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

December 21, 2020 updated by: Octapharma

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

58

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G1C9
        • Octapharma Research Site
    • Newfoundland and Labrador
      • St. John's, Newfoundland and Labrador, Canada
        • Octapharma Research Site
    • Ontario
      • Hamilton, Ontario, Canada, L8N 4K1
        • McMaster University
  • Croatia
      • Zagreb, Croatia
        • University hospital centre Zagreb
  • Finland
      • Helsinki, Finland, 00290
        • Helsinki University Hospital
  • France
      • Bron, France, 69677
        • Centre Régional de Traitement de l'hémophilie
      • Clermont-Ferrand, France
        • CHU Estaing
      • La Réunion, France, 97400
        • Centre Hospitalier Universitaire Félix Guyon
      • Nantes, France, 44093
        • Centre Régional de Traitement de l'hémophilie
      • Toulouse, France, 31059
        • Hôpital Purpan - Centre de Traitment Regional de l'Hemophilie Pole
  • Japan
      • Maebashi, Japan
        • Gunma University Hospital
      • Osaka, Japan
        • Osaka National Hospital
      • Tokyo, Japan
        • Ogikubo Hospital
      • Tokyo, Japan
        • Teikyo University Hospital
    • Aichi
      • Nagoya, Aichi, Japan
        • Nagoya University Hospital
    • Fukuoka
      • Kitakyushu, Fukuoka, Japan
        • Hospital of the Univ of Occupational and Environmental Health
    • Kanagawa
      • Kawasaki, Kanagawa, Japan
        • St. Marianna Univ School of Medicine Hospital
    • Nara
      • Kashihara, Nara, Japan
        • Nara Medical University Hospital
  • Netherlands
      • Groningen, Netherlands, 9713
        • University Medical Center Groningen
  • North Macedonia
      • Skopje, North Macedonia
        • PHI Institute of Transfusion Medicine of Republic of Macedonia
  • Slovenia
      • Ljubljana, Slovenia
        • University Medical Centre Ljubljana
  • United States
    • California
      • Sacramento, California, United States, 95817
        • Octapharma Research Site
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Octapharma Research Site
    • District of Columbia
      • Washington, District of Columbia, United States, 20057
        • Octapharma Research Site
    • Florida
      • Miami, Florida, United States, 33136
        • Octapharma Research Site
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Octapharma Research Site
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Octapharma Research Site
    • Tennessee
      • Memphis, Tennessee, United States, 38163
        • Octapharma Research Site
    • Texas
      • Houston, Texas, United States, 77030
        • Octapharma Research Site
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Octapharma Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Human-cl rhFVIII
Biological: Human cl rhFVIII

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized Total Bleeding Rate of Individually Tailored Prophylaxis
Time Frame: 6 months
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
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized Spontaneous Bleeding Rate of Individually Tailored Prophylaxis
Time Frame: 6 months
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
6 months
Annualized Total Bleeding Rate in Patients With 2x/Week (or Less) Prophylaxis
Time Frame: 6 months
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
6 months
Median Prophylactic Dosing Interval
Time Frame: 6 months
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
6 months
Mean Prophylactic Dosing Interval
Time Frame: 6 months
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
6 months
AUC Divided by the Dose (AUCnorm) 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
AUCnorm of Human-cl rhFVIII measured using the one-stage (OS) assay
Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
In-vivo Recovery (IVR) 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
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
Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
Half Life (t1/2) 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
T1/2 of Human-cl rhFVIII measured using the one-stage (OS) assay
Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
Mean Residence Time (MRT) 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
MRT of Human-cl rhFVIII measured using the one-stage (OS) assay
Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
Clearance (CL) 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
CL of Human-cl rhFVIII measured using the one-stage (OS) assay
Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
Volume of Distribution at Steady State (Vss) 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
Vss of Human-cl rhFVIII measured using the one-stage (OS) assay
Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection
Usage of Human-cl rhFVIII (FVIII IU/kg BW Per Week Per Patient)
Time Frame: 6 months
Average weekly consumption of Human-cl rhFVIII reported as IU/kg BW per week per patient was determined during individualized prophylactic treatment
6 months
Number of Patients With Adverse Events (AEs)
Time Frame: At each study visit over the study duration (7-9 months)
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
At each study visit over the study duration (7-9 months)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Octapharma

Investigators

  • Principal Investigator: Craig M Kessler, MD, Georgetown University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 1, 2015

Primary Completion (Actual)

September 1, 2018

Study Completion (Actual)

September 1, 2018

Study Registration Dates

First Submitted

September 30, 2014

First Submitted That Met QC Criteria

October 1, 2014

First Posted (Estimate)

October 6, 2014

Study Record Updates

Last Update Posted (Actual)

January 19, 2021

Last Update Submitted That Met QC Criteria

December 21, 2020

Last Verified

December 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GENA-21B

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Severe Haemophilia A

Clinical Trials on Human cl rhFVIII

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Qatar

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe