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

Phase 3

Completed

• Conditions: Severe Hemophilia A
• Interventions: Biological: Human-cl rhFVIII

• Registration Number: NCT01341912
• Lead Sponsor: Octapharma

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2011-04-22
• Study First Submitted QC: 2011-04-25
• Study First Posted: 2011-04-26
• Study Start: 2011-06
• Primary Completion: 2012-08
• Study Completion: 2012-08
• Results First Submitted: 2013-10-11
• Results First Submitted QC: 2013-12-16
• Results First Posted: 2014-02-03
• Status Verified: 2020-03
• Last Update Submitted: 2020-03-03
• Last Update Posted: 2020-03-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 3

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Human-cl rhFVIII	Human-cl rhFVIII	Recombinant FVIII derived from a human cell line.

Primary Outcome Measures

Name	Time	Method
Long-term Immunogenicity	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 Outcome Measures

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

Trial Locations

Locations (2)

Georgetown University; 🇺🇸 Washington, District of Columbia, United States

Haematological Hospital SHAT "Joan Pavel"; 🇧🇬 Sofia, Bulgaria