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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

Tracking Information
First Submitted Date  ICMJE January 23, 2013
First Posted Date  ICMJE January 25, 2013
Last Update Posted Date August 21, 2020
Actual Study Start Date  ICMJE May 29, 2013
Actual Primary Completion Date March 19, 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 19, 2020)
  • Annualized number of all bleeds [ Time Frame: At least 50 exposure days (ED) over 6 months, on average 245 days ]
  • 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)
  • 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 ]
  • Characterization of a potential immune response [ Time Frame: 12 weeks ]
  • Inhibitor development in the extension study [ Time Frame: At least 50 additional EDs to achieve at least 100 cumulative EDs, on average 5 years ]
Original Primary Outcome Measures  ICMJE
 (submitted: January 23, 2013)
  • Annualized number of all bleeds [ Time Frame: Up to 11 months ]
  • Pharmacokinetic profile of BAY94-9027 based on blood concentration over the defined time period [ Time Frame: 6 time points from pre-infusion to 72 hours post-infusion ]
  • Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent) [ Time Frame: Up to 11 months ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 19, 2020)
  • Inhibitor development in the main study [ Time Frame: After 10 to 15 and 50 exposure days (ED) over 6 months, on average 245 days ]
  • Assessment of incremental recovery in main study [ Time Frame: At least 50 exposure days (ED) over 6 months, on average 245 days ]
  • 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) ]
Original Secondary Outcome Measures  ICMJE
 (submitted: January 23, 2013)
  • Inhibitor development after 10 to 15 EDs (exposure days) [ Time Frame: Up to 15 weeks ]
  • Inhibitor development after 50 EDs [ Time Frame: Up to 11 months ]
  • Number of participants with adverse events as a measure of safety and tolerability [ Time Frame: Up to 11 months ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A
Official Title  ICMJE 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
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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hemophilia A
Intervention  ICMJE
  • 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
  • Biological: BAY94-9027
    Twice per week prophylaxis: 25-60 IU/kg, intravenous infusion, for 12 weeks
  • 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
Study Arms  ICMJE
  • 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.
    Intervention: Biological: BAY94-9027
  • 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.
    Intervention: Biological: BAY94-9027
  • 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.
    Intervention: Biological: BAY94-9027
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 23, 2017)
73
Original Estimated Enrollment  ICMJE
 (submitted: January 23, 2013)
50
Actual Study Completion Date  ICMJE February 19, 2020
Actual Primary Completion Date March 19, 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE up to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Austria,   Belgium,   Bulgaria,   Canada,   Greece,   Israel,   Italy,   Lithuania,   Netherlands,   New Zealand,   Norway,   Poland,   Romania,   Spain,   United Kingdom,   United States
Removed Location Countries Germany,   Hungary,   Slovenia
 
Administrative Information
NCT Number  ICMJE NCT01775618
Other Study ID Numbers  ICMJE 15912
2012-004434-42 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Bayer
Study Sponsor  ICMJE Bayer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Bayer Study Director Bayer
PRS Account Bayer
Verification Date August 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP