Plain English Summary
Background and study aims
FVIII concentrates are the only available treatment for patients with severe haemophilia A. However, patients are at risk of developing resistance (inhibitor) to FVIII, which stops the treatment from working, and patients may also suffer from an allergic reaction. The drug under investigation, human-cl rhFVIII, is a newly developed recombinant FVIII concentrate from a human cell line, which may have less immunogenic potential (ability to provoke an immune response) compared to FVIII concentrates from hamster cell lines or plasma-derived FVIII concentrates. The main aim of the study is to investigate the immunogenicity of the new product in previously untreated patients with severe haemophilia A. This population is at the highest risk of developing inhibitors. Previous studies of the new product in already treated patients (adults and children) did not show a single case of inhibitor development.
Who can participate?
Previously untreated patients with severe haemophilia A.
What does the study involve?
All patients will receive the newly developed recombinant FVIII concentrate injection. The study involves regular blood sampling to screen for inhibitors. All patients adverse events are documented.
What are the possible benefits and risks of participating?
Human-cl rhFVIII may have less immunogenic potential compared to recombinant FVIII concentrates from hamster cell lines or plasma-derived FVIII concentrates. However, as for all FVIII concentrates, patients are at risk of developing an inhibitor to FVIII and may suffer from an allergic reaction.
Where is the study run from?
The study is planned to be conducted at about 45 study sites in 16 countries worldwide.
When is the study starting and how long is it expected to run for?
The study started in March 2013, and is planned to be completed in 2018.
Who is funding the study?
Octapharma AG, Switzerland
Who is the main contact?
Martina Jansen
Octapharma PPG
Clinical Research & Development Haematology
Oberlaaerstrasse 235
1100 Vienna, Austria
Study website
Additional identifiers
EudraCT/CTIS number
2012-002554-23
IRAS number
ClinicalTrials.gov number
NCT01712438
Protocol/serial number
GENA-05
Study information
Scientific title
Immunogenicity, efficacy and safety of treatment with Human-cl rhFVIII in previously untreated patients with severe haemophilia A: a prospective, multinational, open-label, non-controlled study
Acronym
Study hypothesis
Immunogenicity of Human-cl rhFVIII in previously untreated patients with severe haemophilia A is low.
Ethics approval(s)
Canada, HIREB Hamilton: 11 March 2013
Germany, Ethics Committee University Münster: 08 July 2013
Spain, Vall d`Hebron, Barcelona: 11 January 2013
France, CPP Ouest V, Nanterre: 07 February 2013
UK, NRES Committee London-Central: 19 February 2013
Georgia, Committee of Institute of Haematology, Tiflis: 17 January 2013
Moldova, National Ethics Committee, Chisinau: 29 January 2013
Poland, EC Medical University Warsaw: 12 February 2013
Russia, Izmailowska EC: 26 June 2013
Ukraine, National Academy of Medical Science: 04 February 2013
Study design
Prospective multicentre multinational open-label non-controlled study
Primary study design
Interventional
Secondary study design
Non randomised study
Study setting(s)
Hospital
Study type
Screening
Patient information sheet
Not available in web format, please use the contact details below to request a patient information sheet
Condition
Severe haemophilia A
Intervention
There is only one study arm. All patients receive the same investigational medicinal product (IMP) intravenously. The dose, frequency and duration are flexible, and depend on the individual clinical condition of the patient.
Intervention type
Drug
Pharmaceutical study type(s)
Phase
Phase III
Drug/device/biological/vaccine name(s)
Human-cl rhFVIII
Primary outcome measure
The immunogenic potential of the IMP. Each patient is tested for the development of inhibitors at treatment start, every three to four exposure days to the IMP, latterly every ten exposure days (latest every three months).
Secondary outcome measures
Safety, efficacy and tolerability: Efficacy (by assessing each treatment of a bleeding episode, or the rate of bleeds in case of prophylactic treatment) and safety (adverse events) are observed during the entire study duration, which is planned for a total of 100 exposure days with the IMP, but not longer than 5 years.
Overall study start date
01/03/2013
Overall study end date
24/03/2020
Reason abandoned (if study stopped)
Eligibility
Participant inclusion criteria
1. Male, no age limitations, but due to the required patient population it can be expected that the majority of patients going to be included are babies and small children.
2. Severe haemophilia A (FVIII:C < 1%)
3. No previous treatment with FVIII concentrates or other blood products containing FVIII
4. Voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted (obtained from the patients parent/legal guardian)
Participant type(s)
Patient
Age group
Child
Sex
Male
Target number of participants
100
Participant exclusion criteria
1. Diagnosis with a coagulation disorder other than haemophilia A
2. Severe liver or kidney disease (alanine amino transferase (ALT) or aspartate transaminase (AST) levels >5 times of upper limit of normal, creatinine >120 µmol/L)
3. Concomitant treatment with any systemic immunosuppressive drug
4. Participation in another interventional clinical study currently or during the past 4 weeks.
Recruitment start date
01/03/2013
Recruitment end date
30/06/2016
Locations
Countries of recruitment
Brazil, Canada, Colombia, England, France, Georgia, Germany, India, Moldova, Morocco, Poland, Russian Federation, Spain, Ukraine, United Kingdom, United States of America, Venezuela
Study participating centre
Great Ormond Street Hospital for Children, NHS Trust
London
WC1N 3JH
United Kingdom
Sponsor information
Organisation
Octapharma AG (Switzerland)
Sponsor details
Seidenstrasse 2
Lachen
8853
Switzerland
Sponsor type
Industry
Website
ROR
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
Individual participant data (IPD) sharing plan
Not provided at time of registration
IPD sharing plan summary
Not provided at time of registration
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Interim results article | interim results | 01/03/2018 | 14/05/2019 | Yes | No |
| Basic results | EU Clinical Trials Register results | 23/08/2020 | 20/05/2022 | No | No |
| Basic results | ClinicalTrials.gov results | 21/10/2019 | 23/05/2022 | No | No |