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)

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

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.

Study Overview

• Status: Completed
• Conditions: Hemophilia A
• Intervention / Treatment: Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222); Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

• Study Type: Observational
• Enrollment (Actual): 43

Contacts and Locations

Study Locations

• Italy
  • Multiple Locations, Italy

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 17 years to 60 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Non-Probability Sample

Study Population

A total of approximately 50 patients with a diagnosis of severe haemophilia A (FVIII < 1%) , who are already on Kogenate Bayer treatment ongoing for long-term secondary prophylaxis or on-demand therapy and have properly completed the follow-up in the original Potter study are expected to be enrolled.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of joint bleeding episodes per year	5 years period

Secondary Outcome Measures

Outcome Measure	Time Frame
Total number of bleeding episodes per year	5 years period
Total amount of Recombinant Factor VIII consumption	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

Collaborators and Investigators

• Sponsor: Bayer

Study record dates

Study Major Dates

• Study Start (Actual): April 14, 2016
• Primary Completion (Actual): March 16, 2017
• Study Completion (Actual): June 21, 2018

Study Registration Dates

• First Submitted: October 20, 2015
• First Submitted That Met QC Criteria: October 20, 2015
• First Posted (Estimate): October 21, 2015

Study Record Updates

• Last Update Posted (Actual): November 18, 2020
• Last Update Submitted That Met QC Criteria: November 17, 2020
• Last Verified: November 1, 2020

More Information

Terms related to this study

• Keywords: Hemophilia A, Congenital
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Coagulants; Factor VIII
• Other Study ID Numbers: 18251 (Other Identifier: City of Hope Medical Center)