Back to resultsHuman Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
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 focused on measuring Previously untreated patients
Eligibility Criteria
Inclusion Criteria:
- Male patients
- Severe Hemophilia A (FVIII:C <1%)
- No previous treatment with FVIII concentrates or other blood products containing FVIII
Exclusion Criteria:
- Diagnosis with a coagulation disorder other than Hemophilia A
- Severe liver or kidney disease
- Concomitant treatment with any systemic immunosuppressive drug
Sites / Locations
- UC Davis
- All Children's Hospital
- Harvard Children's Hospital Boston
- Republican Scientific Practical Center for Pediatric Oncology and Hematology
- University of Alberta
- BC Children's Hospital
- Mc Master Children's Hospital
- Hospital for Sick Children
- Hopital Ste-Justine
- L'hôpital Côte de Nacre - CHU de Caen
- Centre de traitement de l'hemophilie, Hôpital Bicêtre
- Hopital de la Timone
- Hôtel-Dieu de Nantes, Centre Regional de Traitement de l'hemophilie
- Hôpital Necker
- CHU de Rennes - Hôpital Pontchaillou
- Hopital Trousseau - CHU Tours
- Institute of Haematology and Transfusiology
- Institut für Experimentelle Hämatologie und Transfusionsmedizin (IHT)
- University Hospital Frankfurt/M
- Universitätsmedizin der Johannes-Gutenberg-Universität Mainz
- Kasturba Medical College, Dr. TMA Pai Hospital
- Sahyadri Speciality Hospital, Haematology & BMT Unit
- Christian Medical College & Hospital, Dept of Haematology
- Univ. Di Perugia
- Centro di Referimento per le Malattie Emorragiche e Trombotiche
- Scientific Research Institute of Mother and Child Health Care
- Centre Hospitalier Ibn Sina
- University Medical School Warsaw
- HSJ - Hospital de São João, EPE
- Morozovsky Children's Hospital
- Haemophilia Centre, University Clinical Centre
- Unitat d'hemofilia, Hospital Universitari Vall d'Hebron
- Hospital Universitario La Paz
- National Children's Specialized Hospital "OHMATDET"
- Institute of Blood Pathology and Transfusion Medicine
- Cambridge University Hospital
- 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 using the modified Bethesda assay (Nijmegen modification). The definitions for thresholds were ≥0.6 to <5 BU/mL for a "low titre" inhibitor and ≥5 BU/mL for a "high-titre" inhibitor.
Secondary Outcome Measures
Frequency of Spontaneous Break-through Bleeds
The annualized bleeding rate (ABR) was calculated during inhibitor-free periods for spontaneous bleeding events (BEs) during prophylactic treatment with Human cl rhFVIII
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds
A personal efficacy assessment to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes. Efficacy was assessed using a four-point scale (excellent, good, moderate, none).
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).
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01712438
Brief Title
Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
Official Title
Immunogenicity, Efficacy and Safety of Treatment With Human-cl-rhFVIII in Previously Untreated Patients With Severe Hemophilia A
Study Type
Interventional
2. Study Status
Record Verification Date
December 2020
Overall Recruitment Status
Completed
Study Start Date
February 2013 (undefined)
Primary Completion Date
December 14, 2018 (Actual)
Study Completion Date
December 20, 2019 (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
Investigate 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
Keywords
Previously untreated patients
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
110 (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 using the modified Bethesda assay (Nijmegen modification). The definitions for thresholds were ≥0.6 to <5 BU/mL for a "low titre" inhibitor and ≥5 BU/mL for a "high-titre" inhibitor.
Time Frame
maximum 5 years (100 exposure days)
Secondary Outcome Measure Information:
Title
Frequency of Spontaneous Break-through Bleeds
Description
The annualized bleeding rate (ABR) was calculated during inhibitor-free periods for spontaneous bleeding events (BEs) during prophylactic treatment with Human cl rhFVIII
Time Frame
Maximum 5 years (100 exposure days)
Title
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds
Description
A personal efficacy assessment to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes. Efficacy was assessed using a four-point scale (excellent, good, moderate, none).
Time Frame
Maximum 5 years (100 exposure days)
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).
