NCT02116855

Individualized Prophylaxis for Severe Hemophilia A Children

Individualized Tertiary Low Dose Prophylaxis for Severe Hemophilia A Children With Arthropathy in China

• Status: Unknown
• Phase: N/A
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Beijing Children's Hospital · Academic / Other
• Sex: Male
• Age: 6 Years – 10 Years
• Healthy volunteers: Not accepted

Summary

A multi centre two year long term escalating dose tertiary prophylaxis study on the efficacy and cost saving of individualized low dose prophylaxis regimens for boys with severe hemophilia A in China staring with a low dose regimen in step I, an escalated low dose regimen in step II and a tailored dose regimen based on individual PK profiles in step III. The dose escalation criteria are adjusted according to patterns and frequencies of joint bleeding and assessed in each subject every 3 months. Efficacy of the 3 different dose regimens are measured by the Annualized Joint Bleeding rate (AJBR) as a primary end point and the Hemophilia Joint Health Score (HJHS ) and QoL scores (CHO-KLAT and PedsQoL) , image studies of target joints by Ultrasound, X-ray and MRI examinations, consumption of factor VIII and inhibitor rates as secondary end points.

Conditions

• Hemophilia

Interventions

Type	Name	Description
OTHER	according to the efficacy of AJBRs

Timeline

• Start date: 2014-06-01
• Primary completion: 2016-06-01
• First posted: 2014-04-17
• Last updated: 2014-04-17