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

Phase 3CompletedNCT01341912

Octapharma3 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Severe Hemophilia A

Interventions

Human-cl rhFVIIIBIOLOGICAL

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.

Eligibility

Sex: MALEMin age: 12 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (2)

Georgetown University

Washington D.C., District of Columbia, United States

Haematological Hospital SHAT "Joan Pavel"

Sofia, Bulgaria

Outcomes

Primary Outcomes

Long-term Immunogenicity

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.

Time frame: up to 3 years

Secondary Outcomes

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

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.

Time frame: up to 3 years

Data from ClinicalTrials.gov

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