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

ID: NCT01775618
Status: Active, not recruiting
Phase: Phase 3
Start Date: May 29, 2013
First Submitted: January 23, 2013
Last Updated: February 22, 2018
Results: N/A
Organization: Bayer
Sponsors & Collaborators: Bayer
Location: Argentina, Austria, Belgium, Bulgaria, Canada, Greece, Israel, Italy, Lithuania, Netherlands, New Zealand, Norway, Poland, Romania, Spain, United Kingdom, United States
Conditions: Hemophilia A
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Study Description

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

Condition or disease Intervention/treatment Phase

Hemophilia A

Biological: BAY94-9027
Other Names
Biological: BAY94-9027
Other Names
Phase 3

Tracking Information

First Submitted DateJanuary 23, 2013
Last Update Posted DateFebruary 22, 2018
Actual Start DateMay 29, 2013
Anticipated Completion DateFebruary 17, 2020
Actual Primary Completion DateMarch 19, 2015
Results First Submitted DateN/A
Received Results Disposit DateN/A

Current Primary Outcome Measures

  • Characterization of a potential immune response [Time Frame: Up to 12 weeks]

  • Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent) [Time Frame: Up to 11 months]

  • Pharmacokinetics 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]

    Pharmacokinetics profile includes maximum concentration (Cmax), incremental recovery, mean residence time (MRT), apparent volume of distribution at steady state (Vss), half-life, area under the curve (AUC), and clearance, which are calculated values based on time-weighed blood concentration of drug.

  • Annualized number of all bleeds [Time Frame: Up to 11 months]

Original Primary Outcome Measures

Not Available

Current Secondary Outcome Measures

  • 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]

Original Secondary Outcome Measures

Not Available

Study Design

Brief TitleSafety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A
Official TitleA 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

Study TypeInterventional
Study PhasePhase 3
Estimated Enrollment
73
Allocation
Non-Randomized
Interventional Model
Single Group Assignment
Masking
None (Open Label)
Primary Purpose
Treatment
Conditions
Hemophilia A
Target Follow-Up Duration N/A
Biospecimen:
N/A
Sampling MethodN/A
Study PopulationN/A
Intervention
Biological: BAY94-9027

Prophylaxis treatment 25-60 IU/kg at least 1x/week.

Other Names
Biological: BAY94-9027

Prophylaxis treatment 25-60 IU/kg 2x/week for 12 weeks.

Other Names
Study Groups/Cohorts
BAY 94-9027
Patients will receive BAY 94-9027 intravenous prophylaxis injection in the main study.

Expansion group (Part 2)
Patients in expansion group will receive BAY 94-9027 intravenous prophylaxis injection in study part 2 .

Study Arms
Experimental BAY 94-9027
Patients will receive BAY 94-9027 intravenous prophylaxis injection in the main study.
Biological : BAY94-9027
Prophylaxis treatment 25-60 IU/kg at least 1x/week.

Experimental Expansion group (Part 2)
Patients in expansion group will receive BAY 94-9027 intravenous prophylaxis injection in study part 2 .
Biological : BAY94-9027
Prophylaxis treatment 25-60 IU/kg 2x/week for 12 weeks.

Arm Intervention/Treatment
Experimental BAY 94-9027
Patients will receive BAY 94-9027 intravenous prophylaxis injection in the main study.
Biological : BAY94-9027
Experimental Expansion group (Part 2)
Patients in expansion group will receive BAY 94-9027 intravenous prophylaxis injection in study part 2 .
Biological : BAY94-9027

Recruitment Information

Recruitment Status:Active, not recruiting
Enrollment73
Completion DateFebruary 17, 2020
Eligibility 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/mm3
- Creatinine > 2x the upper limit of normal
- Aspartate aminotransferase (AST) / Alanine aminotransferase (ALT) > 5x the upper limit of normal
GenderMale
Age N/A to 12 Years
Accepts Healthy VolunteersNo
Contacts
Not Available
Listed Location Countries
Argentina
Austria
Belgium
Bulgaria
Canada
Greece
Israel
Italy
Lithuania
Netherlands
New Zealand
Norway
Poland
Romania
Spain
United Kingdom
United States
Germany
Hungary
Slovenia

Administrative Information

NCT Number:NCT01775618
Other Study ID Numbers
15912
2012-004434-42
Has Data Monitoring CommitteeYes
U.S. FDA-regulated Product Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Device Product Not Approved or Cleared by U.S. FDA: No
IPD Sharing Statement
Not Available
Responsible Party,
Study Sponsor
Bayer
Collaborators
Not Available
Investigators
Study Director
Bayer Study Director
Bayer