Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients
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|ClinicalTrials.gov Identifier: NCT01817868|
Recruitment Status : Active, not recruiting
First Posted : March 26, 2013
Last Update Posted : May 4, 2018
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.
|Condition or disease||Intervention/treatment|
|Hemophilia||Other: Recombinant Factor VIII (Kogenate, BAY14-2222)|
|Study Type :||Observational|
|Actual Enrollment :||73 participants|
|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|
|Actual Study Start Date :||January 4, 2013|
|Estimated Primary Completion Date :||June 28, 2018|
|Estimated Study Completion Date :||September 28, 2018|
Other: Recombinant Factor VIII (Kogenate, BAY14-2222)
All dosage, frequency and duration for drugs will be under the decision of the treating physician.
- Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group [ Time Frame: After 4 years ]
- Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group [ Time Frame: After 4 years ]
- Number of overall bleeding episodes [ Time Frame: After 4 years ]
- Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score) [ Time Frame: After 4 years ]
- Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score) [ Time Frame: After 4 years ]
- Cost-effectiveness (cost of additional joint bleed) [ Time Frame: After 4 years ]
- Cost-utility [ Time Frame: After 4 years ]The costs of care of subjects with haemophilia will be evaluated adopting the perspective of the payer, i.e. the Social Security Institution [SSI].
- Comparison of patient compliance between prophylaxis and on-demand therapy groups [ Time Frame: After 4 years ]
- Number of spontaneous bleeds [ Time Frame: After 4 years ]
- Quality of life as measured with the SF-36 [ Time Frame: Baseline and after 4 years ]
- Quality of life as measured with Hemo-QoL [ Time Frame: Baseline and after 4 years ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01817868
|Multiple Locations, Turkey|
|Study Director:||Bayer Study Director||Bayer|