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Trial to Evaluate the Efficacy and Safety of a New Full Length Recombinant Human FVIII for Hemophilia A (Leopold I)

This study has been completed.
Sponsor:
Information provided by:
Bayer
ClinicalTrials.gov Identifier:
NCT01029340
First received: December 8, 2009
Last updated: April 16, 2013
Last verified: April 2013
History of Changes
  Purpose

The study will assess the pharmacokinetics (part A) safety, tolerability, and efficacy of prophylaxis treatment (2 to 3 times a week) (part B) with BAY 81-8973 over a one year period (split into two six month treatment periods). The study will compare 2 different methods (assays) for measuring the amount of study drug, the chromogenic substrate assay per European Pharmacopeia (CS/EP) with the classical assay (Chromogenic Substrate Adjusted, CS/ADJ). During one six month period patients will receive the study drug where the dose has been measured using the" (CS/EP) and during the other six months period the dose will be measured based on the Chromogenic Substrate Adjusted assay CS/ADJ)


Condition Intervention Phase
Blood Coagulation Disorders
Hemophilia A
 Biological: Recombinant Factor VIII (BAY81-8973)
Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
 Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Two Part Randomized Cross-Over Trial to Evaluate the Pharmacokinetics, Efficacy, and Safety Profile of Plasma Protein-Free Recombinant FVIII Formulated With Sucrose (BAY81-8973) in Previously Treated Subjects With Severe Hemophilia A Under Prophylaxis Therapy

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Bayer:

Primary Outcome Measures:
  • Part A: Pharmacokinetics after a single dose [ Time Frame: 48 hours ] [ Designated as safety issue: No ]
  • Part B: Number of all bleeds in one year [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • In vivo recovery (blood levels) of study drug 15 minutes after one dose [ Time Frame: 15 minutes ] [ Designated as safety issue: No ]
  • comparison of study drug potency determinations as measured by bleeding rate [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Potential for inhibitory antibody formation during prophylactic treatment with study drug [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
  • Potential for antibody formation to human heat shock protein 70 and hamster proteins during prophylactic treatment with study drug [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
  • Surgical outcomes during prophylactic treatment with study drug [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Control of bleeding with study drug [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Quality of life and pharmacoeconomic parameters during prophylactic treatment with study drug [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Enrollment: 76
Study Start Date: December 2009
Study Completion Date: March 2013
Primary Completion Date: June 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Arm 1 Biological: Recombinant Factor VIII (BAY81-8973)
Single dose of BAY81-8973 crossed over to single dose of Kogenate FS
Active Comparator: Arm 2 Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Single dose of Kogenate FS crossed over to Single dose of BAY81-8973
Experimental: Arm 3 Biological: Recombinant Factor VIII (BAY81-8973)
Prophylaxis with dosing 2-3 times per week with BAY81-8973 measured by Chromogenic Substrate Assay/Adjusted to Label Potency for 6 months crossed over to study drug measured by Chromogenic Substrate Assay Per European Pharmacopeia for 6 months
Experimental: Arm 4 Biological: Recombinant Factor VIII (BAY81-8973)
Prophylaxis with dosing 2-3 times per week with BAY81-8973 measured by Chromogenic Substrate Assay Potency Per European Pharmacopeia for 6 months crossed over to study drug measured by Chromogenic Substrate Assay/Adjusted to Label Potency for 6 months.
Experimental: Arm 5 Biological: Recombinant Factor VIII (BAY81-8973)
treatment for major surgical intervention for up to 3 weeks

  Eligibility

Ages Eligible for Study:   12 Years to 65 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male, aged 12 to 65 years
  • Severe hemophilia A defined as < 1% FVIII:C
  • >/= 150 days of previous treatment with FVIII in lifetime
  • Currently receiving on-demand or any type of prophylaxis treatment regimen with any FVIII product
  • No history of or current FVIII inhibitors

Exclusion Criteria:

  • Presence of another bleeding disease that is different from hemophilia A (e.g., von Willebrand disease, hemophilia B)
  • Low platelet count, abnormal kidney function, or liver disease
  • Received treatment with immune suppressing drugs within the last 3 months prior or requires treatment during the study. (Some drugs for hepatitis C, HIV, and steroids are allowed)
  • Receiving or has received other experimental drugs within 3 months prior to study entry
  • Allergy to Factor VIII or hamsters or mouse protein
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01029340

  Show 61 Study Locations
Sponsors and Collaborators
Bayer
Investigators
Study Director: Bayer Study Director Bayer
  More Information

Additional Information:
Click here and search for drug information provided by the FDA.  This link exits the ClinicalTrials.gov site
Click here and search for information on any recalls, market or product safety alerts by the FDA which might have occurred with this product.  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: Therapeutic Area Head, Bayer HealthCare Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT01029340     History of Changes
Other Study ID Numbers: 12954, 2009-012149-43
Study First Received: December 8, 2009
Last Updated: April 16, 2013
Health Authority: Austria: Agency for Health and Food Safety
China: Food and Drug Administration
Finland: Finnish Medicines Agency
Germany: Paul-Ehrlich-Institut
Hong Kong: Department of Health
Indonesia: National Agency of Drug and Food Control
Israel: Ministry of Health
Italy: The Italian Medicines Agency
Norway: Norwegian Medicines Agency
Spain: Spanish Agency of Medicines
Sweden: Medical Products Agency
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Taiwan: Department of Health
Denmark: Danish Medicines Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
United States: Food and Drug Administration

Keywords provided by Bayer:
Hemophilia A
Factor VIII
Prophylaxis

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders
Hemostatic Disorders
Hematologic Diseases
Vascular Diseases
Cardiovascular Diseases
Hemorrhagic Disorders
Blood Coagulation Disorders, Inherited
 Coagulation Protein Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 23, 2013