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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


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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