A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A (Leopold II)

October 14, 2016 updated by: Bayer

A Phase II/III, Randomized, Cross-over, Open-label Trial to Demonstrate Superiority of Prophylaxis Over On-demand Therapy in Previously Treated Subjects With Severe Hemophilia A Treated With Plasma Protein-free Recombinant FVIII Formulated With Sucrose (BAY 81-8973)

The objective of the trial is to demonstrate that 2-3 times per week prophylaxis therapy with BAY81-8973 is superior to on-demand therapy with BAY81-8973 in patients with severe Hemophilia A. The hypothesis is that prophylaxis will result in fewer bleeds than on-demand treatment.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

80

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

  • Argentina
      • Corrientes, Argentina, W3410AVV
    • Ciudad Auton. de Buenos Aires
      • Buenos Aires, Ciudad Auton. de Buenos Aires, Argentina, C1221ADC
    • Santa Fe
      • Rosario, Santa Fe, Argentina, S2000CKF
  • China
      • Beijing, China, 100730
      • Shanghai, China, 200025
      • Tianjin, China, 300020
    • Guangdong
      • Guangzhou, Guangdong, China, 510515
    • Jiangsu
      • Suzhou, Jiangsu, China, 215006
  • Colombia
    • Distrito Capital de Bogotá
      • Bogotá, Distrito Capital de Bogotá, Colombia
    • Santander
      • Bucaramanga, Santander, Colombia
  • Czech Republic
      • Olomouc, Czech Republic, 77520
  • India
      • Hyderabad, India, 500034
  • Indonesia
      • Jakarta, Indonesia, 10430
  • Japan
      • Hiroshima, Japan, 734-8551
    • Hyogo
      • Nishinomiya, Hyogo, Japan, 663-8501
    • Nara
      • Kashihara, Nara, Japan, 634-8522
    • Tokyo
      • Shinjuku-ku, Tokyo, Japan, 160-0023
      • Suginami, Tokyo, Japan, 167-0035
  • Mexico
      • San Luis Potosí, Mexico, 78200
    • Jalisco
      • Guadalajara, Jalisco, Mexico, 44280
  • Romania
      • Baia Mare, Romania, 430031
      • Bucharest, Romania, 022328
      • Bucharest, Romania, 011026
    • Timis
      • Timisoara, Timis, Romania, 300011
  • Russian Federation
      • Barnaul, Russian Federation, 656050
      • Khabarovsk, Russian Federation, 680009
      • St. Petersburg, Russian Federation, 191186
      • Yekaterinburg, Russian Federation, 620149
  • Serbia
      • Beograd, Serbia, 11000
      • Kragujevac, Serbia, 34000
      • Nis, Serbia, 18000
      • Novi Sad, Serbia, 21000
  • Slovakia
      • Bratislava, Slovakia, 851 07
  • South Africa
    • Gauteng
      • Johannesburg, Gauteng, South Africa, 2193
      • Pretoria, Gauteng, South Africa, 0001
  • Taiwan
      • Taichung, Taiwan, 40447
      • Taipei, Taiwan, 11217
  • Thailand
      • Bangkok, Thailand, 10400
      • Bangkok, Thailand, 10700
  • Turkey
      • Adana, Turkey, 01330
      • Antalya, Turkey, 07059
      • Izmir, Turkey, 35-100
  • Ukraine
      • Kiev, Ukraine
      • Lviv, Ukraine, 79044
      • Simferopol, Ukraine, 95023
  • United States
    • Ohio
      • Dayton, Ohio, United States, 45404
    • Texas
      • Houston, Texas, United States, 77030

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

12 years to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  • Male, aged 12 to 65 years
  • Severe hemophilia A
  • History of more than 150 exposure days (ED) with clotting factor concentrates
  • Currently receiving episodic treatment with FVIII; no regular prophylaxis for more than 6 consecutive months in the past 5 years
  • No current Factor VIII inhibitor or history of inhibitor
  • Willing to use electronic patient diary

Exclusion Criteria:

  • Presence of another bleeding disease that is different from hemophilia A
  • Thrombocytopenia
  • Abnormal renal function
  • Presence of active liver disease
  • Known hypersensitivity to FVIII

