Condition category
Haematological Disorders
Date applied
Date assigned
Last edited
Retrospectively registered
Overall trial status
Recruitment status

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

Trial website

Contact information



Primary contact

Dr Raina Liesner


Contact details

Great Ormond Street Hospital for Children
NHS Trust
Haemophilia Centre
Great Ormond Street
United Kingdom

Additional identifiers

EudraCT number

2012-002554-23 number


Protocol/serial number


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


Study hypothesis

Immunogenicity of Human-cl rhFVIII in previously untreated patients with severe haemophilia A is low.

Ethics approval

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


Secondary study design

Non randomised controlled trial

Trial setting


Trial type


Patient information sheet

Not available in web format, please use the contact details below to request a patient information sheet


Severe haemophilia A


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



Phase III

Drug names

Human-cl rhFVIII

Primary outcome measures

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 trial start date


Overall trial end date


Reason abandoned


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 patient’s parent/legal guardian)

Participant type


Age group




Target number of participants


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


Recruitment end date



Countries of recruitment

Brazil, Canada, Colombia, France, Georgia, Germany, India, Moldova, Morocco, Poland, Russian Federation, Spain, Ukraine, United Kingdom, United States of America, Venezuela

Trial participating centre

Great Ormond Street Hospital for Children, NHS Trust
United Kingdom

Sponsor information


Octapharma AG (Switzerland)

Sponsor details

Seidenstrasse 2

Sponsor type




Funder type


Funder name

Octapharma AG (Switzerland)

Alternative name(s)

Funding Body Type

Funding Body Subtype


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