A prospective controlled study on the effect on bleeding events and joint function in young adults with severe hemophilia A after a 6 month prophylaxis treatment compared to on demand treatment

Phase 1

• Conditions: Hemophilia A; MedDRA version: 7.1 Level: low Classification code 10018937

• Registration Number: EUCTR2005-002757-45-GB
• Lead Sponsor: Bayer HealthCare LLC/Biological Products

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2005-09-12
• Study Completion: 2008-01-31
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 0

Inclusion Criteria

• Male
• Severe hemophilia A (< 1% FVIII) and severe clinical behavior with an average of 2 relevant bleeds per month, no additional bleeding disorder
• 30-45 years of age
• Previously treated subject (>100 Exposure days to any FVIII)
• On on-demand therapy with any FVIII
• No history of inhibitor (screening must include a negative inhibitor assay)
• Informed consent signed
• Available results for HIV, HAV, HBV, HCV (can be tested at screening if not available)

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• Current or prior inhibitor (inhibitor titer of = 0.6 Bethesda units (BU))
• Planned elective orthopedic surgery during the study duration (13 months)
• Presence of an additional bleeding disorder or other medical or psychological condition which could affect treatment efficacy and QoL outcome (e.g. severe symptomatic HIV infection, advanced liver disease, renal failure).
• History of anaphylactic or other severe reaction to previous AHF treatment
• Known hypersensitivity to the active substance, to mouse or hamster protein or to any of the excipients

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<br> Main Objective: Primary objective is to evaluate the effect of prophylactic treatment on the number of joint bleeds in severe hemophilia A subjects compared to on-demand treatment in a one-group two-treatment schedule design.<br> ;<br> Secondary Objective: Secondary objective is to evaluate the effect of prophylactic treatment on the joint function, the number of all bleeds, and on the quality of life compared to on-demand treatment. Health-economic data will also be assessed.<br> ;Primary end point(s): The primary endpoint will be the number of joint bleeds which will be recorded during 6 months (i.e. months 7 – 13)of prophylactic treatment and will be compared to the number of joint bleeds during the 6 months (i.e. months 1 – 6) on-demand treatment. The first month on prophylaxis will be a run-in period for stabilization and washout and will not be evaluated.