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: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Arm 1: rFVIII on demand first CS/EP then CS/ADJ
Participants received on-demand treatment with recombinant factor VIII (rFVIII, BAY81-8973) assayed by CS/EP (Chromogenic Substrate Assay per European Pharmacopoeia) for 6 months, followed by cross-over to study drug assayed by CS/ADJ (Chromogenic Substrate Assay/label adjusted to one-stage assay) for 6 months.
Biological: rFVIII (BAY81-8973) on demand
Participants received on-demand treatment with rFVIII (BAY81-8973) assayed by CS/EP (Chromogenic Substrate Assay per European Pharmacopoeia) for 6 months and by CS/ADJ (Chromogenic Substrate Assay/label adjusted to one-stage assay) for 6 months, sequence according to randomization
Experimental: Arm 2: rFVIII on demand first CS/ADJ then CS/EP
Participants received on-demand treatment with rFVIII (BAY81-8973) assayed by CS/ADJ for 6 months, followed by cross-over to study drug assayed by CS/EP for 6 months.
Biological: rFVIII (BAY81-8973) on demand
Participants received on-demand treatment with rFVIII (BAY81-8973) assayed by CS/EP (Chromogenic Substrate Assay per European Pharmacopoeia) for 6 months and by CS/ADJ (Chromogenic Substrate Assay/label adjusted to one-stage assay) for 6 months, sequence according to randomization
Experimental: Arm 3: rFVIII prophylaxis low-dose first CS/EP then CS/ADJ
Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII(BAY81-8973) measured by CS/ EP for 6 months then crossed over to study drug measured by CS/ADJ for 6 months.
Biological: rFVIII (BAY81-8973) prophylaxis low-dose
Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.
Experimental: Arm 4: rFVIII prophylaxis low-dose first CS/ADJ then CS/EP
Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII (BAY81-8973) measured by CS/ADJ for 6 months then crossed over to study drug measured by CS/ EP for 6 months.
Biological: rFVIII (BAY81-8973) prophylaxis low-dose
Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.
Experimental: Arm 5: rFVIII prophylaxis high-dose first CS/EP then CS/ADJ
Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII (BAY81-8973) measured by CS/ EP for 6 months then crossed over to study drug measured by CS/ADJ for 6 months.
Biological: rFVIII (BAY81-8973) prophylaxis high-dose
Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.
Experimental: Arm 6: rFVIII prophylaxis high-dose first CS/ADJ then CS/EP
Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII(BAY81-8973) measured by CS/ADJ for 6 months then crossed over to study drug measured by CS/ EP for 6 months.
Biological: rFVIII (BAY81-8973) prophylaxis high-dose
Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized Number of All Bleeds
Time Frame: Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)
The annualized number of bleeds experienced by participants
Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized Number of All Bleeds During CS/EP Period
Time Frame: Up to 6 months (6 months on CS/EP potency assignment)
The annualized number of bleeds experienced by participants while they were taking rFVIII (BAY81-8973) assayed by CS/EP
Up to 6 months (6 months on CS/EP potency assignment)
Annualized Number of All Bleeds During CS/ADJ Period
Time Frame: Up to 6 months (6 months on CS/ADJ potency assignment)
The annualized number of bleeds experienced by participants while they were taking rFVIII (BAY81-8973) assayed by CS/ADJ
Up to 6 months (6 months on CS/ADJ potency assignment)
Percentage of Bleeds Per Participant Controlled With ≤ 2 Injections in Participants Treated on Demand With rFVIII (BAY81-8973)
Time Frame: Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)
The percentage of bleeds per participant on on-demand treatment that stopped after two or fewer injections
Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Bleeds During Treatment
Time Frame: 12 months
The number of bleeds experienced by each participant
12 months
Number of Participants With Inhibitory Antibody Formation
Time Frame: 3, 6, 9 and 12 months after baseline
A test to ensure that participants have not developed antibodies that will interfere with the action of rFVIII (BAY81-8973)
3, 6, 9 and 12 months after baseline

Collaborators and Investigators

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

Sponsor

Bayer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

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

January 1, 2011

Primary Completion (Actual)

December 1, 2012

Study Completion (Actual)

December 1, 2012

Study Registration Dates

First Submitted

November 2, 2010

First Submitted That Met QC Criteria

November 2, 2010

First Posted (Estimate)

November 3, 2010

Study Record Updates

Last Update Posted (Estimate)

November 28, 2016

Last Update Submitted That Met QC Criteria

October 14, 2016

Last Verified

October 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 14319
  • 2009-012150-20 (EudraCT Number)

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.

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