check_circleStudy Completed

Hemophilia

Bayer Identifier:

16368

ClinicalTrials.gov Identifier:

NCT01817868

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.

Key Participants Requirements

Sex

Both

Age

18 - N/A
  • - 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


  • - 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

Trial summary

Enrollment Goal
73
Trial Dates
January 2013 - May 2019
Phase
N/A
Could I Receive a placebo
No
Products
Kogenate FS (Recombinant Factor VIII, BAY14-2222)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Completed
Many LocationsMany Locations, Turkey

Primary Outcome

  • Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group
    date_rangeTime Frame:
    After 4 years
    enhanced_encryption
    Safety Issue:
    No
  • Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group
    date_rangeTime Frame:
    After 4 years
    enhanced_encryption
    Safety Issue:
    No

Secondary Outcome

  • Number of overall bleeding episodes
    date_rangeTime Frame:
    After 4 years
    enhanced_encryption
    Safety Issue:
    No
  • Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score)
    date_rangeTime Frame:
    After 4 years
    enhanced_encryption
    Safety Issue:
    No
  • Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score)
    date_rangeTime Frame:
    After 4 years
    enhanced_encryption
    Safety Issue:
    No
  • Cost-effectiveness (cost of additional joint bleed)
    date_rangeTime Frame:
    After 4 years
    enhanced_encryption
    Safety Issue:
    No
  • Cost-utility
    The 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 years
    enhanced_encryption
    Safety Issue:
    No
  • Comparison of patient compliance between prophylaxis and on-demand therapy groups
    date_rangeTime Frame:
    After 4 years
    enhanced_encryption
    Safety Issue:
    No
  • Number of spontaneous bleeds
    date_rangeTime Frame:
    After 4 years
    enhanced_encryption
    Safety Issue:
    No
  • Quality of life as measured with the SF-36
    date_rangeTime Frame:
    Baseline and after 4 years
    enhanced_encryption
    Safety Issue:
    No
  • Quality of life as measured with Hemo-QoL
    date_rangeTime Frame:
    Baseline and after 4 years
    enhanced_encryption
    Safety Issue:
    No

Trial design

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
Trial Type
Observational
Intervention Type
Other
Trial Purpose
N/A
Allocation
N/A
Blinding
N/A
Assignment
N/A
Trial Arms
N/A

Additional Information

Click here to find results for studies related to Bayer products.