Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A

Phase 3

Completed

• Conditions: Severe Hemophilia A
• Interventions: Biological: recombinant Factor VIII

• Registration Number: NCT01125813
• Lead Sponsor: Octapharma

Brief Summary

This study will determine the efficacy of human-cl rhFVIII in previously treated patients with severe hemophilia A during prophylactic treatment, treatment of bleeding episodes and in surgical prophylaxis.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2010-05-17
• Study First Submitted QC: 2010-05-17
• Study First Posted: 2010-05-18
• Study Start: 2010-06
• Primary Completion: 2012-01
• Study Completion: 2012-01
• Results First Submitted: 2013-01-21
• Results First Submitted QC: 2013-03-11
• Results First Posted: 2013-04-22
• Status Verified: 2017-08
• Last Update Submitted: 2017-08-09
• Last Update Posted: 2017-09-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 32

Inclusion Criteria

• Severe hemophilia A ((FVIII:C <= 1%)
• Male subjects >= 12 years of age
• Previously treated with FVIII concentrate, at least 50 EDs
• Immunocompetent (CD4+ count > 200/ul)
• Negative for anti- HIV; if positive, viral load < 200 particles/u; or <400,000 copies/mL

Exclusion Criteria

• Other coagulation disorder than hemophilia A
• Present of past FVIII inhibitor activity (.= 0.6 BU)
• Severe liver and kidney disease
• Receiving of scheduled to receive immuno-modulating drugs

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
human cl-rhFVIII	recombinant Factor VIII	-

Primary Outcome Measures

Name	Time	Method
Efficacy Assessment After a Total of at Least 50 EDs Per Subject at the End of the Study at 6 Months	At least 50 Exposure Days and at least 6 months	Frequency of spontaneous breakthrough bleeds/months under prophylactic treatment.
Efficacy of Treating Bleeding Episodes	After each bleeding episode, up to 6 month	At the end of a bleeding episode, efficacy was assessed as: * 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 Efficacy was rated

Trial Locations

Locations (11)

Medizinische Universitaet Wien; 🇦🇹 Wien, Austria

Haematological Hospital Joan Pavel; 🇧🇬 Sofia, Bulgaria

Werlhof Institut fuer Haemostaseologie GmbH; 🇩🇪 Hannover, Niedersachsen, Germany

Universitaetsklinikum Hamburg-Eppendorf; 🇩🇪 Hamburg, Germany

SRH Kurpfalzkrankenhaus Heidelberg; 🇩🇪 Heidelberg, Germany

Universitaetsklinikum; 🇩🇪 Bonn, Germany

Basingstoke & North Hampshire NHS Foundation Trust; 🇬🇧 Basingstoke, United Kingdom

Royal Free Hospital; 🇬🇧 London, United Kingdom

Royal Hallamshire Hospital; 🇬🇧 Sheffield, United Kingdom

Manchester Royal Infirmary; 🇬🇧 Manchester, United Kingdom

University Hospital of Wales; 🇬🇧 Cardiff, United Kingdom