Overview

This trial is active, not recruiting.

Condition hemophilia
Treatment recombinant factor viii (kogenate, bay14-2222)
Sponsor Bayer
Start date January 2013
End date June 2018
Trial size 73 participants
Trial identifier NCT01817868, 16368, KG1210TR

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model cohort
Time perspective prospective
Arm
recombinant factor viii (kogenate, bay14-2222)
All dosage, frequency and duration for drugs will be under the decision of the treating physician.

Primary Outcomes

Measure
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

Secondary Outcomes

Measure
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
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

Eligibility Criteria

Male or female participants at least 18 years old.

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

Additional Information

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
Trial information was received from ClinicalTrials.gov and was last updated in September 2016.
Information provided to ClinicalTrials.gov by Bayer.