Prophylaxis Versus On-demand Therapy Through Economic Report

Hemophilia A Clinical Trial

— POTTER  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01159587
• Other study ID #: 11856
• Status: Completed
• Phase: N/A
• First received: July 8, 2010
• Last updated: April 12, 2011
• Start date: July 2004
• Est. completion date: December 2010

Study information

• Verified date: April 2011
• Source: Bayer
• Contact: n/a
• Is FDA regulated: No
• Health authority: Italy: ECs
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 58
• Est. completion date: December 2010
• Est. primary completion date: December 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years to 55 Years

Eligibility

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 Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Hemophilia A

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Bayer

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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		Every 6 months	Yes
Secondary	Efficacy, safety, pharmacoeconomic impact of secondary prophylaxis versus on demand group in terms of overall bleeding episodes		Every 6 months	No
Secondary	Muscolo skeletal evaluation by Orthopedic Joint Score		Every 12 months	No
Secondary	Radiological Evaluation by Pettersson Score		Baseline and after 3-5 years	No
Secondary	Health related quality of life		Every 12 months	No
Secondary	cost-effectiveness and utility, patient compliance, adverse events		Every 6 months	Yes

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