A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children (CHIPS)
Primary Purpose
Haemophilia
Status
Unknown status
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
Observe the patient's condition and then change the regimen
Sponsored by
About this trial
This is an interventional treatment trial for Haemophilia focused on measuring haemophilia, prophylaxis, children
Eligibility Criteria
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.
Sites / Locations
- Beijing Children's Hospital, Capital Medical UniversityRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
single arm
Arm Description
Outcomes
Primary Outcome Measures
Change of Ultrasound HEAD-US score of index joints
Secondary Outcome Measures
Full Information
NCT ID
NCT02999308
First Posted
September 1, 2016
Last Updated
December 17, 2016
Sponsor
Beijing Children's Hospital
1. Study Identification
Unique Protocol Identification Number
NCT02999308
Brief Title
A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children
Acronym
CHIPS
Official Title
Hematology Oncology Center
Study Type
Interventional
2. Study Status
Record Verification Date
December 2016
Overall Recruitment Status
Unknown status
Study Start Date
August 2016 (undefined)
Primary Completion Date
September 2017 (Anticipated)
Study Completion Date
December 2017 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Beijing Children's Hospital
4. Oversight
Data Monitoring Committee
Yes
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Haemophilia
Keywords
haemophilia, prophylaxis, children
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
single arm
Arm Type
Experimental
Intervention Type
Other
Intervention Name(s)
Observe the patient's condition and then change the regimen
Intervention Description
The patients will be assessed every 3 months and the treatment regimen will be modified by the result of assessment.
Primary Outcome Measure Information:
Title
Change of Ultrasound HEAD-US score of index joints
Time Frame
baseline,3 months, 6 months,9 months and 12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Wu Runhui, MD, PhD
Phone
0086-010-59617621
Email
runhuiwu@hotmail.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Wu Runhui, MD, PhD
Organizational Affiliation
Beijing Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Beijing Children's Hospital, Capital Medical University
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100054
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Wu Runhui, MD, PhD
Phone
0086-010-59617621
Email
runhuiwu@hotmail.com
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Citations:
PubMed Identifier
8846496
Citation
Berntorp E, Boulyjenkov V, Brettler D, Chandy M, Jones P, Lee C, Lusher J, Mannucci P, Peak I, Rickard K, et al. Modern treatment of haemophilia. Bull World Health Organ. 1995;73(5):691-701.
Results Reference
result
PubMed Identifier
1640190
Citation
Nilsson IM, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992 Jul;232(1):25-32. doi: 10.1111/j.1365-2796.1992.tb00546.x.
Results Reference
result
Learn more about this trial
A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children
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