Prophylaxis Versus On-demand Therapy Through Economic Report (POTTER)

April 12, 2011 updated by: Bayer

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

58

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Many Locations, Italy

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years to 55 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

Haemophilics

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Group 1
Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Long-term secondary prophylaxis with product administered 20-30 UI /kg three times weekly
Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
On-demand treatment with product given only for bleeding episodes
Group 2
Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Long-term secondary prophylaxis with product administered 20-30 UI /kg three times weekly
Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
On-demand treatment with product given only for bleeding episodes

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
Every 6 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Efficacy, safety, pharmacoeconomic impact of secondary prophylaxis versus on demand group in terms of overall bleeding episodes
Time Frame: Every 6 months
Every 6 months
Muscolo skeletal evaluation by Orthopedic Joint Score
Time Frame: Every 12 months
Every 12 months
Radiological Evaluation by Pettersson Score
Time Frame: Baseline and after 3-5 years
Baseline and after 3-5 years
Health related quality of life
Time Frame: Every 12 months
Every 12 months
cost-effectiveness and utility, patient compliance, adverse events
Time Frame: Every 6 months
Every 6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Bayer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2004

Primary Completion (Actual)

December 1, 2010

Study Completion (Actual)

December 1, 2010

Study Registration Dates

First Submitted

July 8, 2010

First Submitted That Met QC Criteria

July 8, 2010

First Posted (Estimate)

July 9, 2010

Study Record Updates

Last Update Posted (Estimate)

April 13, 2011

Last Update Submitted That Met QC Criteria

April 12, 2011

Last Verified

April 1, 2011

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 11856

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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