|
ISRCTN
|
ISRCTN99606748
|
|
ClinicalTrials.gov identifier
|
|
|
Public title
|
Clinical study in which the long term effect of Human-cl rhFVIII is investigated in children with severe haemophilia A, who were previously treated in the GENA-03 study
|
|
Scientific title
|
Clinical study in which the immunogenicity, tolerability and efficacy of Human-cl rhFVIII is investigated in children with severe haemophilia A, who were previously treated in the GENA-03 study
|
|
Acronym
|
N/A
|
|
Serial number at source
|
GENA-13
|
|
Study hypothesis
|
Investigation of long-term efficacy and safety of Human-cl rhFVIII in children with severe haemophilia A (2 - 13 years old), followed up for an open prophylactic treatment until product registration.
Follow up to http://www.controlled-trials.com/ISRCTN71212110
|
|
Lay summary
|
Lay summary under review
|
|
Ethics approval
|
National Research Ethics Service, Riverside, Brighton (UK), 27 September 2011, ref 11/LO/1159
University Hospital Brno Ethics Committee, (Czech Republic), 24 August 2011 ref: GENA-13
Bioethics Committee, Medical University of Warsaw (Poland), 20 September 2011, ref KB/157/2011
People Protection Committee, Paris [Ile de France II] (France), 05 October 2011, ref 2011-08-02
|
|
Study design
|
Prospective open-label trial
|
|
Countries of recruitment
|
Czech Republic, France, Poland, Russian Federation, Turkey, United Kingdom
|
|
Disease/condition/study domain
|
Severe haemophilia A
|
|
Participants - inclusion criteria
|
1. Evaluable completion of the preceeding study GENA-03 by having a study participation period of 6 months, provided that prophylaxis with Human-cl rhFVIII is continued without intermediate interruption
2. Voluntarily given, fully informed written and signed consent obtained from the parents (or legal guardians) before any study-related procedures are conducted. The need for obtaining assent will depend on the subjects’ developmental stage and intellectual capacity
3. Capability to understand and comply with the relevant aspects of the study
|
|
Participants - exclusion criteria
|
1. Development of FVIII inhibitors (³0.6 Bethesda Units [BU]) in the course of the GENA-03 study
2. Any severe liver or kidney disease (ALT and AST levels >5 times of upper limit of normal, creatinine >120 µmol/L)
|
|
Anticipated start date
|
31/10/2011
|
|
Anticipated end date
|
31/03/2014
|
|
Status of trial
|
Ongoing |
|
Patient information material
|
Not available in web format, please use the contact details below to request a patient information sheet
|
|
Target number of participants
|
max. 60
|
|
Interventions
|
There is just one treatment arm: prophylactic treatment with rFVIII.
The investigational medicinal product (IMP) name is “Human-cl rhFVIII”, which is a recombinant factor 8 concentrate from a human cell-line. The product is injected every other day or three times a week (depending on the clinical needs of the patient), at a prophylactic dose of 30 – 40 IU/kg bodyweight. The study will continue until the patients can switch to the registered and launched product. The expected duration of the individual treatment is at least 2 years.
|
|
Primary outcome measure(s)
|
Long-term immunogenicity:
Inhibitor activity will be determined by the modified Bethesda assay (Nijmegen modification) using congenital FVIII-deficient human plasma spiked with Human-cl rhFVIII as a test base, at tri-monthly intervals until study completion. At the same time-points, anti-rhFVIII antibodies will be measured. These parameters will also be determined in case inhibitor development is suspected. Sampling for inhibitor and antibody measurements should be performed not less than 48 hours after the previous administration of any FVIII product, if possible. In case of positive inhibitor results, an inhibitor retesting using a second separately drawn sample should be performed. Long-term clinical tolerability: Will be assessed by monitoring Adverse Events (AEs) throughout the study duration.
|
|
Secondary outcome measure(s)
|
1. Efficacy of prophylactic treatment: Efficacy is to be assessed by evaluating the number of spontaneous breakthrough BEs, as well as by collecting data regarding frequency of IMP injections and regarding the prophylactic IMP doses needed. Overall study drug consumption data (FVIII (IU/kg), per month and per year), per subject and in total, will be analysed.
All efficacy ratings having been assessed as “excellent” and “good” will be reported as “successfully treated”. The proportion of BEs successfully treated with Human-cl rhFVIII will be evaluated both for all BEs, as well as broken down to BEs of different severity.
2. Efficacy of surgical prophylaxis: Efficacy will be assessed by the surgeon at the end of surgery (i.e. after last suture), and postoperatively (i.e. at date of discharge or on postoperative day 6, whichever occurs later), by both the surgeon and the haematologist using predefined study criteria, namely “excellent”, “good”, “moderate” or “none”. All efficacy ratings of surgical procedures having been assessed “excellent” and “good” will be reported as “successfully treated”.
|
|
Sources of funding
|
Octapharma AG (Switzerland)
|
|
Trial website
|
|
|
Publications
|
|
|
Contact name
|
Dr
Raina
Liesner
|
|
Address
|
Haemophilia Comprehensive Care Centre
Level 5, Royal London Hospital for Integrated Medicine (RLHIM)
Great Ormond Street Hospital
Great Ormond Street
|
|
City/town
|
London
|
|
Zip/Postcode
|
WC1N 3JH
|
|
Country
|
United Kingdom
|
|
Sponsor
|
Octapharma AG (Switzerland)
|
|
Address
|
Seidenstrasse 2
|
|
City/town
|
Lachen
|
|
Zip/Postcode
|
8853
|
|
Country
|
Switzerland
|
|
Tel
|
+41 554 512 141
|
|
Email
|
martina.jansen@octapharma.com
|
|
Sponsor website:
|
http://www.octapharma.com/en
|
|
Date applied
|
30/12/2011
|
|
Last edited
|
04/01/2012
|
|
Date ISRCTN assigned
|
03/01/2012
|
