check_circleStudy Completed
Hemophilia
Bayer Identifier:
16368
ClinicalTrials.gov Identifier:
EudraCT Number:
Not Available
EU CT Number:
Not Available
Comparison of efficacy, safety and costs of recombinant FVIII products between on-demand and secondary prophylaxis groups in haemophilia A patients
Trial purpose
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.
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.
Key Participants Requirements
Sex
BothAge
18 - N/ATrial summary
Enrollment Goal
73Trial Dates
January 2013 - May 2019Phase
N/ACould I Receive a placebo
NoProducts
Kogenate FS (Recombinant Factor VIII, BAY14-2222)Accepts Healthy Volunteer
NoWhere to participate
Status | Institution | Location |
---|---|---|
Completed | Many Locations | Many Locations, Turkey |
Primary Outcome
- Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand groupdate_rangeTime Frame:After 4 yearsenhanced_encryptionNoSafety Issue:
- Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand groupdate_rangeTime Frame:After 4 yearsenhanced_encryptionNoSafety Issue:
Secondary Outcome
- Number of overall bleeding episodesdate_rangeTime Frame:After 4 yearsenhanced_encryptionNoSafety Issue:
- Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score)date_rangeTime Frame:After 4 yearsenhanced_encryptionNoSafety Issue:
- Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score)date_rangeTime Frame:After 4 yearsenhanced_encryptionNoSafety Issue:
- Cost-effectiveness (cost of additional joint bleed)date_rangeTime Frame:After 4 yearsenhanced_encryptionNoSafety Issue:
- Cost-utilityThe costs of care of subjects with haemophilia will be evaluated adopting the perspective of the payer, i.e. the Social Security Institution [SSI].date_rangeTime Frame:After 4 yearsenhanced_encryptionNoSafety Issue:
- Comparison of patient compliance between prophylaxis and on-demand therapy groupsdate_rangeTime Frame:After 4 yearsenhanced_encryptionNoSafety Issue:
- Number of spontaneous bleedsdate_rangeTime Frame:After 4 yearsenhanced_encryptionNoSafety Issue:
- Quality of life as measured with the SF-36date_rangeTime Frame:Baseline and after 4 yearsenhanced_encryptionNoSafety Issue:
- Quality of life as measured with Hemo-QoLdate_rangeTime Frame:Baseline and after 4 yearsenhanced_encryptionNoSafety Issue:
Trial design
Trial Type
ObservationalIntervention Type
OtherTrial Purpose
N/AAllocation
N/ABlinding
N/AAssignment
N/ATrial Arms
N/A