A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children (CHIPS)

December 17, 2016 updated by: Runhui WU, Beijing Children's Hospital

Hematology Oncology Center

In the past, due to economic and medical resource constraints, the hemophilia comprehensive care in China was suboptimal. The BCH data of both retrospective and prospective studies reveals that for 4-6y and 6-9y patients with severe hemophilia respectively: 45% and 82% of the patients have suffered from joint bleeding, with mean AJBR of 4.18 and 4.95; and 24.2% and 33.3% of them with AJBR>10 times, led to arthropathies and made their quality of life be heavy affected. Now, with the development of economy and medical science in China, prophylaxis regimens have been initiated in more and more children with hemophilia. Considering the difference between prophylaxis regimens, the frequency of joint bleeding was reduced significantly, the quality of life of hemophilia kids improved. An assessment scoring system for the appropriate validation of individualized prophylaxis treatment regimens are urgently needed.

Before, the most important assessed indication for hemophilia prophylaxis was the frequency of joint bleeding. But increasing evidences are showing that there is a discrepancy between real joint damage and joint bleeding frequency. The single indicator of joint bleeding frequency is not sufficient to evaluate the joint status of hemophilia children.

Under the World Health Organization's ICF guidelines, the assessment scoring system for selecting prophylaxis for children with hemophilia should include the tools currently available for assessment of structure/function of the joint, patient activities and patient participation in hemophilia healthcare. According to ICF of WHO, the most common bleeding parts are elbows, knees and ankles, therefore the assessment of children with hemophilia should include the evaluation of the structure, the function of these 6 Index joints, the capacity of activities and the capacity of participation of children. These will constitute a comprehensive hemophilia evaluation system.

In China, exploration of the optimal and individualized prophylaxis regimen is urgent, and the comprehensive evaluation system should include joint structure and function, body's activities and individual participation, thus may be more appropriate for the individualized prophylaxis for Chinese children with hemophilia.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

40

Phase

  • Phase 4

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

Study Locations

  • China
    • Beijing
      • Beijing, Beijing, China, 100054
        • Recruiting
        • Beijing Children's Hospital, Capital Medical University
        • 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

1 year to 7 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Severe hemophilia A (FVIII: C<2%),
  2. Age 1-7y
  3. Historical bleeding in any knee, elbow and ankle joint (s),
  4. >50 EDs irrespective of FVIII product, including human coagulate factor and recombinant factor VIII
  5. No inhibitor present or history/family history,
  6. On-demand or low-dose prophylaxis (the dose per kg.week less or as Step 1 prophylaxis regimens factor consumption)
  7. Regular clinical visit with accessible data,
  8. Informed consent will be obtained from patient legal guardians before the enrollment.

Exclusion Criteria:

  • 1. Other bleeding disorders or systemic disorders, or don't fit for enrollment according to the investigator, 2. FVIII inhibitor development:>0.6 BU (confirmed by two separate tests), 3. Unable to follow the protocol or refuse to continue the participation.

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: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: single arm
Other: Observe the patient's condition and then change the regimen
The patients will be assessed every 3 months and the treatment regimen will be modified by the result of assessment.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change of Ultrasound HEAD-US score of index joints
Time Frame: baseline,3 months, 6 months,9 months and 12 months
baseline,3 months, 6 months,9 months and 12 months

Collaborators and Investigators

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

Sponsor

Beijing Children's Hospital

Investigators

  • Principal Investigator: Wu Runhui, MD, PhD, Beijing Children's Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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

August 1, 2016

Primary Completion (Anticipated)

September 1, 2017

Study Completion (Anticipated)

December 1, 2017

Study Registration Dates

First Submitted

September 1, 2016

First Submitted That Met QC Criteria

December 17, 2016

First Posted (Estimate)

December 21, 2016

Study Record Updates

Last Update Posted (Estimate)

December 21, 2016

Last Update Submitted That Met QC Criteria

December 17, 2016

Last Verified

December 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CHIPS

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

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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