check_circleStudy Completed

Hemophilia A

Bayer Identifier:

11856

ClinicalTrials.gov Identifier:

NCT01159587

EudraCT Number:

Not Available

EU CT Number:

Not Available
Prophylaxis versus On-demand Therapy Through Economic Report

Trial 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.

Key Participants Requirements

Sex

Both

Age

12 - 55 Years

  • - 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

  • - 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

Trial summary

Enrollment Goal
58
Trial Dates
July 2004 - December 2010
Phase
N/A
Could I Receive a placebo
No
Products
Kogenate FS (Recombinant Factor VIII, BAY14-2222)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Completed
Many LocationsMany Locations, Italy

Primary Outcome

  • 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
    date_rangeTime Frame:
    Every 6 months
    enhanced_encryption
    Safety Issue:
    Yes

Secondary Outcome

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

Trial design

Observational study evaluating efficacy and costs of secondary prophylaxis vs on-demand therapy with Kogenate Bayer in patients with severe haemophilia A. POTTER
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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