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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Octapharma
ClinicalTrials.gov Identifier:
NCT01125813
First received: May 17, 2010
Last updated: March 11, 2013
Last verified: March 2013
  Purpose

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.


Condition Intervention Phase
Severe Hemophilia A
Biological: recombinant Factor VIII
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Prevention
Official Title: Clinical Study to Investigate the Efficacy, Safety, and Immunogenicity of Human-cl rhFVIII in Previously Treated Patients With Severe Hemophilia A

Resource links provided by NLM:


Further study details as provided by Octapharma:

Primary Outcome Measures:
  • Efficacy Assessment After a Total of at Least 50 EDs Per Subject at the End of the Study at 6 Months [ Time Frame: At least 50 Exposure Days and at least 6 months ] [ Designated as safety issue: No ]
    Frequency of spontaneous breakthrough bleeds/months under prophylactic treatment.

  • Efficacy of Treating Bleeding Episodes [ Time Frame: After each bleeding episode, up to 6 month ] [ Designated as safety issue: No ]

    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


Enrollment: 32
Study Start Date: June 2010
Study Completion Date: January 2012
Primary Completion Date: January 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: human cl-rhFVIII Biological: recombinant Factor VIII
intravenous infusion of factor FVIII every other day.
Other Name: human-cl rhFVIII

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01125813

Locations
Austria
Medizinische Universitaet Wien
Wien, Austria, 1090
Bulgaria
Haematological Hospital Joan Pavel
Sofia, Bulgaria, 1233
Germany
Werlhof Institut fuer Haemostaseologie GmbH
Hannover, Niedersachsen, Germany, 30159
Universitaetsklinikum
Bonn, Germany
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, Germany, 20246
SRH Kurpfalzkrankenhaus Heidelberg
Heidelberg, Germany, 69123
United Kingdom
Basingstoke & North Hampshire NHS Foundation Trust
Basingstoke, United Kingdom, RG24 9NA
University Hospital of Wales
Cardiff, United Kingdom
Royal Free Hospital
London, United Kingdom
Manchester Royal Infirmary
Manchester, United Kingdom
Royal Hallamshire Hospital
Sheffield, United Kingdom
Sponsors and Collaborators
Octapharma
Investigators
Principal Investigator: Johannes Oldenburg, Prof. Universitaetsklinikum Bonn
  More Information

No publications provided

Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT01125813     History of Changes
Other Study ID Numbers: GENA-08
Study First Received: May 17, 2010
Results First Received: January 21, 2013
Last Updated: March 11, 2013
Health Authority: Germany: Paul Ehrlich Institute
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Austria: AGES
Bulgaria: Bulgarian Drug Agency

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 16, 2014