Back to resultsStudy to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
Primary Purpose
Severe Hemophilia A
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Human-cl rhFVIII
Sponsored by
About this trial
This is an interventional treatment trial for Severe Hemophilia A
Eligibility Criteria
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
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
Sites / Locations
- Georgetown University
- Haematological Hospital SHAT "Joan Pavel"
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Human-cl rhFVIII
Arm Description
Recombinant FVIII derived from a human cell line.
Outcomes
Primary Outcome Measures
Long-term Immunogenicity
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
To Determine Long-term Efficacy of Human-cl rhFVIII in the Treatment of Bleeding Episodes and in Surgical Prophylaxis
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01341912
Brief Title
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
Official Title
Clinical Study to Investigate the Long-Term Efficacy, Safety and Immunogenicity of Human-cl rhFVIII in Previously Treated Patients With Severe Haemophilia A - Extension Study to GENA-01
Study Type
Interventional
2. Study Status
Record Verification Date
March 2020
Overall Recruitment Status
Completed
Study Start Date
June 2011 (undefined)
Primary Completion Date
August 2012 (Actual)
Study Completion Date
August 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Octapharma
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Severe Hemophilia A
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Human-cl rhFVIII
Arm Type
Experimental
Arm Description
Recombinant FVIII derived from a human cell line.
Intervention Type
Biological
Intervention Name(s)
Human-cl rhFVIII
Intervention Description
Human-cl rhFVIII is administered intravenously on demand for bleeding episodes and prophylactically in case of surgery. The dosage depends on the severity of the bleeding episodes and the surgery.
Primary Outcome Measure Information:
Title
Long-term Immunogenicity
Description
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.
Time Frame
up to 3 years
Secondary Outcome Measure Information:
Title
To Determine Long-term Efficacy of Human-cl rhFVIII in the Treatment of Bleeding Episodes and in Surgical Prophylaxis
Description
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.
Time Frame
up to 3 years
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
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
Facility Information:
Facility Name
Georgetown University
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20057
Country
United States
Facility Name
Haematological Hospital SHAT "Joan Pavel"
City
Sofia
Country
Bulgaria
12. IPD Sharing Statement
Learn more about this trial
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
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