BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A

Hemophilia A Clinical Trial

Official title:

Randomized, Active-controlled, Double-blind, Parallel Design Study to Evaluate the Efficacy and Safety of a Once-a-week Prophylaxis Treatment With BAY79-4980 Compared to Three Times-per-week Prophylaxis With rFVIII-FS in Previously Treated Patients With Severe Hemophilia A

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00623727
• Other study ID #: 12781
• Secondary ID: 2007-003718-32
• Status: Terminated
• Phase: Phase 2
• First received: January 9, 2008
• Last updated: July 8, 2013
• Start date: June 2008
• Est. completion date: October 2010

Study information

• Verified date: July 2013
• Source: Bayer
• Contact: n/a
• Is FDA regulated: No
• Health authority: Austria: Agency for Health and Food SafetyBelgium: Federal Agency for Medicinal Products and Health ProductsCanada: Health CanadaDenmark: Danish Medicines AgencyFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)Germany: Paul-Ehrlich-InstitutIsrael: Ministry of HealthItaly: The Italian Medicines AgencyNetherlands: The Central Committee on Research Involving Human Subjects (CCMO)Norway: Norwegian Medicines AgencyPoland: Office for Registration of Medicinal Products, Medical Devices and Biocidal ProductsSpain: Spanish Agency of MedicinesUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyCroatia: Ministry of Health and Social CareTurkey: Ministry of HealthUnited States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

A study to assess treatment with a new formulation of recombinant factor VIII reconstituted with liposomes (BAY79-4980) to evaluate whether a once-a-week treatment is safe and can prevent bleeds in subjects with severe haemophilia A.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 143
• Est. completion date: October 2010
• Est. primary completion date: April 2010
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years to 70 Years

Eligibility

Inclusion Criteria:

• Males aged 12 to 70 years

• Subjects with severe hemophilia A (< 1% factor VIII [FVIII]:C)

• Subjects with equal or greater than 150 exposure days (EDs) with any FVIII in total

• Subjects who have been on-demand treatment with a minimum of 1 relevant bleed per month or have been on secondary prophylaxis treatment with not more than a 3x/week schedule

• Subjects with bleeding events and/or treatments during the last 6 months prior to study entry which are documented in the subjects medical records

• Subjects with no measurable inhibitor activity

• Subjects with no history of FVIII inhibitor antibody formation

• Written informed consent by subject and parent / legal representative, if < 18 years

Exclusion Criteria:

• Subjects who are receiving primary prophylaxis

• Subjects on prophylaxis with documented requirements of > 75 IU/kg/week

• Subjects with any other bleeding disease beside hemophilia A (i.e., von Willebrand disease)

• Subjects with abnormal renal function

• Subjects with elevated hepatic transaminases

• Subjects on treatment with immunomodulatory agents within the last 3 months prior to study entry or during the study

• Subjects with known hypersensitivity to the active substance, mouse or hamster protein, liposomes or polyethyleneglycol (PEG)

• Subjects who require any pre-medication for FVIII injections

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• rFVIII-FS/pegylated liposomes (BAY79-4980)
35 IU/kg body weight intravenous 1x/week for 52 weeks This arm will be stopped by 30.04.10 the subjects will be offered to change to the active comparator arm
• rFVIII-FS/WFI (BAY14-2222)
25 IU/kg body weight intravenous 3x/week for 52 weeks

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Bayer

Countries where clinical trial is conducted

United States,  Argentina,  Australia,  Austria,  Belgium,  Canada,  Chile,  Croatia,  Denmark,  Estonia,  France,  Germany,  Israel,  Italy,  Lithuania,  Netherlands,  New Zealand,  Norway,  Poland,  Romania,  South Africa,  Spain,  Switzerland,  Taiwan,  Turkey,  United Kingdom, 

References & Publications (2)

Di Minno G, Cerbone AM, Coppola A, Cimino E, Di Capua M, Pamparana F, Tufano A, Di Minno MN. Longer-acting factor VIII to overcome limitations in haemophilia management: the PEGylated liposomes formulation issue. Haemophilia. 2010 Jan;16 Suppl 1:2-6. doi: — View Citation

Powell J, Martinowitz U, Windyga J, Di Minno G, Hellmann A, Pabinger I, Maas Enriquez M, Schwartz L, Ingerslev J; LipLong Study Investigators. Efficacy and safety of prophylaxis with once-weekly BAY 79-4980 compared with thrice-weekly rFVIII-FS in haemoph — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Number of Bleeds Per Year	Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. Number of bleeds 3 weeks after the first infusion per 12 months	up to one year	No
Other	Percentage of Bleeds Treated by Various Numbers of Injections	Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event.	up to one year	No
Other	Total rFVIII Consumption Per Year	Total number of units per kg of study medication (rFVIII) administered to participant for one year. rFVIII is recombinant factor VIII, factor VIII is functional coagulation factor	up to one year	No
Other	Percentage of Participants With Less Than 9 Total Bleeds Per Year in the Open Label Extension Period	Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event.	6 months after start of open label extension period	No
Primary	Percentage of Participants With Less Than 9 Total Bleeds Per Year	Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event.	up to one year	No
Secondary	Percentage of Participants With Less Than 5 Joint Bleeds Per Year	Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event.	up to one year	No
Secondary	Number of Joint Bleeds Per Participant Per Year in Responders	Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. Responders were the subjects with less than 9 total bleeds per year	up to one year	No

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