Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A."

Completed

• Conditions: Hemophilia A
• Interventions: Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

• Registration Number: NCT02581969
• Lead Sponsor: Bayer

Brief Summary

Following the performance of the POTTER observational study, whose primary objective was to collect data on the benefits of secondary prophylaxis versus on demand treatment in terms of prevention of bleeding episodes, the present study aims to extend the observation time up to 10 years, of the same population involved in the previous study.

The present study has been designed to allow a focus on long term disease-related damage at joint level in subjects with severe haemophilia A, as well as on the pharmacoeconomics impact of the two different treatment regimens.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2015-10-20
• Study First Submitted QC: 2015-10-20
• Study First Posted: 2015-10-21
• Study Start: 2016-04-14
• Primary Completion: 2017-03-16
• Study Completion: 2018-06-21
• Status Verified: 2020-11
• Last Update Submitted: 2020-11-17
• Last Update Posted: 2020-11-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 43

Inclusion Criteria

• Completion of follow-up in the original Potter study without having being excluded from the Intention To Treat efficacy analysis of the primary endpoint;
• Written informed consent specifically issued for the 5-year extension.

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Exclusion Criteria

• Switching of treatment to a Factor VIII concentrate different from Kogenate Bayer/Helixate NexGen after the end of the previous follow-up period.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Prophylaxis	Recombinant Factor VIII (Kogenate FS, BAY14-2222)	Prophylaxis group: treatment is based on regularly repeated infusions of clotting factor, 20-30 IU/kg -3 times a week
On-demand	Recombinant Factor VIII (Kogenate FS, BAY14-2222)	On-demand group: treatment administered when bleeding episode occur

Primary Outcome Measures

Name	Time	Method
Number of joint bleeding episodes per year	5 years period

Secondary Outcome Measures

Name	Time	Method
Total amount of Recombinant Factor VIII consumption	5 years period
Total number of bleeding episodes per year	5 years period
Changes of prophylaxis dose	5 years period
Changes of frequency of infusions	5 years period
Musculoskeletal evaluation by calculating the World Federation of Haemophilia Orthopedic Joint Score (Gilbert score)	5 years period
Proportions of patients who will change the therapeutic regimen during the study	5 years period
Musculoskeletal evaluation by calculating the Radiological score (Pettersson Score)	5 years period
Evaluating the Questionnaires of Health-Related Quality of Life _Haemo Quality of Life (HaemoQol)	5 years period
Evaluating the Questionnaires of Health-Related Quality of Life _Short Form 36 questions (SF36)	5 years period
Evaluating the Questionnaires of Health-Related Quality of Life _ Euro Quality 5 Dimension (EQ5D)	5 years period
Number of days missed from work or school of patients and caregivers because of all the events haemophilia-related	5 years period
Number of patients with poor compliance	5 years period
Number of patients with adverse events	5 years period