Clinical study to investigate the long-term safety and efficacy of human cell line recombinant Factor VIII (human-cl rhFVIII) in previously treated patients with severe haemophilia A

ISRCTN	ISRCTN90038418
DOI	https://doi.org/10.1186/ISRCTN90038418
Clinical Trials Information System (CTIS)	2009-014422-41
Protocol serial number	GENA-04
Sponsor	Octapharma AG (Switzerland)
Funder	Octapharma AG (Switzerland)

Submission date: 09/11/2009
Registration date: 16/11/2009
Last edited: 04/01/2012

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Haematological Disorders

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Record updated in last year

Plain English summary of protocol

Not provided at time of registration

Contact information

Ms Martina Jansen
Scientific

Oberlaaerstrasse 235
Vienna
1100
Austria

Phone	+43 (0)1 61032 1208
Email	martina.jansen@octapharma.com

Study information

Primary study design	Interventional
Study design	Prospective open-label clinical trial
Secondary study design	Non randomised controlled trial
Study type	Participant information sheet
Scientific title
Study objectives	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(s)	Ethics Committee at the Federal Supervision Service for Public Health and Social Affairs approved on the 9th September 2009 (ref: "Case EC-37284)
Health condition(s) or problem(s) studied	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 / device / biological / vaccine name(s)	Human cell line recombinant Factor VIII (human-cl rhFVIII)
Primary outcome measure(s)	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.
Key secondary outcome measure(s)	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.
Completion date	01/07/2011

Eligibility

Participant type(s)	Patient
Age group	Adult
Lower age limit	18 Years
Sex	Male
Target sample size at registration	22
Key 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
Key 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
Date of first enrolment	01/11/2009
Date of final enrolment	01/07/2011

Locations

Countries of recruitment

Austria
Russian Federation

Study participating centre

Oberlaaerstrasse 235

Vienna
1100
Austria

Results and Publications

Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Participant information sheet	Participant information sheet	11/11/2025	11/11/2025	No	Yes