Text Size

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

This study has been completed.
Sponsor:
Information provided by:
Bayer
ClinicalTrials.gov Identifier:
NCT01159587
First received: July 8, 2010
Last updated: April 12, 2011
Last verified: April 2011
History of Changes
  Purpose

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.


Condition Intervention
Hemophilia A
 Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A. POTTER

Resource links provided by NLM:

MedlinePlus related topics: Hemophilia
U.S. FDA Resources

Further study details as provided by Bayer:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Efficacy, safety, pharmacoeconomic impact of secondary prophylaxis versus on demand group in terms of overall bleeding episodes [ Time Frame: Every 6 months ] [ Designated as safety issue: No ]
  • Muscolo skeletal evaluation by Orthopedic Joint Score [ Time Frame: Every 12 months ] [ Designated as safety issue: No ]
  • Radiological Evaluation by Pettersson Score [ Time Frame: Baseline and after 3-5 years ] [ Designated as safety issue: No ]
  • Health related quality of life [ Time Frame: Every 12 months ] [ Designated as safety issue: No ]
  • cost-effectiveness and utility, patient compliance, adverse events [ Time Frame: Every 6 months ] [ Designated as safety issue: Yes ]

Enrollment: 58
Study Start Date: July 2004
Study Completion Date: December 2010
Primary Completion Date: December 2010 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Group 1 Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Long-term secondary prophylaxis with product administered 20-30 UI /kg three times weekly
Group 2 Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
On-demand treatment with product given only for bleeding episodes

  Eligibility

Ages Eligible for Study:   12 Years to 55 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

Haemophilics

Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01159587

Locations
Italy
Many Locations, Italy
Sponsors and Collaborators
Bayer
Investigators
Study Director: Bayer Study Director Bayer
  More Information

Additional Information:
Click here and search for drug information provided by the FDA.  This link exits the ClinicalTrials.gov site
Click here and search for information on any recalls, market or product safety alerts by the FDA which might have occurred with this product.  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: Medical Director, Bayser Schering Pharma AG
ClinicalTrials.gov Identifier: NCT01159587     History of Changes
Other Study ID Numbers: 11856
Study First Received: July 8, 2010
Last Updated: April 12, 2011
Health Authority: Italy: ECs

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
 Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 16, 2013