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

This study has been completed.
Sponsor:
Information provided by:
Bayer
ClinicalTrials.gov Identifier:
NCT01233258
First received: November 2, 2010
Last updated: January 25, 2013
Last verified: January 2013
  Purpose

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.


Condition Intervention Phase
Hemophilia A
Biological: Recombinant Factor VIII (BAY81-8973)
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Official 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 BAY81-8973 (rFVIII Formulated With Sucrose)

Resource links provided by NLM:


Further study details as provided by Bayer:

Primary Outcome Measures:
  • Annualized number of all bleeds [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Annualized number of all bleeds with 2 potency assignments [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Response to treatment of bleeds as measured by proportion of bleeds treated with 1 or 2 infusions [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Enrollment: 80
Study Start Date: January 2011
Study Completion Date: December 2012
Primary Completion Date: December 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Arm 1 Biological: Recombinant Factor VIII (BAY81-8973)
On-demand treatment with BAY81-8973 assayed by CS/EP (Chromogenic substrate/European Pharmacopoeia assay) for 6 months, followed by cross-over to BAY81-8973 assayed by CS/ADJ (Chromogenic substrate/one-stage adjusted assay) for 6 months.
Experimental: Arm 2 Biological: Recombinant Factor VIII (BAY81-8973)
Prophylaxis with high-dose BAY81-8973 assayed by CS/EP for 6 months, followed by cross-over to BAY81-8973 assayed by CS/ADJ for 6 months.
Experimental: Arm 3 Biological: Recombinant Factor VIII (BAY81-8973)
Prophylaxis with low-dose BAY81-8973 assayed by CS/EP for 6 months, followed by cross-over to BAY81-8973 assayed by CS/ADJ for 6 months.
Experimental: Arm 4 Biological: Recombinant Factor VIII (BAY81-8973)
Prophylaxis with high-dose BAY81-8973 assayed by CS/ADJ for 6 months, followed by cross-over to BAY81-8973 assayed by CS/EP for 6 months.
Experimental: Arm 5 Biological: Recombinant Factor VIII (BAY81-8973)
Prophylaxis with low-dose BAY81-8973 assayed by CS/ADJ for 6 months, followed by cross-over to BAY81-8973 assayed by CS/EP for 6 months.
Experimental: Arm 6 Biological: Recombinant Factor VIII (BAY81-8973)
On-demand treatment with BAY81-8973 assayed by CS/ADJ for 6 months, followed by cross-over to BAY81-8973 assayed by CS/EP for 6 months .

  Eligibility

Ages Eligible for Study:   12 Years to 65 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01233258

  Hide Study Locations
Locations
United States, Ohio
Dayton, Ohio, United States, 45404
United States, Texas
Houston, Texas, United States, 77030
Argentina
Buenos Aires, Ciudad Auton. de Buenos Aires, Argentina, C1221ADC
Rosario, Santa Fe, Argentina, S2000CKF
Corrientes, Argentina, W3410AVV
China, Guangdong
Guangzhou, Guangdong, China, 510515
China, Jiangsu
Suzhou, Jiangsu, China, 215006
China
Beijing, China, 100730
Shanghai, China, 200025
Tianjin, China
Colombia
Bogotá, Distrito Capital de Bogotá, Colombia
Bucaramanga, Santander, Colombia
Czech Republic
Olomouc, Czech Republic, 77520
India
Hyderabad, India, 500034
Indonesia
Jakarta, Indonesia, 10430
Japan
Nishinomiya, Hyogo, Japan, 663-8501
Kashihara, Nara, Japan, 634-8522
Shinjuku-ku, Tokyo, Japan, 160-0023
Suginami, Tokyo, Japan, 167-0035
Hiroshima, Japan, 734-8551
Mexico
Guadalajara, Jalisco, Mexico, 44280
San Luis Potosí, Mexico, 78200
Romania
Timisoara, Timis, Romania, 300011
Baia Mare, Romania, 430031
Bucharest, Romania, 11026
Bucharest, Romania, 022328
Russian Federation
Barnaul, Russian Federation, 656050
Ekaterinburg, Russian Federation, 620149
Khabarovsk, Russian Federation, 680009
St. Petersburg, Russian Federation, 191186
Serbia
Beograd, Serbia, 11000
Kragujevac, Serbia, 34000
Nis, Serbia, 18000
Novi Sad, Serbia, 21000
Slovakia
Bratislava, Slovakia, 851 07
South Africa
Johannesburg, Gauteng, South Africa, 2132
Pretoria, Gauteng, South Africa, 0001
Taiwan
Taichung, Taiwan, 40447
Taipei, Taiwan, 11217
Thailand
Bangkok, Thailand, 10700
Bangkok, Thailand, 10400
Turkey
Adana, Turkey, 01330
Antalya, Turkey, 07059
Izmir, Turkey, 35-100
Ukraine
Kiev, Ukraine
Lviv, Ukraine, 79044
Simferopol, Ukraine, 95023
Sponsors and Collaborators
Bayer
Investigators
Study Director: Bayer Study Director Bayer
  More Information

Additional Information:
No publications provided

Responsible Party: Therapeutic Area Head, Bayer Healthcare AG
ClinicalTrials.gov Identifier: NCT01233258     History of Changes
Other Study ID Numbers: 14319, 2009-012150-20
Study First Received: November 2, 2010
Last Updated: January 25, 2013
Health Authority: Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
China: Food and Drug Administration
Colombia: INVIMA Instituto Nacional de Vigilancia de Medicamentos y Alimentos
Czech Republic: State Institute for Drug Control
Japan: Pharmaceuticals and Medical Devices Agency
Mexico: Federal Commission for Protection Against Health Risks
Romania: National Medicines Agency
Russia: Ministry of Health of the Russian Federation
Serbia and Montenegro: Agency for Drugs and Medicinal Devices
Slovakia: State Institute for Drug Control
South Africa: Medicines Control Council
Taiwan : Food and Drug Administration
Turkey: Ministry of Health
Ukraine: State Pharmacological Center - Ministry of Health
United States: Food and Drug Administration

Keywords provided by Bayer:
Haemophilia treatment
rFVIII

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 16, 2013