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 |
| EudraCT/CTIS number | 2009-014422-41 |
| Secondary identifying numbers | GENA-04 |
- 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
Scientific
Oberlaaerstrasse 235
Vienna
1100
Austria
| Phone | +43 (0)1 61032 1208 |
|---|---|
| martina.jansen@octapharma.com |
Study information
| Study design | Prospective open-label clinical trial |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Non randomised controlled trial |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details below to request patient information material |
| 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 |
| Pharmaceutical study type(s) | |
| Phase | Not Applicable |
| Drug / device / biological / vaccine name(s) | 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 study start date | 01/11/2009 |
| Completion date | 01/07/2011 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Male |
| Target number of participants | 22 (Recruitment completed) |
| 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
1100
Austria
Sponsor information
Octapharma AG (Switzerland)
Industry
Industry
Seidenstrasse 2
Lachen
CH-8853
Switzerland
| sigurd.knaub@octapharma.ch | |
| Website | http://www.octapharma.com |
"ROR" |
https://ror.org/002k5fe57 |
Funders
Funder type
Industry
Octapharma AG (Switzerland)
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
