CHIPS: A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children

Sponsor

Beijing Children's Hospital (Other)

Overall Status

Unknown status

CT.gov ID

NCT02999308

Collaborator

(none)

Enrollment

Location

Arm

Duration (Months)

2.5

Patients Per Site Per Month

Study Details

Study Description

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

Study Design

Study Type:

Interventional

Anticipated Enrollment :

40 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Hematology Oncology Center

Study Start Date :

Aug 1, 2016

Anticipated Primary Completion Date :

Sep 1, 2017

Anticipated Study Completion Date :

Dec 1, 2017

Arms and Interventions

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.

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.

Outcome Measures

Primary Outcome Measures

Change of Ultrasound HEAD-US score of index joints [baseline,3 months, 6 months,9 months and 12 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year to 7 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Severe hemophilia A (FVIII: C<2%),
Age 1-7y
Historical bleeding in any knee, elbow and ankle joint (s),
50 EDs irrespective of FVIII product, including human coagulate factor and recombinant factor VIII
No inhibitor present or history/family history,
On-demand or low-dose prophylaxis (the dose per kg.week less or as Step 1 prophylaxis regimens factor consumption)
Regular clinical visit with accessible data,
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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Beijing Children's Hospital, Capital Medical University	Beijing	Beijing	China	100054

Sponsors and Collaborators

Beijing Children's Hospital

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Runhui WU, Chief doctor, Beijing Children's Hospital

ClinicalTrials.gov Identifier:

NCT02999308

Other Study ID Numbers:

CHIPS

First Posted:

Dec 21, 2016

Last Update Posted:

Dec 21, 2016

Last Verified:

Dec 1, 2016

Individual Participant Data (IPD) Sharing Statement:

Undecided

Plan to Share IPD:

Undecided

Keywords provided by Runhui WU, Chief doctor, Beijing Children's Hospital

haemophilia

prophylaxis

children

Additional relevant MeSH terms:

Hemophilia A

Study Results

No Results Posted as of Dec 21, 2016