check_circleStudy Completed

Blood Coagulation Disorders, Hemophilia A

Bayer Identifier:

12954

ClinicalTrials.gov Identifier:

NCT01029340

EudraCT Number:

2009-012149-43

EU CT Number:

Not Available
Trial to Evaluate the Efficacy and Safety of a new full length recombinant human FVIII for Hemophilia A

Trial 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 BAY81-8973 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)

Key Participants Requirements

Sex

Male

Age

12 - 65 Years
  • - 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
  • - 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, Human immunodeficiency virus (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

Trial summary

Enrollment Goal
74
Trial Dates
December 2009 - March 2013
Phase
Phase 3
Could I Receive a placebo
No
Products
Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Terminated
Oxford, OX3 7LJ, United Kingdom
Completed
Sheffield, S10 2JF, United Kingdom
Completed
Sacramento, 95817, United States
Completed
Krakow, 31-501, Poland
Completed
A Coruña, 15006, Spain
Completed
Valencia, 46026, Spain
Terminated
Göteborg, 413 45, Sweden
Terminated
Malmö, 205 02, Sweden
Terminated
Karolinska Universitetssjukhuset i SolnaStockholm, 171 76, Sweden
Completed
Parktown, 2132, South Africa
Terminated
Wien, 1090, Austria
Terminated
Jakarta, 10430, Indonesia
Terminated
Bangalore, 34, India
Terminated
Pune, 411005, India
Completed
Tampa, 33607, United States
Completed
Boston, 02115, United States
Completed
Cleveland, 44106-2602, United States
Terminated
Orange, 92868, United States
Completed
Warszawa, 02-776, Poland
Completed
Barcelona, 08035, Spain
Terminated
Santander, 39008, Spain
Terminated
Jaén, 23007, Spain
Completed
DK-Aarhus N, 8200, Denmark
Terminated
Oslo, 0027, Norway
Completed
Pretoria, 0001, South Africa
Completed
Ramat Gan, 5262000, Israel
Terminated
Jakarta, 10430, Indonesia
Terminated
Mumbai, 400012, India
Terminated
Changhua, 500, Taiwan
Completed
Hongkong, Hong Kong
Terminated
Bangkok, 10400, Thailand
Terminated
Karachi, 75300, Pakistan
Terminated
Taipei, 11217, Taiwan
Terminated
Taipei, 10016, Taiwan
Terminated
Bangkok, 10330, Thailand
Completed
Bonn, 53127, Germany
Terminated
Mainz, 55131, Germany
Terminated
Frankfurt, 60596, Germany
Terminated
Magdeburg, 39112, Germany
Terminated
Dundee, DD1 9SY, United Kingdom
Terminated
East Lansing, 48823, United States
Completed
Kansas City, 64108-9898, United States
Completed
Milano, 20122, Italy
Completed
Catanzaro, 88100, Italy
Completed
Vicenza, 36100, Italy
Completed
Napoli, 80131, Italy
Terminated
Napoli, 80144, Italy
Terminated
Roma, 00168, Italy
Completed
Oviedo, 33006, Spain
Terminated
Bahia Blanca, B8001HXM, Argentina
Terminated
Rosario, S2000CKF, Argentina
Terminated
Graz, 8036, Austria
Completed
Izmir, 35-100, Turkey
Completed
Adana, 01330, Turkey
Completed
Antalya, 07059, Turkey
Terminated
Zagreb, 10000, Croatia
Terminated
Nis, 18000, Serbia
Terminated
Belgrade, 11000, Serbia
Terminated
Novi Sad, 21000, Serbia
Terminated
Beograd, 11000, Serbia
Terminated
London, SE1 7EH, United Kingdom
Terminated
Homburg, 66421, Germany

Primary Outcome

  • Part A - Area under the drug concentration-time curve (AUC)
    To examine the Pharmacokinetic (PK) characteristics of BAY 81-8973 and ensure that the new drug is similar to Kogenate FS. All results are based on the chromogenic assay.
    date_rangeTime Frame:
    Samples taken at pre-injection, and at 0.25, 0.5, 1, 3, 6, 8, 24, 30 and 48 hours post injection. AUC calculated from time of injection to infinity.
    enhanced_encryption
    Safety Issue:
    No
  • Part A - Half-life (t 1/2)
    To examine the PK characteristics of BAY81-8973 and ensure that the new drug is similar to Kogenate FS. All results are based on the chromogenic assay.
    date_rangeTime Frame:
    Samples taken at pre-injection, and at 0.25, 0.5, 1, 3, 6, 8, 24, 30 and 48 hours post injection.
    enhanced_encryption
    Safety Issue:
    No
  • Part B - Annualized number of total bleeds
    The annualized number of bleeds experienced by participants
    date_rangeTime Frame:
    12 months after randomization
    enhanced_encryption
    Safety Issue:
    No

