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

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ClinicalTrials.gov Identifier: NCT02999308
Recruitment Status : Unknown
Verified December 2016 by Runhui WU, Beijing Children's Hospital.
Recruitment status was:  Recruiting
First Posted : December 21, 2016
Last Update Posted : December 21, 2016
Sponsor:
Information provided by (Responsible Party):
Runhui WU, Beijing Children's Hospital

Brief Summary:

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.


Condition or disease Intervention/treatment Phase
Haemophilia Other: Observe the patient's condition and then change the regimen Phase 4

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Hematology Oncology Center
Study Start Date : August 2016
Estimated Primary Completion Date : September 2017
Estimated Study Completion Date : December 2017

Resource links provided by the National Library of Medicine


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.




Primary Outcome Measures :
  1. Change of Ultrasound HEAD-US score of index joints [ Time Frame: baseline,3 months, 6 months,9 months and 12 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 7 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02999308


Contacts
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Contact: Wu Runhui, MD, PhD 0086-010-59617621 runhuiwu@hotmail.com

Locations
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China, Beijing
Beijing Children's Hospital, Capital Medical University Recruiting
Beijing, Beijing, China, 100054
Contact: Wu Runhui, MD, PhD    0086-010-59617621    runhuiwu@hotmail.com   
Sponsors and Collaborators
Beijing Children's Hospital
Investigators
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Principal Investigator: Wu Runhui, MD, PhD Beijing Children's Hospital

Publications of Results:
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Responsible Party: Runhui WU, Chief doctor, Beijing Children's Hospital
ClinicalTrials.gov Identifier: NCT02999308     History of Changes
Other Study ID Numbers: CHIPS
First Posted: December 21, 2016    Key Record Dates
Last Update Posted: December 21, 2016
Last Verified: December 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Runhui WU, Beijing Children's Hospital:
haemophilia
prophylaxis
children

Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn