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

Severe Hemophilia A Clinical Trial

Official title:

Clinical Study to Investigate the Efficacy, Safety, and Immunogenicity of Human-cl rhFVIII in Previously Treated Patients With Severe Hemophilia A

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01125813
• Other study ID #: GENA-08
• Status: Completed
• Phase: Phase 3
• First received: May 17, 2010
• Last updated: March 11, 2013
• Start date: June 2010
• Est. completion date: January 2012

Study information

• Verified date: March 2013
• Source: Octapharma
• Contact: n/a
• Is FDA regulated: No
• Health authority: Germany: Paul Ehrlich InstituteUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyAustria: AGESBulgaria: Bulgarian Drug Agency
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 32
• Est. completion date: January 2012
• Est. primary completion date: January 2012
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years and older

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Prevention

Related Conditions & MeSH terms

• Hemophilia A
• Severe Hemophilia A

Intervention

Biological:
• recombinant Factor VIII
intravenous infusion of factor FVIII every other day.

Locations

Country	Name	City	State
Austria	Medizinische Universitaet Wien	Wien
Bulgaria	Haematological Hospital Joan Pavel	Sofia
Germany	Universitaetsklinikum	Bonn
Germany	Universitaetsklinikum Hamburg-Eppendorf	Hamburg
Germany	Werlhof Institut fuer Haemostaseologie GmbH	Hannover	Niedersachsen
Germany	SRH Kurpfalzkrankenhaus Heidelberg	Heidelberg
United Kingdom	Basingstoke & North Hampshire NHS Foundation Trust	Basingstoke
United Kingdom	University Hospital of Wales	Cardiff
United Kingdom	Royal Free Hospital	London
United Kingdom	Manchester Royal Infirmary	Manchester
United Kingdom	Royal Hallamshire Hospital	Sheffield

Sponsors (1)

Lead Sponsor	Collaborator
Octapharma

Countries where clinical trial is conducted

Austria,  Bulgaria,  Germany,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Efficacy Assessment After a Total of at Least 50 EDs Per Subject at the End of the Study at 6 Months	Frequency of spontaneous breakthrough bleeds/months under prophylactic treatment.	At least 50 Exposure Days and at least 6 months	No
Primary	Efficacy of Treating Bleeding Episodes	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	After each bleeding episode, up to 6 month	No