Secondary Outcome

  • Part B - The in vivo recovery values of Human factor VIII (FVIII)
    The amount of Factor VIII found in blood samples taken after the injection of the study drug at the beginning of the CS/EP treatment period.
    date_rangeTime Frame:
    15-30 minutes after the injection
    enhanced_encryption
    Safety Issue:
    No
  • Part B - Annualized number of bleeds in each 6-month potency assignment period
    The annualized number of bleeds experienced by participants in each of the two treatment periods
    date_rangeTime Frame:
    6 months on each potency
    enhanced_encryption
    Safety Issue:
    No
  • Part B - Control of bleeding as measured by the number of injections required to treat a bleed
    The number of injections needed by participants to stop a bleed
    date_rangeTime Frame:
    6 months on each potency
    enhanced_encryption
    Safety Issue:
    No
  • Part B - changes from baseline at 12 months in quality of life (QoL) as measured by Transformed Total Score of Haemo-QoL questionnaire
    A measure of how treatment with BAY81-8973 affected the daily life of participants. the scoring system has 100 points. 0 is the worst possible score. 100 is the best possible score. Positive changes from baseline indicate an improvement in quality of life and negative changes indicate a deterioration.
    date_rangeTime Frame:
    Baseline and 12 months
    enhanced_encryption
    Safety Issue:
    No
  • Part B - Changes from baseline at 12 months in Utility Index as measured by EQ–5D questionaire
    A measure of how treatment with BAY81-8973 affected the daily life of participants. 1.0 = Best possible score, -0.594 = Worst possible score. Positive changes from baseline indicate an improvement and negative changes indicate a deterioration.
    date_rangeTime Frame:
    Baseline and 12 months
    enhanced_encryption
    Safety Issue:
    No
  • Part A - Number of participants with inhibitory antibody formation
    A test to ensure that participants have not developed antibodies that will interfere with the action of BAY81-8973
    date_rangeTime Frame:
    Up to 6 weeks after first injection of study drug
    enhanced_encryption
    Safety Issue:
    Yes
  • Part B - Number of participants with incidence of inhibitory antibody formation
    A test to ensure that participants have not developed antibodies that will interfere with the action of BAY81-8973
    date_rangeTime Frame:
    Up to 12 months after drug administration
    enhanced_encryption
    Safety Issue:
    Yes
  • Part C - Number of participants with incidence of inhibitory antibody formation
    A test to ensure that participants have not developed antibodies that will interfere with the action of BAY81-8973
    date_rangeTime Frame:
    before and 3 weeks after surgery
    enhanced_encryption
    Safety Issue:
    Yes
  • Part A - Number of participants with incidence of antibody formation to heat-shock protein (HSP-70)
    A test to analyze the formation of antibodies to HSP-70
    date_rangeTime Frame:
    Up to 6 weeks after drug administration
    enhanced_encryption
    Safety Issue:
    Yes
  • Part B - Number of participants with incidence of antibody formation to heat-shock protein (HSP-70)
    A test to analyze the formation of antibodies to HSP-70
    date_rangeTime Frame:
    Up to 12 months after drug administration
    enhanced_encryption
    Safety Issue:
    Yes
  • Part C - Number of participants with incidence of antibody formation to heat-shock protein (HSP-70)
    A test to analyze the formation of antibodies to HSP-70
    date_rangeTime Frame:
    before and 3 weeks after surgery
    enhanced_encryption
    Safety Issue:
    Yes
  • Part A - Number of participants with incidence of antibody formation to host cell proteins (HCP)
    A test to ensure that participants have not developed antibodies to HCP during the study
    date_rangeTime Frame:
    Up to 4 weeks after drug administration
    enhanced_encryption
    Safety Issue:
    Yes
  • Part B - Number of participants with incidence of antibody formation to host cell proteins (HCP)
    A test to ensure that participants have not developed antibodies to HCP during the study
    date_rangeTime Frame:
    Up to 12 months after drug administration
    enhanced_encryption
    Safety Issue:
    Yes
  • Part C - Number of participants with incidence of antibody formation to host cell proteins (HCP)
    A test to ensure that participants have not developed antibodies to HCP during the study
    date_rangeTime Frame:
    before and 3 weeks after surgery
    enhanced_encryption
    Safety Issue:
    Yes
  • Part B - Number of participants with assessment of the hemostasis during major surgery
    An assessment made by surgeons of how effective BAY81-8973 was in stopping bleeding during major operations
    date_rangeTime Frame:
    An average of 1 month after start of treatment
    enhanced_encryption
    Safety Issue:
    Yes
  • Part C - Number of participants with assessment of the hemostasis during major surgery
    An assessment made by surgeons of how effective BAY81-8973 was in stopping bleeding during major operations
    date_rangeTime Frame:
    at the time of surgery
    enhanced_encryption
    Safety Issue:
    Yes

Trial design

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
Trial Type
Interventional
Intervention Type
Biological/Vaccine
Trial Purpose
Treatment
Allocation
Randomized
Blinding
Open Label
Assignment
Crossover Assignment
Trial Arms
5

Additional Information

Click here and search for drug information provided by the FDA.Click here and search for information on any recalls, market or product safety alerts by the FDA which might have occurred with this product.Click here to find results for studies related to Bayer Healthcare products.Click here to find information about studies related to Bayer Healthcare products conducted in Europe