Brief Title
Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients
Official Title
Evaluation of Efficacy, Safety and Costs of Recombinant FVIII Products Applied to Severe Hemophilia A Patients: Observational Data Collection Study Evaluating On-demand Treatment and Secondary Prophylaxis
Brief Summary
The project is an observational, multi-central, prospective, non-interventional and open-label data collection study on secondary prophylaxis with recombinant FVIII products in adolescents and adults with severe hemophilia A (FVIII < 1%). It will be a controlled observation of patients on secondary prophylaxis versus on-demand treatment regimen. Patients will be enrolled preferably on a 1:1 basis with regards to prophylaxis and on-demand treatment. The patient enrollment period will be 2 years with a follow-up (observation period) of 2 years for each patient. Based on the primary effectiveness parameters (joint bleeds and overall bleeds per year) an observation period of 2 years is considered sufficient although it has to be admitted that it is rather short to assess the progression of orthopedic status. Previously treated prophylaxis patients with at least 50 exposure days and patients with continuing prophylaxis treatment will be included.
Study Type
Observational
Primary Outcome
Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group
Secondary Outcome
Number of overall bleeding episodes
Condition
Hemophilia
Intervention
Recombinant Factor VIII (Kogenate, BAY14-2222)
Study Arms / Comparison Groups
Group 1
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Other
Estimated Enrollment
73
Start Date
January 4, 2013
Completion Date
May 23, 2019
Primary Completion Date
July 16, 2018
Eligibility Criteria
Inclusion Criteria: - Age ≥ 18 years - Severe hemophilia A (FVIII<1%) diagnosis - Prior treatment or ongoing treatment with on-demand or prophylaxis regimens according to Turkish guidelines and requirements - Previously treated patients with at least >50 exposure days - Written informed consent signed by patient/legal representative Exclusion Criteria: - Currently on immune tolerance treatment - Platelet count < 75,000/mm3 - Participation in another study - Existence of inhibitors in the past and in the last currently available blood sample prior to study start (Bethesda titer < 0.6 BU/ml) - Existence of inhibitor history in family members who also are diagnosed with hemophilia A - Having been on primary prophylaxis as defined in the introduction - Participation in another study
Gender
All
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Bayer Study Director, ,
Location Countries
Turkey
Location Countries
Turkey
Administrative Informations
NCT ID
NCT01817868
Organization ID
16368
Secondary IDs
KG1210TR
Responsible Party
Sponsor
Study Sponsor
Bayer
Study Sponsor
Bayer Study Director, Study Director, Bayer
Verification Date
May 2020