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

• ClinicalTrials.gov Identifier: NCT01125813
• Recruitment Status: Completed
• First Posted: May 18, 2010
• Results First Posted: April 22, 2013
• Last Update Posted: September 11, 2017
• Sponsor: Octapharma
• Information provided by (Responsible Party): Octapharma

Tracking Information

• First Submitted Date: May 17, 2010
• First Posted Date: May 18, 2010
• Results First Submitted Date: January 21, 2013
• Results First Posted Date: April 22, 2013
• Last Update Posted Date: September 11, 2017
• Study Start Date: June 2010
• Actual Primary Completion Date: January 2012 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 11, 2013)

• 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 ]
  Frequency of spontaneous breakthrough bleeds/months under prophylactic treatment.
• Efficacy of Treating Bleeding Episodes [ Time Frame: 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

• Original Primary Outcome Measures (submitted: May 17, 2010): Efficacy Assessment after a total of at least 50 EDs per subject at the end of the study at 6 months [ Time Frame: February 2011 ]
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A
• Official Title: Clinical Study to Investigate the Efficacy, Safety, and Immunogenicity of Human-cl rhFVIII in Previously Treated Patients With Severe Hemophilia A
• 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 Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Prevention
• Condition: Severe Hemophilia A

Intervention

Biological: recombinant Factor VIII

intravenous infusion of factor FVIII every other day.

Other Name: human-cl rhFVIII

Study Arms

Experimental: human cl-rhFVIII

Intervention: Biological: recombinant Factor VIII

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: May 17, 2010): 32
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: January 2012
• Actual Primary Completion Date: January 2012 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: Male

• Ages: 12 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Austria, Bulgaria, Germany, United Kingdom

Administrative Information

• NCT Number: NCT01125813
• Other Study ID Numbers: GENA-08
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Octapharma
• Original Responsible Party: Head of Clinical Research and Development, Hematology
• Current Study Sponsor: Octapharma
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Johannes Oldenburg, Prof.; Universitaetsklinikum Bonn

• PRS Account: Octapharma
• Verification Date: August 2017