Contact information
Type
Scientific
Primary contact
Ms Martina Jansen
ORCID ID
Contact details
Oberlaaerstrasse 235
Vienna
1100
Austria
+43 (0)1 61032 1208
martina.jansen@octapharma.com
Additional identifiers
EudraCT number
2009-014422-41
ClinicalTrials.gov number
Protocol/serial number
GENA-04
Study information
Scientific title
Acronym
Study hypothesis
Investigation of the long-term immunogenic potential of human cell line recombinant Factor VIII (human-cl rhFVIII).
As of 03/01/2012, the anticipated end date was corrected from 01/01/2012 to 01/07/2011.
Ethics approval
Ethics Committee at the Federal Supervision Service for Public Health and Social Affairs approved on the 9th September 2009 (ref: "Case EC-37284)
Study design
Prospective open-label clinical trial
Primary study design
Interventional
Secondary study design
Non randomised controlled trial
Trial setting
Hospitals
Trial type
Treatment
Patient information sheet
Not available in web format, please use the contact details below to request patient information material
Condition
Severe haemophilia A
Intervention
All patients will be treated in accordance with their needs until the product is registered and launched in the country of conductance. There are no further interventions planned beside the three-monthly control of FVIII recovery and inhibitor development.
Intervention type
Drug
Phase
Not Applicable
Drug names
Human cell line recombinant Factor VIII (human-cl rhFVIII)
Primary outcome measure
Immunogenicity: Inhibitor activity will be determined by the modified Bethesda assay (Nijmegen modification) at three months intervals until study completion. At the same time-points the anti-rhFVIII antibodies will be measured.
Secondary outcome measures
1. Clinical tolerability: assessed by monitoring vital signs (blood pressure, heart rate, respiratory rate, and body temperature will be assessed at pre-defined time-points)
2. Laboratory parameters:
2.1. Haematological parameters - red blood cell count, white blood cell count, haemoglobin, haematocrit, and platelet count
2.2. ALAT, ASAT, serum creatinine
3. Adverse events (AEs)
4. Prophylactic treatment: the frequency of bleeds under prophylactic treatment will be calculated. Study drug consumption data (FVIII IU/kg per month, per year) per subject and in total will be evaluated.
5. Treatment of bleeding episodes: efficacy assessment at the end of each BE
6. In-vivo recovery: calculated from the FVIII:C plasma levels measured before infusion and peak level obtained in the 30 or 60 minutes post-infusion sample and the actual potency of Human-cl rhFVIII. FVIII:C in the product and in plasma will be measured both by the chromogenic (CHR) and the one-stage (OS) assay.
Overall trial start date
01/11/2009
Overall trial end date
01/07/2011
Reason abandoned (if study stopped)
Eligibility
Participant inclusion criteria
1. Must have severe haemophilia A (FVIII:C less than 1%; historical value as documented in subject records)
2. Aged greater than 18 years and less than 65 years, male only
3. Body weight 45 kg to 110 kg
4. Previously treated with human-cl rhFVIII (within study GENA-09)
6. Negative for human immunodeficiency virus (HIV) or respective viral load less than 200 particles/µL
7. Freely given written informed consent
Participant type
Patient
Age group
Adult
Gender
Male
Target number of participants
22 (Recruitment completed)
Participant exclusion criteria
1. Other coagulation disorder than haemophilia A
2. Present or past FVIII inhibitor activity (greater than 0.6 BU)
3. Severe liver or kidney disease (alanine aminotransferase [ALAT] and aspartate aminotransferase [ASAT] levels greater than 5 times of upper limit of normal, creatinine greater than 120 µmol/L)
4. Receiving or scheduled to receive immuno-modulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to greater than 10 mg/day), or similar drugs
5. Participation in another clinical study currently or during the past month, except GENA-09
Recruitment start date
01/11/2009
Recruitment end date
01/07/2011
Locations
Countries of recruitment
Russian Federation
Trial participating centre
Oberlaaerstrasse 235
Vienna
1100
Austria
Sponsor information
Organisation
Octapharma AG (Switzerland)
Sponsor details
Seidenstrasse 2
Lachen
CH-8853
Switzerland
sigurd.knaub@octapharma.ch
Sponsor type
Industry
Website
Funders
Funder type
Industry
Funder name
Octapharma AG (Switzerland)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Basic results (scientific)
Publication list