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

NCT ID: NCT01029340

Last Updated: 2016-11-28

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

74 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-12-31

Study Completion Date

2013-03-31

Brief Summary

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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)

Detailed Description

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Conditions

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Blood Coagulation Disorders Hemophilia A

Keywords

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Hemophilia A Factor VIII Prophylaxis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Arm 1: Recombinant Factor VIII (BAY81-8973) then Kogenate FS

Part A - Arm 1: Participants first received one single intravenous (IV) injection of BAY81-8973 50 IU/kg, then 1 single IV injection of Kogenate FS (BAY14-2222) 50 IU/kg with a wash-out period of at least 2-3 days in between

Group Type EXPERIMENTAL

Recombinant Factor VIII (BAY81-8973)

Intervention Type BIOLOGICAL

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

Arm 2: Kogenate FS then Recombinant Factor VIII (BAY81-8973)

Part A - Arm 2: Participants first received one single intravenous (IV) injection of Kogenate FS (BAY14-2222) 50 IU/kg, then 1 single IV injection of BAY81-8973 50 IU/kg with a wash-out period of at least 2-3 days in between

Group Type EXPERIMENTAL

Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Intervention Type BIOLOGICAL

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

Arm 3: Recombinant Factor VIII by CS/EP then by CS/ADJ

Part B - Arm 3: Participants received IV injection of BAY81-8973 at 20-50 IU/kg 2-3 times per week with BAY81-8973 measured by Chromogenic Substrate Assay Potency Per European Pharmacopeia for 6 months and then crossed over to study drug measured by Chromogenic Substrate Assay/Adjusted to Label Potency for 6 months

Group Type EXPERIMENTAL

Recombinant Factor VIII (BAY81-8973)

Intervention Type BIOLOGICAL

Participants received IV injections of BAY81-8973 at 20-50 IU/kg 2-3 times per week with BAY81-8973 measured by Chromogenic Substrate Assay Potency Per European Pharmacopeia (CS/EP) for 6 months and by Chromogenic Substrate Assay/Adjusted to Label Potency (CS/ADJ) for 6 months, sequence according to randomization.

Arm 4: Recombinant Factor VIII by CS/ADJ then by CS/EP

Part B - Arm 4:. Participants received IV injection of BAY81-8973 at 20-50 IU/kg 2-3 times per week with BAY81-8973 measured by Chromogenic Substrate Assay/Adjusted to Label Potency for 6 months and then crossed over to study drug measured by Chromogenic Substrate Assay Per European Pharmacopeia for 6 months

Group Type EXPERIMENTAL

Recombinant Factor VIII (BAY81-8973)

Intervention Type BIOLOGICAL

Participants received IV injections of BAY81-8973 at 20-50 IU/kg 2-3 times per week with BAY81-8973 measured by Chromogenic Substrate Assay Potency Per European Pharmacopeia (CS/EP) for 6 months and by Chromogenic Substrate Assay/Adjusted to Label Potency (CS/ADJ) for 6 months, sequence according to randomization.

Arm 5: Recombinant Factor VIII by CS/EP

Part C - Arm 5: Participants received a loading dose of approximately 50 IU/kg of BAY 81-8973 before the first surgical incision followed by further treatment with BAY 81-8973 according to surgical requirements for up to 3 weeks

Group Type EXPERIMENTAL

Recombinant Factor VIII (BAY81-8973)

Intervention Type BIOLOGICAL

Participants received a loading dose of approximately 50 IU/kg of BAY 81-8973 before the first surgical incision followed by further treatment with BAY 81-8973 according to surgical requirements for up to 3 weeks

Interventions

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Recombinant Factor VIII (BAY81-8973)

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

Intervention Type BIOLOGICAL

Recombinant Factor VIII (Kogenate FS, BAY14-2222)

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

Intervention Type BIOLOGICAL

Recombinant Factor VIII (BAY81-8973)

Participants received IV injections of BAY81-8973 at 20-50 IU/kg 2-3 times per week with BAY81-8973 measured by Chromogenic Substrate Assay Potency Per European Pharmacopeia (CS/EP) for 6 months and by Chromogenic Substrate Assay/Adjusted to Label Potency (CS/ADJ) for 6 months, sequence according to randomization.

