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Completed

NCT Number: NCT01233258

A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A

The objective of the trial is to demonstrate that 2-3 times per week prophylaxis therapy with BAY81-8973 is superior to on-demand therapy with BAY81-8973 in patients with severe Hemophilia A. The hypothesis is that prophylaxis will result in fewer bleeds than on-demand treatment.

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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

12 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Buenos Aires, Ciudad Auton. de 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

  • Male, aged 12 to 65 years
  • Severe hemophilia A
  • History of more than 150 exposure days (ED) with clotting factor concentrates
  • Currently receiving episodic treatment with FVIII; no regular prophylaxis for more than 6 consecutive months in the past 5 years
  • No current Factor VIII inhibitor or history of inhibitor
  • Willing to use electronic patient diary

Exclusion criteria

  • Presence of another bleeding disease that is different from hemophilia A
  • Thrombocytopenia
  • Abnormal renal function
  • Presence of active liver disease
  • Known hypersensitivity to FVIII

Treatment and study plan

rFVIII (BAY81-8973) on demand

Biological

Participants received on-demand treatment with rFVIII (BAY81-8973) assayed by CS/EP (Chromogenic Substrate Assay per European Pharmacopoeia) for 6 months and by CS/ADJ (Chromogenic Substrate Assay/label adjusted to one-stage assay) for 6 months, sequence according to randomization

rFVIII (BAY81-8973) prophylaxis low-dose

Biological

Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.

rFVIII (BAY81-8973) prophylaxis high-dose

Biological

Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.

Primary outcomes

  1. Annualized Number of All Bleeds

    Time frame: Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)

    The annualized number of bleeds experienced by participants

Secondary outcomes

  1. Annualized Number of All Bleeds During CS/EP Period

    Time frame: Up to 6 months (6 months on CS/EP potency assignment)

    The annualized number of bleeds experienced by participants while they were taking rFVIII (BAY81-8973) assayed by CS/EP

  2. Annualized Number of All Bleeds During CS/ADJ Period

    Time frame: Up to 6 months (6 months on CS/ADJ potency assignment)

    The annualized number of bleeds experienced by participants while they were taking rFVIII (BAY81-8973) assayed by CS/ADJ

  3. Percentage of Bleeds Per Participant Controlled With ≤ 2 Injections in Participants Treated on Demand With rFVIII (BAY81-8973)

    Time frame: Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)

    The percentage of bleeds per participant on on-demand treatment that stopped after two or fewer injections

Other outcomes

  1. Number of Bleeds During Treatment

    Time frame: 12 months

    The number of bleeds experienced by each participant

  2. Number of Participants With Inhibitory Antibody Formation

    Time frame: 3, 6, 9 and 12 months after baseline

    A test to ensure that participants have not developed antibodies that will interfere with the action of rFVIII (BAY81-8973)

Sponsors and collaborators

Lead sponsor

Bayer

Industry

Registry information

Official study title

A Phase II/III, Randomized, Cross-over, Open-label Trial to Demonstrate Superiority of Prophylaxis Over On-demand Therapy in Previously Treated Subjects With Severe Hemophilia A Treated With Plasma Protein-free Recombinant FVIII Formulated With Sucrose (BAY 81-8973)

Acronym: Leopold II

Important dates

Study start
2011
Primary completion
2012
Study completion
2012
First posted
Nov 3, 2010
Registry last updated
Nov 28, 2016

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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