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Completed

NCT Number: NCT01159587

Prophylaxis Versus On-demand Therapy Through Economic Report

The project is a controlled observational, multicenter, prospective data collection on secondary prophylaxis with Kogenate Bayer in adolescents and adults with severe haemophilia A (FVIII < 1%).

The observational period will cover at least 5 years per patient.

The long-term secondary prophylaxis group will be compared versus on-demand treatment group by the assessment of orthopedic status progression and pharmacoeconomics evaluation.

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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

12 year–55 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Many Locations, Italy

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • age ≥ 12 years and ≤ 55 years
  • severe haemophilia A (FVIII < 1%)
  • absence of inhibitors (Bethesda titre < 0.6 BU/ml)
  • Previous Treated Patients (prior exposure days > 200)
  • Kogenate Bayer administered 20-30 IU/kg -3 times a week, for the prophylaxis group
  • ≥ 6 joint bleeds requiring treatment with FVIII concentrates in the previous 6 months before enrollment, for on-demand group
  • written informed consent

Exclusion criteria

  • concomitant severe and chronic diseases or congenital skeletal malformation
  • unreliability of patient or likelihood of follow-up failure
  • presence of inhibitors or history of inhibitors (in the previous 2 years)
  • currently on immune tolerance treatment
  • hepatic cirrhosis or liver disease in rapid progression
  • AIDS
  • platelet count < 75,000/mm3
  • presence of conditions that influence negatively patient´s compliance
  • participation in another study

Treatment and study plan

Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Drug

Long-term secondary prophylaxis with product administered 20-30 UI /kg three times weekly

Primary outcomes

  1. Evaluate the efficacy, safety and pharmacoeconomic of long-term secondary prophylaxis with Kogenate Bayer in comparison to on-demand treatment with Kogenate Bayer in terms of joint bleeding episodes per year

    Time frame: Every 6 months

Secondary outcomes

  1. Efficacy, safety, pharmacoeconomic impact of secondary prophylaxis versus on demand group in terms of overall bleeding episodes

    Time frame: Every 6 months

  2. Muscolo skeletal evaluation by Orthopedic Joint Score

    Time frame: Every 12 months

  3. Radiological Evaluation by Pettersson Score

    Time frame: Baseline and after 3-5 years

  4. Health related quality of life

    Time frame: Every 12 months

  5. cost-effectiveness and utility, patient compliance, adverse events

    Time frame: Every 6 months

Sponsors and collaborators

Lead sponsor

Bayer

Industry

Registry information

Official study title

Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A. POTTER

Acronym: POTTER

Important dates

Study start
2004
Primary completion
2010
Study completion
2010
First posted
Jul 9, 2010
Registry last updated
Apr 13, 2011

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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