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Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A

Primary Purpose

Severe Haemophilia A

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Human cl rhFVIII
Sponsored by
Octapharma
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Severe Haemophilia A

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • Severe Haemophilia A (FVIII:C < 1%)
  • Male patients >= 18 years of age
  • Previous treatment with a FVIII concentrate for at least 150 EDs
  • Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start
  • Immunocompetence (CD4+ count > 200/uL)

Exclusion Criteria:

  • Any coagulation disorder other than Haemophilia A
  • Present of past FVIII inhibitor activity
  • Severe liver or kidney disease

Sites / Locations

  • Octapharma Research Site
  • Octapharma Research Site
  • Octapharma Research Site
  • Octapharma Research Site
  • Octapharma Research Site
  • Octapharma Research Site
  • Octapharma Research Site
  • Octapharma Research Site
  • Octapharma Research Site
  • Octapharma Research Site
  • Octapharma Research Site
  • McMaster University
  • University Hospital Centre Zagreb
  • Helsinki University Hospital
  • Centre Régional de Traitement de l'Hémophilie
  • CHU Estaing
  • Centre Hospitalier Universitaire Félix Guyon
  • Centre Régional de Traitement de l'hémophilie
  • Hôpital Purpan - Centre de Traitment Regional de l'Hemophilie Pole
  • Nagoya University Hospital
  • Hospital of the Univ of Occupational and Environmental Health
  • St. Marianna Univ School of Medicine Hospital
  • Nara Medical University Hospital
  • Gunma University Hospital
  • Osaka National Hospital
  • Ogikubo Hospital
  • Teikyo University Hospital
  • University Medical Center Groningen
  • PHI Institute of Transfusion Medicine of Republic of Macedonia
  • University Medical Centre Ljubljana

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Human-cl rhFVIII

Arm Description

Outcomes

Primary Outcome Measures

Annualized Total Bleeding Rate of Individually Tailored Prophylaxis
Total annualized bleeding rate (ABR) of individually tailored prophylaxis (GENA-21b) compared to historical bleeding rate in patients having received on-demand treatment (GENA-01) with Human-cl rhFVIII

Secondary Outcome Measures

Annualized Spontaneous Bleeding Rate of Individually Tailored Prophylaxis
Spontaneous annualized bleeding rate (ABR) of individually tailored prophylaxis (GENA-21b) compared to historical bleeding rate in patients having received on-demand treatment (GENA-01) with Human-cl rhFVIII
Annualized Total Bleeding Rate in Patients With 2x/Week (or Less) Prophylaxis
Total annualized bleeding rate (ABR) in patients with 2x/week (or less) prophylaxis (GENA-21b) compared to historical bleeding rate in patients having received on-demand treatment (GENA-01) with Human-cl rhFVIII
Median Prophylactic Dosing Interval
Median over median actual dosing intervals between two prophylactic treatments per patient. The median time (hours) between two prophylactic doses of Human-cl rhFVIII in the prophylactic treatment Phase II per patient
Mean Prophylactic Dosing Interval
Mean over mean actual dosing intervals between two prophylactic treatments per patient. The mean time (hours) between two prophylactic doses of Human-cl rhFVIII in the prophylactic treatment Phase II per patient
AUC Divided by the Dose (AUCnorm) of Human-cl rhFVIII
AUCnorm of Human-cl rhFVIII measured using the one-stage (OS) assay
In-vivo Recovery (IVR) of Human-cl rhFVIII
IVR of Human-cl rhFVIII measured using the one-stage (OS) assay and will be determined from the FVIII level before the infusion and the peak level after the infusion of Human-cl rhFVIII
Half Life (t1/2) of Human-cl rhFVIII
T1/2 of Human-cl rhFVIII measured using the one-stage (OS) assay
Mean Residence Time (MRT) of Human-cl rhFVIII
MRT of Human-cl rhFVIII measured using the one-stage (OS) assay
Clearance (CL) of Human-cl rhFVIII
CL of Human-cl rhFVIII measured using the one-stage (OS) assay
Volume of Distribution at Steady State (Vss) of Human-cl rhFVIII
Vss of Human-cl rhFVIII measured using the one-stage (OS) assay
Usage of Human-cl rhFVIII (FVIII IU/kg BW Per Week Per Patient)
Average weekly consumption of Human-cl rhFVIII reported as IU/kg BW per week per patient was determined during individualized prophylactic treatment
Number of Patients With Adverse Events (AEs)
AEs were documented at each (scheduled or unscheduled) study visit. Severity and seriousness of all AEs were documented by the investigator according to pre-defined criteria

