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NCT Number: NCT01775618

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

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

Up to 12 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

La Plata, Buenos Aires, Argentina

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

BAY94-9027

Biological

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

Primary outcomes

  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 outcomes

  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)

Sponsors and collaborators

Lead sponsor

Bayer

Industry

Registry information

Official study 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

Important dates

Study start
2013
Primary completion
2015
Study completion
2020
First posted
Jan 25, 2013
Registry last updated
Aug 21, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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