check_circleStudy Completed

Hemophilia A

Bayer Identifier:

18251

ClinicalTrials.gov Identifier:

NCT02581969

EudraCT Number:

Not Available

EU CT Number:

Not Available
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.»

Trial purpose

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.

Key Participants Requirements

Sex

Male

Age

17 - 60 Years
  • - 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.
  • - Switching of treatment to a Factor VIII concentrate different from Kogenate Bayer/Helixate NexGen after the end of the previous follow-up period.

Trial summary

Enrollment Goal
43
Trial Dates
April 2016 - June 2018
Phase
Phase 4
Could I Receive a placebo
No
Products
Kogenate FS (Recombinant Factor VIII, BAY14-2222)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Completed
Many Locations, Italy

Primary Outcome

  • Number of joint bleeding episodes per year
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    No

Secondary Outcome

  • Total number of bleeding episodes per year
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    No
  • Total amount of Recombinant Factor VIII consumption
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    No
  • Changes of prophylaxis dose
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    No
  • Changes of frequency of infusions
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    No
  • Musculoskeletal evaluation by calculating the World Federation of Haemophilia Orthopedic Joint Score (Gilbert score)
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    No
  • Proportions of patients who will change the therapeutic regimen during the study
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    No
  • Musculoskeletal evaluation by calculating the Radiological score (Pettersson Score)
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    No
  • Evaluating the Questionnaires of Health-Related Quality of Life _Haemo Quality of Life (HaemoQol)
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    No
  • Evaluating the Questionnaires of Health-Related Quality of Life _Short Form 36 questions (SF36)
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    No
  • Evaluating the Questionnaires of Health-Related Quality of Life _ Euro Quality 5 Dimension (EQ5D)
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    No
  • Number of days missed from work or school of patients and caregivers because of all the events haemophilia-related
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    No
  • Number of patients with poor compliance
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    Yes
  • Number of patients with adverse events
    date_rangeTime Frame:
    5 years period
    enhanced_encryption
    Safety Issue:
    Yes

Trial design

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
Trial Type
Observational
Intervention Type
Drug
Trial Purpose
N/A
Allocation
N/A
Blinding
N/A
Assignment
N/A
Trial Arms
N/A

Additional Information

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