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Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01775618
Recruitment Status : Completed
First Posted : January 25, 2013
Last Update Posted : August 21, 2020
Sponsor:
Information provided by (Responsible Party):
Bayer

Brief Summary:

Hemophilia A is an inherited blood disorder in which one protein, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. Hemophilia A causes the clotting process to be slowed and the person experiences bleeds causing serious problems that could lead to disability. The current standard treatment for severe hemophilia A is infusion of FVIII to stop bleeding, or regular scheduled treatment to prevent bleeds from occuring. Due to the short half-life of FVIII, prophylaxis may require treatment as often as every other day.

In this trial safety and efficacy of a long-acting recombinant Factor VIII molecule is being evaluated in 50 male subjects, < 12 years of age, with severe Hemophilia A. These subjects will receive open label treatment with long-acting rFVIII for approximately 6 months (or longer until 50 exposure days) on a regular schedule at least once every 7-days. Doses and dose intervals may be adapted to the subject's clinical need. A second group of patients will receive open label treatment with the same drug for 12 weeks on a regular schedule of 2x/week. Patients will attend the treatment center for routine blood samples and will be required to keep an electronic diary.

Subjects will be offered participation in an optional extension study to collect observations for at least an additional 50 exposure days.


Condition or disease Intervention/treatment Phase
Hemophilia A Biological: BAY94-9027 Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 73 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multi-center, Phase III, Non-controlled, Open-label Trial to Evaluate the Pharmacokinetics, Safety, and Efficacy of BAY94-9027 for Prophylaxis and Treatment of Bleeding in Previously Treated Children (Age <12 Years) With Severe Hemophilia A
Actual Study Start Date : May 29, 2013
Actual Primary Completion Date : March 19, 2015
Actual Study Completion Date : February 19, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arm Intervention/treatment
Experimental: Main study
Participants were treated and prophylaxis administered with BAY94-9027 at a dose of 25-60 international units/kilogram (IU/kg) twice per week or 45-60 IU/kg every 5 days or 60 IU/kg every 7 days as an intravenous (IV) infusion as per clinical needs of each subject up to at least 50 exposure days (EDs) and a minimum of at least 6 months.
Biological: BAY94-9027
Study drug dosing was adjusted to the clinical needs of each subject in the range of 25-60 IU/kg/administration, intravenous infusion, at least 50 EDs and a minimum of at least 6 months

Experimental: Part 2 (Expansion group)
Participants were administered with BAY94-9027 at a dose of 25-60 IU/kg twice per week for prophylaxis for 12 weeks.
Biological: BAY94-9027
Twice per week prophylaxis: 25-60 IU/kg, intravenous infusion, for 12 weeks

Experimental: Extension study
Participants were treated and prophylaxis administered with BAY94-9027 at a dose of 25- 60 IU/kg twice per week or 45-60 IU/kg every 5 days or 60 IU/kg every 7 days as an IV infusion as per clinical needs of each subject for at least 50 EDs or until marketing authorization of the drug.
Biological: BAY94-9027
Study drug dosing was adjusted to the clinical needs of each subject in the range of 25-60 IU/kg/administration, intravenous infusion, at least 50 additional EDs to achieve at least 100 cumulative EDs, or until marketing authorization of the drug




Primary Outcome Measures :
  1. Annualized number of all bleeds [ Time Frame: At least 50 exposure days (ED) over 6 months, on average 245 days ]
  2. Pharmacokinetics profile of BAY94-9027 based on blood concentration over the defined time period [ Time Frame: Pre-dose to 72 hours post-dose ]
    Pharmacokinetics profile includes maximum concentration (Cmax), half-life (t1/2), area under the concentration versus time curve (AUC), mean residence time (MRT), volume of distribution at steady state (Vss), and clearance (CL)

