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Completed

NCT Number: NCT01029340

Trial to Evaluate the Efficacy and Safety of a New Full Length Recombinant Human FVIII for Hemophilia A

The study will assess the pharmacokinetics (part A) safety, tolerability, and efficacy of prophylaxis treatment (2 to 3 times a week) (part B) with BAY81-8973 over a one year period (split into two six month treatment periods). The study will compare 2 different methods (assays) for measuring the amount of study drug, the chromogenic substrate assay per European Pharmacopeia (CS/EP) with the classical assay (Chromogenic Substrate Adjusted, CS/ADJ). During one six month period patients will receive the study drug where the dose has been measured using the" (CS/EP) and during the other six months period the dose will be measured based on the Chromogenic Substrate Adjusted assay CS/ADJ)

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Key information

Conditions

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 Hemophilia A Hemorrhagic Disorders

Age range

12 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Bahía Blanca, Buenos Aires, Argentina

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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 defined as < 1% FVIII:C
  • >/= 150 days of previous treatment with FVIII in lifetime
  • Currently receiving on-demand or any type of prophylaxis treatment regimen with any FVIII product
  • No history of or current FVIII inhibitors

Exclusion criteria

  • Presence of another bleeding disease that is different from hemophilia A (e.g., von Willebrand disease, hemophilia B)
  • Low platelet count, abnormal kidney function, or liver disease
  • Received treatment with immune suppressing drugs within the last 3 months prior or requires treatment during the study. (Some drugs for hepatitis C, Human immunodeficiency virus (HIV), and steroids are allowed)
  • Receiving or has received other experimental drugs within 3 months prior to study entry
  • Allergy to Factor VIII or hamsters or mouse protein

Treatment and study plan

Recombinant Factor VIII (BAY81-8973)

Biological

Single dose of BAY81-8973 crossed over to single dose of Kogenate FS

Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Biological

Single dose of Kogenate FS crossed over to Single dose of BAY81-8973

Primary outcomes

  1. Part A - Area Under the Drug Concentration-time Curve (AUC)

    Time frame: Samples taken at pre-injection, and at 0.25, 0.5, 1, 3, 6, 8, 24, 30 and 48 hours post injection. AUC calculated from time of injection to infinity.

    To examine the Pharmacokinetic (PK) characteristics of BAY 81-8973 and ensure that the new drug is similar to Kogenate FS. All results are based on the chromogenic assay.

  2. Part A - Half-life (t 1/2)

    Time frame: Samples taken at pre-injection, and at 0.25, 0.5, 1, 3, 6, 8, 24, 30 and 48 hours post injection.

    To examine the PK characteristics of BAY81-8973 and ensure that the new drug is similar to Kogenate FS. All results are based on the chromogenic assay.

  3. Part B - Annualized Number of Total Bleeds

    Time frame: 12 months after randomization

    The annualized number of bleeds experienced by participants

Secondary outcomes

  1. Part B - The in Vivo Recovery Values of Human Factor VIII (FVIII)

    Time frame: 15-30 minutes after the injection

    The amount of Factor VIII found in blood samples taken after the injection of the study drug at the beginning of the CS/EP treatment period.

  2. Part B - Annualized Number of Bleeds in Each 6-month Potency Assignment Period

    Time frame: 6 months on each potency

    The annualized number of bleeds experienced by participants in each of the two treatment periods

  3. Part B - Control of Bleeding as Measured by the Number of Injections Required to Treat a Bleed

    Time frame: 6 months on each potency

    The number of injections needed by participants to stop a bleed

  4. Part B - Changes From Baseline at 12 Months in Quality of Life (QoL) as Measured by Transformed Total Score of Haemo-QoL Questionnaire

    Time frame: Baseline and 12 months

    A measure of how treatment with BAY81-8973 affected the daily life of participants. the scoring system has 100 points. 0 is the worst possible score. 100 is the best possible score. Positive changes from baseline indicate an improvement in quality of life and negative changes indicate a deterioration.

  5. Part B - Changes From Baseline at 12 Months in Utility Index as Measured by EQ-5D Questionaire

    Time frame: Baseline and 12 months

    A measure of how treatment with BAY81-8973 affected the daily life of participants. 1.0 = Best possible score, -0.594 = Worst possible score. Positive changes from baseline indicate an improvement and negative changes indicate a deterioration.

  6. Part A - Number of Participants With Inhibitory Antibody Formation

    Time frame: Up to 6 weeks after first injection of study drug

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

  7. Part B - Number of Participants With Incidence of Inhibitory Antibody Formation

    Time frame: Up to 12 months after drug administration

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

  8. Part C - Number of Participants With Incidence of Inhibitory Antibody Formation

    Time frame: before and 3 weeks after surgery

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

  9. Part A - Number of Participants With Incidence of Antibody Formation to Heat-shock Protein (HSP-70)

    Time frame: Up to 6 weeks after drug administration

    A test to analyze the formation of antibodies to HSP-70

  10. Part B - Number of Participants With Incidence of Antibody Formation to Heat-shock Protein (HSP-70)

    Time frame: Up to 12 months after drug administration

    A test to analyze the formation of antibodies to HSP-70

  11. Part C - Number of Participants With Incidence of Antibody Formation to Heat-shock Protein (HSP-70)

    Time frame: before and 3 weeks after surgery

    A test to analyze the formation of antibodies to HSP-70

  12. Part A - Number of Participants With Incidence of Antibody Formation to Host Cell Proteins (HCP)

    Time frame: Up to 4 weeks after drug administration

    A test to ensure that participants have not developed antibodies to HCP during the study

  13. Part B - Number of Participants With Incidence of Antibody Formation to Host Cell Proteins (HCP)

    Time frame: Up to 12 months after drug administration

    A test to ensure that participants have not developed antibodies to HCP during the study

  14. Part C - Number of Participants With Incidence of Antibody Formation to Host Cell Proteins (HCP)

    Time frame: before and 3 weeks after surgery

    A test to ensure that participants have not developed antibodies to HCP during the study

  15. Part B - Number of Participants With Assessment of the Hemostasis During Major Surgery

    Time frame: An average of 1 month after start of treatment

    An assessment made by surgeons of how effective BAY81-8973 was in stopping bleeding during major operations

  16. Part C - Number of Participants With Assessment of the Hemostasis During Major Surgery

    Time frame: at the time of surgery

    An assessment made by surgeons of how effective BAY81-8973 was in stopping bleeding during major operations

Sponsors and collaborators

Lead sponsor

Bayer

Industry

Registry information

Official study title

A Two Part Randomized Cross-Over Trial to Evaluate the Pharmacokinetics, Efficacy, and Safety Profile of Plasma Protein-Free Recombinant FVIII Formulated With Sucrose (BAY81-8973) in Previously Treated Subjects With Severe Hemophilia A Under Prophylaxis Therapy

Acronym: Leopold I

Important dates

Study start
2009
Primary completion
2012
Study completion
2013
First posted
Dec 9, 2009
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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