Intervention Type BIOLOGICAL

Recombinant Factor VIII (BAY81-8973)

Participants received a loading dose of approximately 50 IU/kg of BAY 81-8973 before the first surgical incision followed by further treatment with BAY 81-8973 according to surgical requirements for up to 3 weeks

Intervention Type BIOLOGICAL

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Maximum Eligible Age

65 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Bayer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Bayer Study Director

Role: STUDY_DIRECTOR

Bayer

Locations

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Orange, California, United States

Site Status

Sacramento, California, United States

Site Status

Tampa, Florida, United States

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Boston, Massachusetts, United States

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East Lansing, Michigan, United States

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Kansas City, Missouri, United States

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Cleveland, Ohio, United States

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Bahía Blanca, Buenos Aires, Argentina

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Rosario, Santa Fe Province, Argentina

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Vienna, Vienna, Austria

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Graz, , Austria

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Zagreb, , Croatia

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DK-Aarhus N, , Denmark

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Frankfurt am Main, Hesse, Germany

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Bonn, North Rhine-Westphalia, Germany

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Mainz, Rhineland-Palatinate, Germany

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Homburg, Saarland, Germany

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Magdeburg, Saxony-Anhalt, Germany

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Hong Kong, , Hong Kong

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Bangalore, , India

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Mumbai, , India

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Pune, , India

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Jakarta, , Indonesia

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Ramat Gan, , Israel

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Catanzaro, Calabria, Italy

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Napoli, Campania, Italy

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Napoli, Campania, Italy

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Rome, Lazio, Italy

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Milan, Lombardy, Italy

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Vicenza, Veneto, Italy

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Oslo, , Norway

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Karachi, , Pakistan

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Krakow, , Poland

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Warsaw, , Poland

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Belgrade, , Serbia

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Niš, , Serbia

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Novi Sad, , Serbia

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Pretoria, Gauteng, South Africa

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Parktown, , South Africa

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A Coruña, A Coruña, Spain

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Barcelona, Barcelona, Spain

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Santander, Cantabria, Spain

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Jaén, Jaén, Spain

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Oviedo, Principality of Asturias, Spain

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Valencia, Valencia, Spain

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Gothenburg, , Sweden

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Malmo, , Sweden

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Karolinska Universitetssjukhuset i Solna

Stockholm, , Sweden

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Taipei, Taipei, Taiwan

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Changhua, , Taiwan

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Taipei, , Taiwan

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Bangkok, , Thailand

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Bangkok, , Thailand

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Adana, , Turkey (Türkiye)

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Antalya, , Turkey (Türkiye)

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Izmir, , Turkey (Türkiye)

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Dundee, Dundee City, United Kingdom

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Oxford, Oxfordshire, United Kingdom

Site Status

Sheffield, South Yorkshire, United Kingdom

Site Status

London, , United Kingdom

Site Status

Countries

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Finland United States Argentina Austria Croatia Denmark Germany Hong Kong India Indonesia Israel Italy Norway Pakistan Poland Serbia South Africa Spain Sweden Taiwan Thailand Turkey (Türkiye) United Kingdom

References

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Kitchen S, Katterle Y, Beckmann H, Maas Enriquez M. Chromogenic assay for BAY 81-8973 potency assignment has no impact on clinical outcome or monitoring in patient samples. J Thromb Haemost. 2016 Jun;14(6):1192-9. doi: 10.1111/jth.13322. Epub 2016 May 3.

Reference Type RESULT
PMID: 27002680 (View on PubMed)

Oldenburg J, Windyga J, Hampton K, Lalezari S, Tseneklidou-Stoeter D, Beckmann H, Maas Enriquez M. Safety and efficacy of BAY 81-8973 for surgery in previously treated patients with haemophilia A: results of the LEOPOLD clinical trial programme. Haemophilia. 2016 May;22(3):349-53. doi: 10.1111/hae.12839. Epub 2016 Mar 1.

Reference Type RESULT
PMID: 26931631 (View on PubMed)

Saxena K, Lalezari S, Oldenburg J, Tseneklidou-Stoeter D, Beckmann H, Yoon M, Maas Enriquez M. Efficacy and safety of BAY 81-8973, a full-length recombinant factor VIII: results from the LEOPOLD I trial. Haemophilia. 2016 Sep;22(5):706-12. doi: 10.1111/hae.12952. Epub 2016 Jun 24.

Reference Type DERIVED
PMID: 27339736 (View on PubMed)

Related Links

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http://www.clinicaltrialsregister.eu/

Click here to find information about studies related to Bayer Healthcare products conducted in Europe

Other Identifiers

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2009-012149-43

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

12954

Identifier Type: -

Identifier Source: org_study_id