Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients

Hemophilia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01817868
• Other study ID #: 16368
• Secondary ID: KG1210TR
• Status: Completed
• Start date: January 4, 2013
• Est. completion date: May 23, 2019

Study information

• Verified date: May 2020
• Source: Bayer
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 73
• Est. completion date: May 23, 2019
• Est. primary completion date: July 16, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia
• Hemophilia A

Intervention

Other:
• Recombinant Factor VIII (Kogenate, BAY14-2222)
All dosage, frequency and duration for drugs will be under the decision of the treating physician.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Bayer

Country where clinical trial is conducted

Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group		After 4 years
Primary	Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group		After 4 years
Secondary	Number of overall bleeding episodes		After 4 years
Secondary	Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score)		After 4 years
Secondary	Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score)		After 4 years
Secondary	Cost-effectiveness (cost of additional joint bleed)		After 4 years
Secondary	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].	After 4 years
Secondary	Comparison of patient compliance between prophylaxis and on-demand therapy groups		After 4 years
Secondary	Number of spontaneous bleeds		After 4 years
Secondary	Quality of life as measured with the SF-36		Baseline and after 4 years
Secondary	Quality of life as measured with Hemo-QoL		Baseline and after 4 years

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