Back to resultsStudy to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII
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:
1. Patients who completed GENA-05 in accordance with the study protocol
Exclusion Criteria:
- Severe liver or kidney disease
- Concomitant treatment with any systemic immunosuppressive drug;
- Other FVIII concentrate than Human-cl rhFVIII was received between completion visit of GENA-05 and start of GENA-15 (except emergency cases).
Sites / Locations
- UC Davis Medical Center
- University of Alberta
- BC Children's Hospital
- McMaster Children's Hospital
- Hospital for Sick Children
- Hopital de la Timone
- Hôpital Kremlin Bicètre
- Institute of Hematology and Transfusiology
- Sahyadri Speciality Hospital
- Christian Medical College
- IMSP Mother and Child Institute
- University Medical School
- The National Children Specialized Hospital "OHMATDET"
- Danylo Halytsky Lviv National Medical University
- Great Ormond Street Hospital for Children
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Human-cl rhFVIII
Arm Description
Outcomes
Primary Outcome Measures
Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors
The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development by the modified Bethesda assay (Nijmegen modification) using congenital FVIII-deficient human plasma spiked with Human-cl rhFVIII. The definition threshold for a "positive" inhibitor was if the modified Bethesda assay resulted in a titre ≥0.6 BU/mL at any time point during the observation period.
Secondary Outcome Measures
Frequency of Spontaneous Break-through Bleeds
The annualized bleeding rate (ABR) was calculated during the time of prophylactic treatment with Human-cl rhFVIII for spontaneous bleeding events (BEs).
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds
A personal efficacy assessment (final outcome) to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes (BEs) at the end of a BE. Efficacy was assessed using a four-point scale (excellent, good, moderate, none) by the patient's parent(s)/legal guardian(s) together with the investigator in case of on site treatment.
Efficacy of Human-cl rhFVIII for Surgical Prophylaxis
An overall efficacy assessment to assess the efficacy of human-cl rhFVIII in surgical prophylaxis of minor and major surgeries. The efficacy assessment was analyzed using a four-point scale (excellent, good, moderate, none). If surgeries could not be assessed due to limited data available or having taken place outside the study site, the results were classified as "not done".
The Occurrence of Any Adverse Event (AE)
The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01992549
Brief Title
Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII
Official Title
Extension Study for Patients Who Completed GENA-05 (NuProtect)- to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII
Study Type
Interventional
2. Study Status
Record Verification Date
December 2020
Overall Recruitment Status
Completed
Study Start Date
April 2014 (undefined)
Primary Completion Date
December 27, 2018 (Actual)
Study Completion Date
December 27, 2018 (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 collect long-term data on the inhibitor development rate of Human-cl rhFVIII in previously untreated 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
48 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Human-cl rhFVIII
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
Human-cl rhFVIII
Primary Outcome Measure Information:
Title
Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors
Description
The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development by the modified Bethesda assay (Nijmegen modification) using congenital FVIII-deficient human plasma spiked with Human-cl rhFVIII. The definition threshold for a "positive" inhibitor was if the modified Bethesda assay resulted in a titre ≥0.6 BU/mL at any time point during the observation period.
Time Frame
Maximum two years
Secondary Outcome Measure Information:
Title
Frequency of Spontaneous Break-through Bleeds
Description
The annualized bleeding rate (ABR) was calculated during the time of prophylactic treatment with Human-cl rhFVIII for spontaneous bleeding events (BEs).
Time Frame
Maximum 2 years
Title
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds
Description
A personal efficacy assessment (final outcome) to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes (BEs) at the end of a BE. Efficacy was assessed using a four-point scale (excellent, good, moderate, none) by the patient's parent(s)/legal guardian(s) together with the investigator in case of on site treatment.
Time Frame
Maximum 2 years
Title
Efficacy of Human-cl rhFVIII for Surgical Prophylaxis
Description
An overall efficacy assessment to assess the efficacy of human-cl rhFVIII in surgical prophylaxis of minor and major surgeries. The efficacy assessment was analyzed using a four-point scale (excellent, good, moderate, none). If surgeries could not be assessed due to limited data available or having taken place outside the study site, the results were classified as "not done".
Time Frame
Maximum 2 years
Title
The Occurrence of Any Adverse Event (AE)
Description
The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence.
Time Frame
Maximum 2 years
10. Eligibility
Sex
Male
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
1. Patients who completed GENA-05 in accordance with the study protocol
Exclusion Criteria:
Severe liver or kidney disease
Concomitant treatment with any systemic immunosuppressive drug;
Other FVIII concentrate than Human-cl rhFVIII was received between completion visit of GENA-05 and start of GENA-15 (except emergency cases).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sigurd Knaub, PhD
Organizational Affiliation
Octapharma
Official's Role
Study Director
Facility Information:
Facility Name
UC Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
University of Alberta
City
Edmonton
State/Province
Alberta
Country
Canada
Facility Name
BC Children's Hospital
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6H 3V4
Country
Canada
Facility Name
McMaster Children's Hospital
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8S4K1
Country
Canada
Facility Name
Hospital for Sick Children
City
Toronto
Country
Canada
Facility Name
Hopital de la Timone
City
Marseille
Country
France
Facility Name
Hôpital Kremlin Bicètre
City
Paris
Country
France
Facility Name
Institute of Hematology and Transfusiology
City
Tbilisi
Country
Georgia
Facility Name
Sahyadri Speciality Hospital
City
Pune
ZIP/Postal Code
411004
Country
India
Facility Name
Christian Medical College
City
Vellore
ZIP/Postal Code
632004
Country
India
Facility Name
IMSP Mother and Child Institute
City
Chişinău
Country
Moldova, Republic of
Facility Name
University Medical School
City
Warsaw
Country
Poland
Facility Name
The National Children Specialized Hospital "OHMATDET"
City
Kiev
Country
Ukraine
Facility Name
Danylo Halytsky Lviv National Medical University
City
Lviv
Country
Ukraine
Facility Name
Great Ormond Street Hospital for Children
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII
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