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

August 19, 2020 updated by: Bayer

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

73

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
    • Buenos Aires
      • 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
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
  • Greece
      • Thessaloniki, Greece, 54642
  • Israel
      • Ramat Gan, Israel, 5262000
  • Italy
    • Lombardia
      • Milano, Lombardia, Italy, 20122
    • Sicilia
      • Palermo, Sicilia, Italy, 90127
    • Veneto
      • 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, 022328
      • Bucharest, Romania, 011026
      • Timisoara, Romania, 300011
  • Spain
    • Barcelona
      • Esplugues de LLobregat, Barcelona, Spain, 08950
  • United Kingdom
      • Bristol, United Kingdom, BS2 8AE
      • Manchester, United Kingdom, M13 9WL
      • Sheffield, United Kingdom, S10 2TH
    • Tyne And Wear
      • Newcastle Upon Tyne, Tyne And Wear, United Kingdom, NE1 4LP
  • United States
    • California
      • Sacramento, California, United States, 95817
    • Florida
      • Pensacola, Florida, United States, 32504
    • Ohio
      • Cincinnati, Ohio, United States, 45229
      • Cleveland, Ohio, United States, 44106-6007
      • Columbus, Ohio, United States, 43205-2696
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
    • Utah
      • Salt Lake City, Utah, United States, 84113

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 12 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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
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
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
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
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 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized number of all bleeds
Time Frame: At least 50 exposure days (ED) over 6 months, on average 245 days
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)
Pre-dose to 72 hours post-dose
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
At least 50 exposure days (ED) over 6 months, on average 245 days
Characterization of a potential immune response
Time Frame: 12 weeks
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
At least 50 additional EDs to achieve at least 100 cumulative EDs, on average 5 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Inhibitor development in the main study
Time Frame: After 10 to 15 and 50 exposure days (ED) over 6 months, on average 245 days
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
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)
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)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Bayer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 29, 2013

Primary Completion (Actual)

March 19, 2015

Study Completion (Actual)

February 19, 2020

Study Registration Dates

First Submitted

January 23, 2013

First Submitted That Met QC Criteria

January 23, 2013

First Posted (Estimate)

January 25, 2013

Study Record Updates

Last Update Posted (Actual)

August 21, 2020

Last Update Submitted That Met QC Criteria

August 19, 2020

Last Verified

August 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 15912
  • 2012-004434-42 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Hemophilia A

Clinical Trials on BAY94-9027

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Luxembourg

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe