Condition category
Haematological Disorders
Date applied
09/11/2009
Date assigned
16/11/2009
Last edited
04/01/2012
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

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 measures

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

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

http://www.octapharma.com

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

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes