Clinical Study to Investigate the Long-Term Efficacy, Safety, and Immunogenicity of human-cl rhFVIII in Previously Treated Patients with Severe Haemophilia A – Extension Study to GENA-01

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2010-023242-69-DE

Enrollment

Registered

2011-02-08

Start date

2011-05-31

Completion date

Unknown

Last updated

2013-09-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Severe haemophilia A (FVIII:C <1%) MedDRA version: 12.1 Level: LLT Classification code 10018937 Term: Haemophilia A

Interventions

Product Name: Human-cl rhFVIII Product Code: Human-cl rhFVIII Pharmaceutical Form: Powder and solvent for solution for infusion

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: Completion of GENA-01 study with at least 50 Exposure Days (EDs) and at least 6 months study participation and immediate enrolment into GENA-11. Voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted. Patient must be capable to understand and comply with the relevant aspects of the study protocol. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Development of FVIII inhibitors (=0.6 BU), during the course of the GENA-01 study. Development of any severe liver or kidney disease (ALT and AST levels >5 times of upper limit of normal, creatinine >120 µmol/L) during the course of the GENA-01 study.

Design outcomes

Primary

Measure	Time frame
Main Objective: To determine the long-term immunogenicity of human-cl rhFVIII in previously treated patients with severe haemophilia A. To determine the long-term tolerability of human-cl rhFVIII in previously treated patients with severe haemophilia A. ;Secondary Objective: To determine the long-term efficacy of human-cl rhFVIII in the treatment of bleeding episodes (BEs) and in surgical prophylaxis in previously treated patients with severe haemophilia A.;Primary end point(s): Long-Term Safety and Tolerability 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 three-monthly intervals until study completion. At the same time-points, anti-rhFVIII antibodies will be measured. The same parameters will also be determined in case inhibitor development is suspected. Sampling for inhibitor and antibody measurements should be performed not less than 3 days after the previous administration of any FVIII product, if possible. In case of positive results, 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 participation.	—

Measure

Time frame

Main Objective: To determine the long-term immunogenicity of human-cl rhFVIII in previously treated patients with severe haemophilia A. To determine the long-term tolerability of human-cl rhFVIII in previously treated patients with severe haemophilia A. ;Secondary Objective: To determine the long-term efficacy of human-cl rhFVIII in the treatment of bleeding episodes (BEs) and in surgical prophylaxis in previously treated patients with severe haemophilia A.;Primary end point(s): Long-Term Safety and Tolerability 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 three-monthly intervals until study completion. At the same time-points, anti-rhFVIII antibodies will be measured. The same parameters will also be determined in case inhibitor development is suspected. Sampling for inhibitor and antibody measurements should be performed not less than 3 days after the previous administration of any FVIII product, if possible. In case of positive results, 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 participation.

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Countries

Germany

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026