  3. Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent) [ Time Frame: At least 50 exposure days (ED) over 6 months, on average 245 days ]
  4. Characterization of a potential immune response [ Time Frame: 12 weeks ]
  5. Inhibitor development in the extension study [ Time Frame: At least 50 additional EDs to achieve at least 100 cumulative EDs, on average 5 years ]

Secondary Outcome Measures :
  1. Inhibitor development in the main study [ Time Frame: After 10 to 15 and 50 exposure days (ED) over 6 months, on average 245 days ]
  2. Assessment of incremental recovery in main study [ Time Frame: At least 50 exposure days (ED) over 6 months, on average 245 days ]
  3. Number of participants with adverse events as a measure of safety and tolerability [ Time Frame: From the start of study treatment up to 7 days after the last dose (Main study: on average 245+7 days; Part 2: 12 weeks+7 days; Extension study: on average 5 years+7 days) ]


Information from the National Library of Medicine

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Ages Eligible for Study:   up to 12 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Males < 12 years of age
  • Subjects with severe hemophilia A
  • Previously treated with FVIII for > 50 exposure days

Exclusion Criteria:

  • Subjects with current evidence of or history of inhibitors to FVIII
  • Any other inherited or acquired bleeding disorder
  • Platelet counts < 100,000/mm^3
  • Creatinine > 2x the upper limit of normal
  • Aspartate aminotransferase (AST) / Alanine aminotransferase (ALT) > 5x the upper limit of normal

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01775618


Locations
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United States, California
Sacramento, California, United States, 95817
United States, Florida
Pensacola, Florida, United States, 32504
United States, Ohio
Cincinnati, Ohio, United States, 45229
Cleveland, Ohio, United States, 44106-6007
Columbus, Ohio, United States, 43205-2696
United States, Pennsylvania
Hershey, Pennsylvania, United States, 17033
United States, Utah
Salt Lake City, Utah, United States, 84113
Argentina
La Plata, Buenos Aires, Argentina, 1900
Austria
Wien, Austria, 1090
Belgium
Gent, Belgium, 9000
Leuven, Belgium, 3000
Bulgaria
Plovdiv, Bulgaria, 4002
Sofia, Bulgaria, 1527
Varna, Bulgaria, 9010
Canada, Alberta
Calgary, Alberta, Canada, T3B 6A8
Canada, Ontario
Toronto, Ontario, Canada, M5G 1X8
Greece
Thessaloniki, Greece, 54642
Israel
Ramat Gan, Israel, 5262000
Italy
Milano, Lombardia, Italy, 20122
Palermo, Sicilia, Italy, 90127
Padova, Veneto, Italy, 35128
Lithuania
Vilnius, Lithuania, 08661
Netherlands
Amsterdam, Netherlands
Utrecht, Netherlands, 3584 CX
New Zealand
Christchurch, New Zealand, 8011
Hamilton, New Zealand, 3204
Norway
Oslo, Norway, 0027
Poland
Lodz, Poland, 91-738
Olsztyn, Poland, 10-561
Romania
Bucharest, Romania, 011026
Bucharest, Romania, 022328
Timisoara, Romania, 300011
Spain
Esplugues de LLobregat, Barcelona, Spain, 08950
United Kingdom
Newcastle Upon Tyne, Tyne And Wear, United Kingdom, NE1 4LP
Bristol, United Kingdom, BS2 8AE
Manchester, United Kingdom, M13 9WL
Sheffield, United Kingdom, S10 2TH
Sponsors and Collaborators
Bayer
Investigators
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Study Director: Bayer Study Director Bayer
Additional Information:
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Responsible Party: Bayer
ClinicalTrials.gov Identifier: NCT01775618    
Other Study ID Numbers: 15912
2012-004434-42 ( EudraCT Number )
First Posted: January 25, 2013    Key Record Dates
Last Update Posted: August 21, 2020
Last Verified: August 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Bayer:
Hemophilia A
Factor VIII
Prophylaxis
Pediatric
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants