check_circleStudy Completed

Hemophilia A

Bayer Identifier:

15912

ClinicalTrials.gov Identifier:

NCT01775618

EudraCT Number:

2012-004434-42

EU CT Number:

Not Available
Safety and Efficacy of BAY94-9027 in Previously Treated Male Children with Haemophilia A

Trial purpose

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.

Key Participants Requirements

Sex

Male

Age

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

Trial summary

Enrollment Goal
73
Trial Dates
May 2013 - February 2020
Phase
Phase 3
Could I Receive a placebo
No
Products
Jivi (Damoctocog, BAY94-9027)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Withdrawn
Debrecen, 4032, Hungary
Withdrawn
Budapest, 1089, Hungary
Completed
Milano, 20122, Italy
Completed
Padova, 35128, Italy
Completed
Palermo, 90127, Italy
Completed
Ramat Gan, 5262000, Israel
Completed
UTRECHT, 3584 CX, Netherlands
Completed
AMSTERDAM, Netherlands
Withdrawn
Boston, 02115, United States
Completed
Columbus, 43205-2696, United States
Completed
Varna, 9010, Bulgaria
Completed
Plovdiv, 4002, Bulgaria
Completed
Sofia, 1527, Bulgaria
Completed
Cincinnati, 45229, United States
Completed
Cleveland, 44106-6007, United States
Completed
Hershey, 17033, United States
Withdrawn
Chicago, 60612, United States
Completed
LEUVEN, 3000, Belgium
Completed
La Plata, 1900, Argentina
Completed
Timisoara, 300011, Romania
Completed
Bucharest, 011026, Romania
Completed
Bucharest, 022328, Romania
Withdrawn
Little Rock, 72202, United States
Completed
GENT, 9000, Belgium
Withdrawn
Corrientes, 3400, Argentina
Completed
Newcastle Upon Tyne, NE1 4LP, United Kingdom
Completed
Lodz, 91-738, Poland
Withdrawn
Szczecin, 71-252, Poland
Completed
Olsztyn, 10-561, Poland
Completed
Vilnius, 08661, Lithuania
Completed
Sacramento, 95817, United States
Completed
Manchester, M13 9WL, United Kingdom
Completed
Sheffield, S10 2TH, United Kingdom
Completed
Toronto, M5G 1X8, Canada
Completed
Calgary, T3B 6A8, Canada
Completed
Wien, 1090, Austria
Completed
Pensacola, 32504, United States
Completed
Bristol, BS2 8AE, United Kingdom
Withdrawn
Frankfurt, 60596, Germany
Completed
Esplugues de LLobregat, 08950, Spain
Withdrawn
Sevilla, 41013, Spain
Completed
Christchurch, 8011, New Zealand
Completed
Oslo, 0027, Norway
Completed
Thessaloniki, 54642, Greece
Withdrawn
Ljubljana, 1525, Slovenia
Completed
Hamilton, 3204, New Zealand
Completed
Salt Lake City, 84113, United States

Primary Outcome

  • Annualized number of all bleeds
    date_rangeTime Frame:
    At least 50 exposure days (ED) over 6 months, on average 245 days
    enhanced_encryption
    Safety Issue:
    No
  • Pharmacokinetics profile of BAY94-9027 based on blood concentration over the defined time period
    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)
    date_rangeTime Frame:
    Pre-dose to 72 hours post-dose
    enhanced_encryption
    Safety Issue:
    No
  • Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent)
    date_rangeTime Frame:
    At least 50 exposure days (ED) over 6 months, on average 245 days
    enhanced_encryption
    Safety Issue:
    No
  • Characterization of a potential immune response
    date_rangeTime Frame:
    12 weeks
    enhanced_encryption
    Safety Issue:
    Yes
  • Inhibitor development in the extension study
    date_rangeTime Frame:
    At least 50 additional EDs to achieve at least 100 cumulative EDs, on average 5 years

Secondary Outcome

  • Inhibitor development in the main study
    date_rangeTime Frame:
    After 10 to 15 and 50 exposure days (ED) over 6 months, on average 245 days
    enhanced_encryption
    Safety Issue:
    No
  • Assessment of incremental recovery in main study
    date_rangeTime 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
    date_rangeTime 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)
    enhanced_encryption
    Safety Issue:
    Yes

Trial design

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
Trial Type
Interventional
Intervention Type
Biological/Vaccine
Trial Purpose
Treatment
Allocation
Non-randomized
Blinding
Open Label
Assignment
Single Group Assignment
Trial Arms
3

Additional Information

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