Full Information

First Posted
September 30, 2014
Last Updated
December 21, 2020
Sponsor
Octapharma
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1. Study Identification

Unique Protocol Identification Number
NCT02256917
Brief Title
Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A
Official Title
Prospective, Open-label, Multi-centre Phase 3b Study to Assess the Efficacy and Safety of Personalized Prophylaxis With Human-cl rhFVIII in Previously Treated Adult Patients With Severe Haemophilia A
Study Type
Interventional

2. Study Status

Record Verification Date
December 2020
Overall Recruitment Status
Completed
Study Start Date
May 2015 (Actual)
Primary Completion Date
September 2018 (Actual)
Study Completion Date
September 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Octapharma

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
The rationale of this study is to further fine-tune and individualize prophylactic treatment of patients with severe Haemophilia A with the goal of keeping the trough FVIII level above 1% between doses. Because trough FVIII levels are likely to be important predictors of the efficacy of prophylaxis, the focus of this study is on pharmacokinetic (PK) data.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Severe Haemophilia A

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
58 (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
Annualized Total Bleeding Rate of Individually Tailored Prophylaxis
Description
Total annualized bleeding rate (ABR) of individually tailored prophylaxis (GENA-21b) compared to historical bleeding rate in patients having received on-demand treatment (GENA-01) with Human-cl rhFVIII
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Annualized Spontaneous Bleeding Rate of Individually Tailored Prophylaxis
Description
Spontaneous annualized bleeding rate (ABR) of individually tailored prophylaxis (GENA-21b) compared to historical bleeding rate in patients having received on-demand treatment (GENA-01) with Human-cl rhFVIII
Time Frame
6 months
Title
Annualized Total Bleeding Rate in Patients With 2x/Week (or Less) Prophylaxis
Description
Total annualized bleeding rate (ABR) in patients with 2x/week (or less) prophylaxis (GENA-21b) compared to historical bleeding rate in patients having received on-demand treatment (GENA-01) with Human-cl rhFVIII
Time Frame
6 months
Title
Median Prophylactic Dosing Interval
Description
Median over median actual dosing intervals between two prophylactic treatments per patient. The median time (hours) between two prophylactic doses of Human-cl rhFVIII in the prophylactic treatment Phase II per patient
Time Frame
6 months
Title
Mean Prophylactic Dosing Interval
Description
Mean over mean actual dosing intervals between two prophylactic treatments per patient. The mean time (hours) between two prophylactic doses of Human-cl rhFVIII in the prophylactic treatment Phase II per patient
Time Frame
6 months
Title
AUC Divided by the Dose (AUCnorm) of Human-cl rhFVIII
Description
AUCnorm of Human-cl rhFVIII measured using the one-stage (OS) assay
Time Frame
Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
Title
In-vivo Recovery (IVR) of Human-cl rhFVIII
Description
IVR of Human-cl rhFVIII measured using the one-stage (OS) assay and will be determined from the FVIII level before the infusion and the peak level after the infusion of Human-cl rhFVIII
Time Frame
Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
Title
Half Life (t1/2) of Human-cl rhFVIII
Description
T1/2 of Human-cl rhFVIII measured using the one-stage (OS) assay
Time Frame
Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
Title
Mean Residence Time (MRT) of Human-cl rhFVIII
Description
MRT of Human-cl rhFVIII measured using the one-stage (OS) assay
Time Frame
Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
Title
Clearance (CL) of Human-cl rhFVIII
Description
CL of Human-cl rhFVIII measured using the one-stage (OS) assay
Time Frame
Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
Title
Volume of Distribution at Steady State (Vss) of Human-cl rhFVIII
Description
Vss of Human-cl rhFVIII measured using the one-stage (OS) assay
Time Frame
Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
Title
Usage of Human-cl rhFVIII (FVIII IU/kg BW Per Week Per Patient)
Description
Average weekly consumption of Human-cl rhFVIII reported as IU/kg BW per week per patient was determined during individualized prophylactic treatment
Time Frame
6 months
Title
Number of Patients With Adverse Events (AEs)
Description
AEs were documented at each (scheduled or unscheduled) study visit. Severity and seriousness of all AEs were documented by the investigator according to pre-defined criteria
Time Frame
At each study visit over the study duration (7-9 months)

