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

Hemophilia A Clinical Trial

— Leopold II  

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 Plasma Protein-free Recombinant FVIII Formulated With Sucrose (BAY 81-8973)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01233258
• Other study ID #: 14319
• Secondary ID: 2009-012150-20
• Status: Completed
• Phase: Phase 3
• First received: November 2, 2010
• Last updated: August 12, 2015
• Start date: January 2011
• Est. completion date: December 2012

Study information

• Verified date: August 2015
• Source: Bayer
• Contact: n/a
• Is FDA regulated: No
• Health authority: Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia MedicaChina: Food and Drug AdministrationColombia: INVIMA Instituto Nacional de Vigilancia de Medicamentos y AlimentosCzech Republic: State Institute for Drug ControlJapan: Pharmaceuticals and Medical Devices AgencyMexico: Federal Commission for Protection Against Health RisksRomania: National Medicines AgencyRussia: Ministry of Health of the Russian FederationSerbia and Montenegro: Agency for Drugs and Medicinal DevicesSlovakia: State Institute for Drug ControlSouth Africa: Medicines Control CouncilTaiwan : Food and Drug AdministrationTurkey: Ministry of HealthUkraine: State Pharmacological Center - Ministry of HealthUnited States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 80
• Est. completion date: December 2012
• Est. primary completion date: December 2012
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years to 65 Years

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• rFVIII (BAY81-8973) on demand
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
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
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.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Bayer

Countries where clinical trial is conducted

United States,  Argentina,  China,  Colombia,  Czech Republic,  India,  Indonesia,  Japan,  Mexico,  Romania,  Russian Federation,  Serbia,  Slovakia,  South Africa,  Taiwan,  Thailand,  Turkey,  Ukraine, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Number of Bleeds During Treatment	The number of bleeds experienced by each participant	12 months	No
Other	Number of Participants With Inhibitory Antibody Formation	A test to ensure that participants have not developed antibodies that will interfere with the action of rFVIII (BAY81-8973)	3, 6, 9 and 12 months after baseline	Yes
Primary	Annualized Number of All Bleeds	The annualized number of bleeds experienced by participants	Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)	No
Secondary	Annualized Number of All Bleeds During CS/EP Period	The annualized number of bleeds experienced by participants while they were taking rFVIII (BAY81-8973) assayed by CS/EP	Up to 6 months (6 months on CS/EP potency assignment)	No
Secondary	Annualized Number of All Bleeds During CS/ADJ Period	The annualized number of bleeds experienced by participants while they were taking rFVIII (BAY81-8973) assayed by CS/ADJ	Up to 6 months (6 months on CS/ADJ potency assignment)	No
Secondary	Percentage of Bleeds Per Participant Controlled With = 2 Injections in Participants Treated on Demand With rFVIII (BAY81-8973)	The percentage of bleeds per participant on on-demand treatment that stopped after two or fewer injections	Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design)	No

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