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."

Hemophilia A Clinical Trial

— POTTER 10  

Official title:

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." POTTER-10

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02581969
• Other study ID #: 18251
• Status: Completed
• Start date: April 14, 2016
• Est. completion date: June 21, 2018

Study information

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

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 43
• Est. completion date: June 21, 2018
• Est. primary completion date: March 16, 2017
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 17 Years to 60 Years

Eligibility

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.

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.

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Drug:
• Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Administered 20-30 IU/kg 3 times a week
• Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Administered only for bleeding episodes

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Bayer

Country where clinical trial is conducted

Italy, 

Outcome

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