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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
80

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 1, 2011

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2012

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2012

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 2, 2010

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 2, 2010

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 3, 2010

Study Record Updates

Last Update Posted (Estimate)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 28, 2016

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 14, 2016

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
October 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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