Time Frame
Maximum 5 years (100 exposure days)
Other Pre-specified Outcome Measures:
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
5 years
10. Eligibility
Sex
Male
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male patients
Severe Hemophilia A (FVIII:C <1%)
No previous treatment with FVIII concentrates or other blood products containing FVIII
Exclusion Criteria:
Diagnosis with a coagulation disorder other than Hemophilia A
Severe liver or kidney disease
Concomitant treatment with any systemic immunosuppressive drug
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sigurd Knaub
Organizational Affiliation
Octapharma
Official's Role
Study Director
Facility Information:
Facility Name
UC Davis
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
All Children's Hospital
City
Saint Petersburg
State/Province
Florida
ZIP/Postal Code
33701
Country
United States
Facility Name
Harvard Children's Hospital Boston
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Republican Scientific Practical Center for Pediatric Oncology and Hematology
City
Minsk
Country
Belarus
Facility Name
University of Alberta
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G2V2
Country
Canada
Facility Name
BC Children's Hospital
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6H 3V4
Country
Canada
Facility Name
Mc Master Children's Hospital
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8S4K1
Country
Canada
Facility Name
Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Facility Name
Hopital Ste-Justine
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3T 1C5
Country
Canada
Facility Name
L'hôpital Côte de Nacre - CHU de Caen
City
Caen
Country
France
Facility Name
Centre de traitement de l'hemophilie, Hôpital Bicêtre
City
Le Kremlin-Bicêtre
Country
France
Facility Name
Hopital de la Timone
City
Marseille
Country
France
Facility Name
Hôtel-Dieu de Nantes, Centre Regional de Traitement de l'hemophilie
City
Nantes
Country
France
Facility Name
Hôpital Necker
City
Paris
Country
France
Facility Name
CHU de Rennes - Hôpital Pontchaillou
City
Rennes
Country
France
Facility Name
Hopital Trousseau - CHU Tours
City
Tours
Country
France
Facility Name
Institute of Haematology and Transfusiology
City
Tbilisi
Country
Georgia
Facility Name
Institut für Experimentelle Hämatologie und Transfusionsmedizin (IHT)
City
Bonn
Country
Germany
Facility Name
University Hospital Frankfurt/M
City
Frankfurt
ZIP/Postal Code
60590
Country
Germany
Facility Name
Universitätsmedizin der Johannes-Gutenberg-Universität Mainz
City
Mainz
Country
Germany
Facility Name
Kasturba Medical College, Dr. TMA Pai Hospital
City
Manipala
State/Province
Karnataka
Country
India
Facility Name
Sahyadri Speciality Hospital, Haematology & BMT Unit
City
Pune
Country
India
Facility Name
Christian Medical College & Hospital, Dept of Haematology
City
Vellore
Country
India
Facility Name
Univ. Di Perugia
City
Perugia
Country
Italy
Facility Name
Centro di Referimento per le Malattie Emorragiche e Trombotiche
City
Torino
Country
Italy
Facility Name
Scientific Research Institute of Mother and Child Health Care
City
Chisinau
Country
Moldova, Republic of
Facility Name
Centre Hospitalier Ibn Sina
City
Rabat
Country
Morocco
Facility Name
University Medical School Warsaw
City
Warsaw
ZIP/Postal Code
00-576
Country
Poland
Facility Name
HSJ - Hospital de São João, EPE
City
Porto
Country
Portugal
Facility Name
Morozovsky Children's Hospital
City
Moscow
Country
Russian Federation
Facility Name
Haemophilia Centre, University Clinical Centre
City
Ljubljana
Country
Slovenia
Facility Name
Unitat d'hemofilia, Hospital Universitari Vall d'Hebron
City
Barcelona
Country
Spain
Facility Name
Hospital Universitario La Paz
City
Madrid
Country
Spain
Facility Name
National Children's Specialized Hospital "OHMATDET"
City
Kiev
Country
Ukraine
Facility Name
Institute of Blood Pathology and Transfusion Medicine
City
Lviv
Country
Ukraine
Facility Name
Cambridge University Hospital
City
Cambridge
ZIP/Postal Code
CB2 0QQ
Country
United Kingdom
Facility Name
Great Ormond Street Hospital for Children
City
London
ZIP/Postal Code
WC1N3JH
Country
United Kingdom
12. IPD Sharing Statement
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Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
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