Overview

This trial is active, not recruiting.

Condition hemophilia a
Treatments survey
Sponsor Bayer
Start date January 2015
End date March 2017
Trial size 72 participants
Trial identifier NCT02319070, 17493, KG1411GR

Summary

This study is a prospective, single center, observational, 2-cohort study of adult patients with severe Hemophilia A. There is no randomization procedure and all patients will be treated as per usual clinical practice. Patients will be followed up for 18 months after enrolment.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model cohort
Time perspective prospective
Arm
Adult patients with severe Hemophilia A.(25 patients on Secondary Prophylaxis treatment)
survey
Recombinant antihemophilic factor VIII (FVIII) on Secondary Prophylaxis treatment scheduled according to daily routine
Adult patients with severe Hemophilia A.(50 patients on On Demand treatment)
survey
Recombinant antihemophilic factor VIII (FVIII) On Demand treatment scheduled according to daily routine

Primary Outcomes

Measure
Annual direct medical cost of specialized hemophilia centre for patients with the on-demand strategy
time frame: At 18 months
Annual direct medical cost of specialized hemophilia centre for patients with secondary prophylaxis
time frame: At 18 months

Secondary Outcomes

Measure
Mean cost per joint bleed episodes for overall patients
time frame: At 18 months
Mean cost per joint bleed episodes by cohort for patients
time frame: At 18 months
Number of joint bleed episodes for overall patients
time frame: At 18 months
Number of joint bleed episodes by cohort for patients.
time frame: At 18 months
Severity of joint bleed episodes for overall patients.
time frame: At 18 months
Severity of joint bleed episodes by cohort for patients.
time frame: At 18 months
Mean EQ-VAS score as reported from patients at the time of a major bleed.
time frame: At 18 months
Mean EQ-5D score as reported from patients at the time of a major bleed.
time frame: At 18 months
Mean change from baseline in Quality of Life scores derived from the Haem-A-Qol and EQ-5D questionnaires
time frame: Baseline and at 18 months

Eligibility Criteria

Male participants at least 18 years old.

Inclusion Criteria: - Patients diagnosed with severe Hemophilia A. - Age ≥18 years old - Patients able to read and write - Patients receiving Factor VIII substitute therapy, either on-demand or as secondary prophylaxis. - Patients under the same therapeutic strategy (On Demand or Secondary Prophylaxis) for at least 6 months prior to enrolment. Exclusion Criteria: - Patients that have developed inhibitors against factor VIII. - Patients participating in an investigational program with interventions outside of routine clinical practice.

Additional Information

Official title Prospective, Observational, 2-cohort Study of Adult Patients With Severe Hemophilia A in Greece. Cost,Clinical Outcomes and Quality of Life Comparison Between on Demand and Secondary Prophylaxis Treatment Strategies
Description The primary therapy for hemophilia is coagulation factor replacement, given either episodically on demand (OD) for the treatment of acute bleeds or prophylactically to prevent them. The last years there has been an interest to study the comparative effectiveness of OD vs. SP (Secondary Prophylaxis) treatment strategies so as to create an evidence based platform to guide both clinical decision making and quantify the economic aspects of each treatment alternative. In Greece relevant information is lacking, thus it is imperative to study how patients on different treatment strategies are managed in a real-life setting in Greece, including the main factors associated with increased health care utilization, clinical success and patient well being.
Trial information was received from ClinicalTrials.gov and was last updated in September 2016.
Information provided to ClinicalTrials.gov by Bayer.