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

Not Applicable

Completed

• Conditions: Severe haemophilia A in children; Haematological Disorders; Hereditary factor VIII deficiency

• Registration Number: ISRCTN99606748
• Lead Sponsor: Octapharma AG (Switzerland)

Brief Summary

2015 results in: https://www.ncbi.nlm.nih.gov/pubmed/26370328

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2012-01-03
• Study Completion: 2014-03-31
• Last Update Posted: 30 March 2020

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 60

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

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)

Study & Design

• Study Type: Observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<br> Long-term immunogenicity:<br> 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.<br>