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."

ID: NCT02581969
Status: Active, not recruiting
Phase: N/A
Start Date: April 14, 2016
First Submitted: October 20, 2015
Last Updated: February 22, 2018
Results: N/A
Organization: Bayer
Sponsors & Collaborators: Bayer
Location: Italy
Conditions: Hemophilia A
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Study Description

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

Condition or disease Intervention/treatment Phase

Hemophilia A

Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Other Names
Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Other Names
N/A

Tracking Information

First Submitted DateOctober 20, 2015
Last Update Posted DateFebruary 22, 2018
Actual Start DateApril 14, 2016
Anticipated Completion DateMarch 20, 2018
Actual Primary Completion DateMarch 16, 2017
Results First Submitted DateN/A
Received Results Disposit DateN/A

Current Primary Outcome Measures

  • Number of joint bleeding episodes per year [Time Frame: 5 years period]

Original Primary Outcome Measures

Not Available

Current Secondary Outcome Measures

  • Total number of bleeding episodes per year [Time Frame: 5 years period]

  • Total amount of Recombinant Factor VIII consumption [Time Frame: 5 years period]

  • Changes of prophylaxis dose [Time Frame: 5 years period]

  • Changes of frequency of infusions [Time Frame: 5 years period]

  • Musculoskeletal evaluation by calculating the World Federation of Haemophilia Orthopedic Joint Score (Gilbert score) [Time Frame: 5 years period]

  • Proportions of patients who will change the therapeutic regimen during the study [Time Frame: 5 years period]

  • Musculoskeletal evaluation by calculating the Radiological score (Pettersson Score) [Time Frame: 5 years period]

  • Evaluating the Questionnaires of Health-Related Quality of Life _Haemo Quality of Life (HaemoQol) [Time Frame: 5 years period]

  • Evaluating the Questionnaires of Health-Related Quality of Life _Short Form 36 questions (SF36) [Time Frame: 5 years period]

  • Evaluating the Questionnaires of Health-Related Quality of Life _ Euro Quality 5 Dimension (EQ5D) [Time Frame: 5 years period]

  • Number of days missed from work or school of patients and caregivers because of all the events haemophilia-related [Time Frame: 5 years period]

  • Number of patients with poor compliance [Time Frame: 5 years period]

  • Number of patients with adverse events [Time Frame: 5 years period]

Original Secondary Outcome Measures

Not Available

Study Design

Brief TitleExtension 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."
Official TitleExtension 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
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

Study TypeObservational
Study PhaseN/A
Estimated Enrollment
43
Allocation
Not Available
Interventional Model
Not Available
Masking
Not Available
Primary Purpose
Not Available
Conditions
Hemophilia A
Target Follow-Up Duration N/A
Biospecimen:
N/A
Sampling MethodNon-Probability Sample
Study PopulationA 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.
Intervention
Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Administered 20-30 IU/kg 3 times a week

Other Names
Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Administered only for bleeding episodes

Other Names
Study Groups/Cohorts
Prophylaxis
Prophylaxis group: treatment is based on regularly repeated infusions of clotting factor, 20-30 IU/kg -3 times a week

On-demand
On-demand group: treatment administered when bleeding episode occur

Study Arms
On-demand
On-demand group: treatment administered when bleeding episode occur
Drug : Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Administered only for bleeding episodes

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

Arm Intervention/Treatment
On-demand
On-demand group: treatment administered when bleeding episode occur
Drug : Recombinant Factor VIII (Kogenate FS, BAY14-2222)
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)

Recruitment Information

Recruitment Status:Active, not recruiting
Enrollment43
Completion DateMarch 20, 2018
Eligibility Criteria: 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.
GenderMale
Age17 Years to 60 Years
Accepts Healthy VolunteersNo
Contacts
Not Available
Listed Location Countries
Italy

Administrative Information

NCT Number:NCT02581969
Other Study ID Numbers
18251
Has Data Monitoring CommitteeNo
U.S. FDA-regulated Product Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Device Product Not Approved or Cleared by U.S. FDA: No
IPD Sharing Statement
Not Available
Responsible Party,
Study Sponsor
Bayer
Collaborators
Not Available
Investigators
Study Director
Bayer Study Director
Bayer