Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)

The purpose of the study is to study the long-term efficacy, safety and tolerability of Human-cl rhFVIII in previously treated patients with severe hemophilia A.

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Key information

Conditions

Severe Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia A Hemorrhagic Disorders

Age range

12 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Haematological Hospital SHAT "Joan Pavel", Sofia, Bulgaria

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Completion of GENA-01 study with at least 50 Exposure Days (EDs) and at least 6 months study participation and immediate enrollment into GENA-11

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 level > 5 times of upper limit of normal, creatine >120 micro mol/L) during the course of the GENA-01 study

Treatment and study plan

Human-cl rhFVIII

Biological

Human-cl rhFVIII is administered intravenously on demand for bleeding episodes and prophylactically in case of surgery. The dosage depends on the severity of the bleeding episodes and the surgery.

Primary outcomes

Long-term Immunogenicity

Time frame: up to 3 years

Patients will be monitored for inhibitors against FVIII every 3 months. Blood samples were drawn and inhibitor activity was determined by the modified Bethesda assay (Nijmegen modification) in the central lab.

Secondary outcomes

To Determine Long-term Efficacy of Human-cl rhFVIII in the Treatment of Bleeding Episodes and in Surgical Prophylaxis

Time frame: up to 3 years

The efficacy of human-cl rhFVIII will be determined using a 4 point efficacy assessment scale.

After each infusion of IMP and at the end of a BE, the following efficacy assessment is made by the subject (together with the Investigator in case of on-site treatment):

Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single infusion.

Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 - 12 hours after an infusion requiring up to 2 infusions for complete resolution.

Moderate: Probable or slight beneficial effect within approximately 12 hours after the first infusion requiring more than two infusions for complete resolution.

None: No improvement within 12 hours, or worsening of symptoms, requiring more than 2 infusions for complete resolution.

The assessment was made at the end of a BE in case more than one infusion was needed.

Sponsors and collaborators

Lead sponsor

Octapharma

Industry

Registry information

Official study title

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

Important dates

Study start: 2011
Primary completion: 2012
Study completion: 2012
First posted: Apr 26, 2011
Registry last updated: Mar 17, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed