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Completed

NCT Number: NCT02581969

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."

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

17 year–60 year

Sex eligibility

Male

Study type

Observational

Primary location

Multiple Locations, Italy

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Drug

Administered 20-30 IU/kg 3 times a week

Primary outcomes

  1. Number of joint bleeding episodes per year

    Time frame: 5 years period

Secondary outcomes

  1. Total number of bleeding episodes per year

    Time frame: 5 years period

  2. Total amount of Recombinant Factor VIII consumption

    Time frame: 5 years period

  3. Changes of prophylaxis dose

    Time frame: 5 years period

  4. Changes of frequency of infusions

    Time frame: 5 years period

  5. Musculoskeletal evaluation by calculating the World Federation of Haemophilia Orthopedic Joint Score (Gilbert score)

    Time frame: 5 years period

  6. Proportions of patients who will change the therapeutic regimen during the study

    Time frame: 5 years period

  7. Musculoskeletal evaluation by calculating the Radiological score (Pettersson Score)

    Time frame: 5 years period

  8. Evaluating the Questionnaires of Health-Related Quality of Life _Haemo Quality of Life (HaemoQol)

    Time frame: 5 years period

  9. Evaluating the Questionnaires of Health-Related Quality of Life _Short Form 36 questions (SF36)

    Time frame: 5 years period

  10. Evaluating the Questionnaires of Health-Related Quality of Life _ Euro Quality 5 Dimension (EQ5D)

    Time frame: 5 years period

  11. Number of days missed from work or school of patients and caregivers because of all the events haemophilia-related

    Time frame: 5 years period

  12. Number of patients with poor compliance

    Time frame: 5 years period

  13. Number of patients with adverse events

    Time frame: 5 years period

Sponsors and collaborators

Lead sponsor

Bayer

Industry

Registry information

Official study 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

Acronym: POTTER 10

Important dates

Study start
2016
Primary completion
2017
Study completion
2018
First posted
Oct 21, 2015
Registry last updated
Nov 18, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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