_while_recruiting

Last update:
Update count: 0
Registration date
2011-04-30, 1390/02/10
Registrant information
Name
Bahman Vasseghi
Name of organization / entity
Clinart
Country
United Arab Emirates
Phone
0097144370551
Email address
bahman.vasseghi@clinart.net
Recruitment status
Recruitment complete
Funding source
Fondazione Angelo Bianchi Bonomi- Italy
Expected recruitment start date
2009-04-01, 1388/01/12
Expected recruitment end date
2014-03-31, 1393/01/11
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Inhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) when Exposed to plasma-derived von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates
Public title
Survey of Inhibitors in Plasma-Product Exposed Toddlers
Purpose
Prevention
Inclusion/Exclusion criteria
Inclusion criteria: 1-Male subjects; 2-Any ethnicity; 3-Age <6 years; 4-Severe haemophilia A (FVIII:C <1%), as confirmed by the central laboratory; 5-Previously untreated (0 EDs to any FVIII concentrate or blood products) or minimally treated (<5 EDs) with blood components, namely whole blood, fresh frozen plasma, packed red blood cells, platelets or cryoprecipitate; 6-Negative inhibitor measurement at both local and central laboratory at screening; 7-Ability to comply with study requirements; 8-Signed informed consent of legal tutors. Exclusion criteria: 1-Plasma FVIII level ≥1%, as assayed at the central laboratory; 2-Previous history of FVIII inhibitor; 3-Other congenital or acquired bleeding defects; 4-Concomitant congenital or acquired immunodeficiency; 5-Concomitant treatment with systemic immunosuppressive drugs; 6-Concomitant treatment with any investigational drug.
Age
To 6 years old
Gender
Male
Phase
4
Groups that have been masked
No information
Sample size
Target sample size: 300
Randomization (investigator's opinion)
Randomized
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Parallel
Other design features
Non-Profit, Investigator-initiated, Multicentre and international

Secondary Ids

empty

Ethics committees

1

Ethics committee
Name of ethics committee
National Ethics Committee
Street address
Deputy of research and technology- ministry of health
City
Tehran
Postal code
Approval date
2010-02-02, 1388/11/13
Ethics committee reference number
رپ88 ص - 790

Health conditions studied

1

Description of health condition studied
Hemophilia A
ICD-10 code
D66
ICD-10 code description
Hereditary factor VIII deficiency

Primary outcomes

1

Description
To assess the immunogenicity of plasma derived VWF/FVIII and rFVIII concentrates
Timepoint
in the first 50 EDs or in the first 3 years from enrolment
Method of measurement
by determining the frequency of inhibitor development

Secondary outcomes

1

Description
To evaluate modality of occurrence of inhibitors (number of EDs, titre at onset, etc)
Timepoint
in the first 50 EDs or in the first 3 years from enrolment, whichever comes first
Method of measurement
record and analysis of characteristics during the treatment course

Intervention groups

1

Description
Recombinant Factor VIII concentrate
Category
Treatment - Drugs

2

Description
plasma-derived Factor VIII concentrate
Category
Treatment - Drugs

Recruitment centers

1

Recruitment center
Name of recruitment center
comprehensive care centre for children with hemophilia
Full name of responsible person
Dr. Peyman Eshghi
Street address
Mofid children hospital
City
Tehran

2

Recruitment center
Name of recruitment center
Hemophilia treatment centre
Full name of responsible person
Dr. Mehran Karimi
Street address
Shahid Dastgheib Hospital
City
Shiraz

Sponsors / Funding sources

1

Sponsor
Name of organization / entity
Fondazione Angelo Bianchi Bonomi
Full name of responsible person
Barbara Bianchi Bonomi
Street address
Piazza Castello, 2 20121
City
Milano
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Fondazione Angelo Bianchi Bonomi
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
Clinart
Full name of responsible person
Dr. Bahman Vasseghi
Position
Study manager-Iran/MD
Other areas of specialty/work
Street address
205, building 72, Healthcare city
City
Dubai
Province
Dubai
Postal code
Phone
0097144370551
Fax
Email
bahman.vasseghi@clinart.net
Web page address

Person responsible for scientific inquiries

Contact
Name of organization / entity
Department of Medicine, University of Milan
Full name of responsible person
Prof. Pier Mannuccio Mannucci
Position
study coordinator
Other areas of specialty/work
Street address
Via Pace, 9 – 20122
City
Milano
Province
Milan
Postal code
Phone
0039 02 55035421
Fax
Email
piermannuccio.mannucci@unimi.it
Web page address

Person responsible for updating data

Contact
Name of organization / entity
Clinart
Full name of responsible person
Dr. Bahman Vasseghi
Position
Study manager-Iran/MD
Other areas of specialty/work
Street address
205, Building 72, Healthcare city
City
Dubai
Province
Dubai
Postal code
Phone
0097144370551
Fax
Email
bahman.vasseghi@clinart.net
Web page address

Sharing plan

Deidentified Individual Participant Data Set (IPD)
empty
Study Protocol
empty
Statistical Analysis Plan
empty
Informed Consent Form
empty
Clinical Study Report
empty
Analytic Code
empty
Data Dictionary
empty
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