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Individualized Prophylaxis for Severe Hemophilia A Children

NCT02116855 · Status: UNKNOWN · Phase: NA · Type: INTERVENTIONAL · Enrollment: 50

Last updated 2014-04-17

No results posted yet for this study

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

Interventions

OTHER

according to the efficacy of AJBRs

Sponsors & Collaborators

  • Beijing Children's Hospital

    lead OTHER

Principal Investigators

  • hui R Wu, MD · Beijing Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
SINGLE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
10 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-06-30
Primary Completion
2016-06-30

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02116855 on ClinicalTrials.gov