Individualized Prophylaxis for Severe Hemophilia A Children

April 15, 2014 updated by: Runhui WU, Beijing Children's Hospital

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

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

50

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 10 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  1. Severe hemophilia A with FVIII <1 %
  2. Age from 6 to 10 years
  3. Patients with more than 50 exposure day (ED)
  4. Patients with one or more target joints (a target joint is defined as a joint with 3 or more bleeds in a consecutive 3 months) or disease joints (defined as presence of visible joint swelling and /or limitation of movements and/or joint deformities in the absent of an acute joint bleed )
  5. on-demand treatment more than 12months before the study

Exclusion Criteria:

  1. A history of FVIII inhibitor (titer greater than 0.6 BU) and detectable FVIII inhibitor at screening (titer greater than 0.6 BU)
  2. Chronic renal failure (serum creatinine greater than 2.0 mg /dL)
  3. Chronic Liver disease (ALT greater than 200 U/L))
  4. Patients with clinically documented immunodeficiencies
  5. Patients anticipated to require Major surgery
  6. Patients with competing high risks such as symptomatic HIV infection, Juvenile rheumatoid arthritis, metabolic bone diseases, or other diseases known to mimic or cause joint diseases
  7. Patients live too far from the comprehensive care centre at BCH and had demonstrated previous non compliance to therapies or clinical studies

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: tertiary prophylaxis prophy on Hemophilia A patiets
A multi centre 2 year long term tertiary prophylaxis study designed with a three step escalating dose protocol adjusted by individual joint bleeding patterns/frequencies to study the effect and cost saving of 3 low dose /cost prophylaxis regimens in boys with severe hemophilia A and arthropathy in China. Each patient will start treatment with a low dose regimen in step I and be assessed every 3 months according to the escalating criteria.
Other: according to the efficacy of AJBRs

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
AJBRs (annualized Joint bleeding rates)
Time Frame: three months
three months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Beijing Children's Hospital

Investigators

  • Study Chair: hui R Wu, MD, Beijing Children's Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2014

Primary Completion (Anticipated)

June 1, 2016

Study Registration Dates

First Submitted

April 15, 2014

First Submitted That Met QC Criteria

April 15, 2014

First Posted (Estimate)

April 17, 2014

Study Record Updates

Last Update Posted (Estimate)

April 17, 2014

Last Update Submitted That Met QC Criteria

April 15, 2014

Last Verified

April 1, 2014

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BCH-20140415

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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