HaemophiliaPhase 4CHIPSNCT02999308
A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children
A Phase 4 study testing Observe the patient's condition and then change the regimen in people with Haemophilia, run by Beijing Children's Hospital at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 1 year to 7 years
- Conditions the study is for
- Haemophilia
- What is being tested?
- Observe the patient's condition and then change the regimen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- Aug 2016to Dec 2017
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 7 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, 100054
Wu Runhui, MD, PhD, 0086-010-59617621, runhuiwu@hotmail.com
About this study
Official title: 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.
Read more
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.
- Conditions
- Haemophilia
- Being tested
- Observe the patient's condition and then change the regimen
- Keywords
- haemophilia, prophylaxis, children
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Observe the patient's condition and then change the regimenOther
The patients will be assessed every 3 months and the treatment regimen will be modified by the result of assessment.
| Study group | Role | What they receive |
|---|---|---|
| single arm | Experimental | Not described Receives: Other: Observe the patient's condition and then change the regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of Ultrasound HEAD-US score of index joints
Measured: baseline,3 months, 6 months,9 months and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2016 (estimated)
- Study startAug 2016
- Primary completionSep 2017 (estimated)
- Study completionDec 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for HaemophiliaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Runhui WU, Beijing Children's Hospital
- Investigators
- Wu Runhui, MD, PhD, Principal investigator, Beijing Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02999308
- Sponsor's protocol ID
- CHIPS
- Acronym
- CHIPS
- Status last verified
- Dec 2016
- First submitted
- 1 Sep 2016
- Last updated
- 21 Dec 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Publications
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.
PubMed 8846496Nilsson 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.
PubMed 1640190
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hematology Oncology Center
- ClinicalTrials.gov ID
- NCT02999308
- Acronym / sponsor ID
- CHIPS / CHIPS
- Conditions
- Haemophilia
- Keywords
- haemophilia, prophylaxis, children
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Beijing Children's HospitalUniversity, hospital or other
- Study start
- Aug 2016
- Primary completion
- Sep 2017 (estimated)
- Study completion
- Dec 2017 (estimated)
- First posted
- 21 Dec 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| single arm | Experimental | Other: Observe the patient's condition and then change the regimen |
Who may qualify
- Ages
- 1 year to 7 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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,
Key exclusion criteria (1 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Observe the patient's condition and then change the regimenThe patients will be assessed every 3 months and the treatment regimen will be modified by the result of assessment. | Other | single arm |
Primary outcome
Change of Ultrasound HEAD-US score of index joints
Time frame: baseline,3 months, 6 months,9 months and 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Dec 2016.
- Results publication: PubMed 8846496Berntorp 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 publication: PubMed 1640190Nilsson 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.
- Individual participant dataSharing planned: Undecided
Related trials for Haemophilia
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
RecruitingPhase 3
A Research Study Investigating Denecimig (Mim8) in Adults With Acquired Haemophilia A
Haemophilia A
Active, not recruitingPhase 3
Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa
Haemophilia A (Moderate or Severe)
Enrolling by invitationObservational study
Adverse Event Data Collection From External Registries on Nonacog Beta Pegol
Haemophilia B
Enrolling by invitationObservational study
Adverse Event Data Collection From the EUHASS Registry on Turoctocog Alfa Pegol
Haemophilia A
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Wu Runhui, MD, PhD principal investigator
Beijing Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Observe the patient's condition and then change the regimen in people with Haemophilia, run by Beijing Children's Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 21 Dec 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Wu Runhui, MD, PhD (0086-010-59617621, runhuiwu@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change of Ultrasound HEAD-US score of index joints (time frame: baseline,3 months, 6 months,9 months and 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02999308, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Dec 2016; AzTrials last refreshed its copy on 6 Nov 2026.