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Severe Haemophilia A (FVIII:C < 1%) Male patients >= 18 years of age Previous treatment with a FVIII concentrate for at least 150 EDs Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start Immunocompetence (CD4+ count > 200/uL) Exclusion Criteria: Any coagulation disorder other than Haemophilia A Present of past FVIII inhibitor activity Severe liver or kidney disease
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Craig M Kessler, MD
Organizational Affiliation
Georgetown University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Octapharma Research Site
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Octapharma Research Site
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Octapharma Research Site
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20057
Country
United States
Facility Name
Octapharma Research Site
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Octapharma Research Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Octapharma Research Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46260
Country
United States
Facility Name
Octapharma Research Site
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38163
Country
United States
Facility Name
Octapharma Research Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Octapharma Research Site
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
Octapharma Research Site
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G1C9
Country
Canada
Facility Name
Octapharma Research Site
City
St. John's
State/Province
Newfoundland and Labrador
Country
Canada
Facility Name
McMaster University
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 4K1
Country
Canada
Facility Name
University Hospital Centre Zagreb
City
Zagreb
Country
Croatia
Facility Name
Helsinki University Hospital
City
Helsinki
ZIP/Postal Code
00290
Country
Finland
Facility Name
Centre Régional de Traitement de l'Hémophilie
City
Bron
ZIP/Postal Code
69677
Country
France
Facility Name
CHU Estaing
City
Clermont-Ferrand
Country
France
Facility Name
Centre Hospitalier Universitaire Félix Guyon
City
La Réunion
ZIP/Postal Code
97400
Country
France
Facility Name
Centre Régional de Traitement de l'hémophilie
City
Nantes
ZIP/Postal Code
44093
Country
France
Facility Name
Hôpital Purpan - Centre de Traitment Regional de l'Hemophilie Pole
City
Toulouse
ZIP/Postal Code
31059
Country
France
Facility Name
Nagoya University Hospital
City
Nagoya
State/Province
Aichi
Country
Japan
Facility Name
Hospital of the Univ of Occupational and Environmental Health
City
Kitakyushu
State/Province
Fukuoka
Country
Japan
Facility Name
St. Marianna Univ School of Medicine Hospital
City
Kawasaki
State/Province
Kanagawa
Country
Japan
Facility Name
Nara Medical University Hospital
City
Kashihara
State/Province
Nara
Country
Japan
Facility Name
Gunma University Hospital
City
Maebashi
Country
Japan
Facility Name
Osaka National Hospital
City
Osaka
Country
Japan
Facility Name
Ogikubo Hospital
City
Tokyo
Country
Japan
Facility Name
Teikyo University Hospital
City
Tokyo
Country
Japan
Facility Name
University Medical Center Groningen
City
Groningen
ZIP/Postal Code
9713
Country
Netherlands
Facility Name
PHI Institute of Transfusion Medicine of Republic of Macedonia
City
Skopje
Country
North Macedonia
Facility Name
University Medical Centre Ljubljana
City
Ljubljana
Country
Slovenia

12. IPD Sharing Statement

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Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A

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