HemophiliaNCT02116855
Individualized Prophylaxis for Severe Hemophilia A Children
A study testing according to the efficacy of AJBRs in people with Hemophilia, run by Beijing Children's Hospital.
Trial summary
A multi centre two year long term escalating dose tertiary prophylaxis study on the efficacy and cost saving of individualized low dose prophylaxis regimens for boys with severe hemophilia A in China staring with a low dose regimen in step I, an escalated low dose regimen in step II and a tailored dose regimen based… Read more about this study
- Who is it for?
- Ages 6 to 10 years, men only
- Conditions the study is for
- Hemophilia
- What is being tested?
- according to the efficacy of AJBRs
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- Jun 2014to Jun 2016
- Phase
- Interventional
- 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 6 to 10 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Severe hemophilia A with FVIII <1 %
- Age from 6 to 10 years
- Patients with more than 50 exposure day (ED)
- Patients with one or more target joints (a target joint is defined as a joint with 3 or more bleeds in a consecutive 3 months) or disease joints (defined as presence of visible joint swelling and /or limitation of movements and/or joint deformities in the absent of an acute joint bleed )
- on-demand treatment more than 12months before the study
Who cannot join
- A history of FVIII inhibitor (titer greater than 0.6 BU) and detectable FVIII inhibitor at screening (titer greater than 0.6 BU)
- Chronic renal failure (serum creatinine greater than 2.0 mg /dL)
- Chronic Liver disease (ALT greater than 200 U/L))
- Patients with clinically documented immunodeficiencies
- Patients anticipated to require Major surgery
- Patients with competing high risks such as symptomatic HIV infection, Juvenile rheumatoid arthritis, metabolic bone diseases, or other diseases known to mimic or cause joint diseases
- Patients live too far from the comprehensive care centre at BCH and had demonstrated previous non compliance to therapies or clinical studies
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Individualized Tertiary Low Dose Prophylaxis for Severe Hemophilia A Children With Arthropathy in China
A multi centre two year long term escalating dose tertiary prophylaxis study on the efficacy and cost saving of individualized low dose prophylaxis regimens for boys with severe hemophilia A in China staring with a low dose regimen in step I, an escalated low dose regimen in step II and a tailored dose regimen based on individual PK profiles in step III. The dose escalation criteria are adjusted according to patterns and frequencies of joint bleeding and assessed in each subject every 3 months. Efficacy of the 3 different dose regimens are measured by the Annualized Joint Bleeding rate (AJBR) as a primary end point and the Hemophilia Joint Health Score (HJHS ) and QoL scores (CHO-KLAT and PedsQoL) , image studies of target joints by Ultrasound, X-ray and MRI examinations, consumption of factor VIII and inhibitor rates as secondary end points.
- Conditions
- Hemophilia
- Being tested
- according to the efficacy of AJBRs
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
according to the efficacy of AJBRsOther
| Study group | Role | What they receive |
|---|---|---|
| tertiary prophylaxis prophy on Hemophilia A patiets | Experimental | A multi centre 2 year long term tertiary prophylaxis study designed with a three step escalating dose protocol adjusted by individual joint bleeding patterns/frequencies to study the effect and cost saving of 3 low dose /cost prophylaxis regimens in boys with severe hemophilia A and arthropathy in China. Each patient will start treatment with a low dose regimen in step I and be assessed every 3 months according to the escalating criteria. Receives: Other: according to the efficacy of AJBRs |
What is being measured
The primary outcome is the main question the study is designed to answer.
AJBRs (annualized Joint bleeding rates)
Measured: three months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2014 (estimated)
- Study startJun 2014
- Primary completionJun 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 50 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
- hui R Wu, MD, Study chair, Beijing Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02116855
- Sponsor's protocol ID
- BCH-20140415
- Status last verified
- Apr 2014
- First submitted
- 15 Apr 2014
- Last updated
- 17 Apr 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Individualized Tertiary Low Dose Prophylaxis for Severe Hemophilia A Children With Arthropathy in China
- ClinicalTrials.gov ID
- NCT02116855
- Acronym / sponsor ID
- BCH-20140415
- Conditions
- Hemophilia
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Beijing Children's HospitalUniversity, hospital or other
- Study start
- Jun 2014
- Primary completion
- Jun 2016 (estimated)
- First posted
- 17 Apr 2014 (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.
A multi centre two year long term escalating dose tertiary prophylaxis study on the efficacy and cost saving of individualized low dose prophylaxis regimens for boys with severe hemophilia A in China staring with a low dose regimen in step I, an escalated low dose regimen in step II and a tailored dose regimen based on individual PK profiles in step III. The dose escalation criteria are adjusted according to patterns and frequencies of joint bleeding and assessed in each subject every 3 months. Efficacy of the 3 different dose regimens are measured by the Annualized Joint Bleeding rate (AJBR) as a primary end point and the Hemophilia Joint Health Score (HJHS ) and QoL scores (CHO-KLAT and PedsQoL) , image studies of target joints by Ultrasound, X-ray and MRI examinations, consumption of factor VIII and inhibitor rates as secondary end points.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| tertiary prophylaxis prophy on Hemophilia A patiets | Experimental | Other: according to the efficacy of AJBRsA multi centre 2 year long term tertiary prophylaxis study designed with a three step escalating dose protocol adjusted by individual joint bleeding patterns/frequencies to study the effect and cost saving of 3 low dose /cost prophylaxis regimens in boys with severe hemophilia A and arthropathy in China. Each patient will start treatment with a low dose regimen in step I and be assessed every 3 months according to the escalating criteria. |
Who may qualify
- Ages
- 6 to 10 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Severe hemophilia A with FVIII <1 %
- Age from 6 to 10 years
- Patients with more than 50 exposure day (ED)
- Patients with one or more target joints (a target joint is defined as a joint with 3 or more bleeds in a consecutive 3 months) or disease joints (defined as presence of visible joint swelling and /or limitation of movements and/or joint deformities in the absent of an acute joint bleed )
- on-demand treatment more than 12months before the study
Key exclusion criteria (7 listed)
- A history of FVIII inhibitor (titer greater than 0.6 BU) and detectable FVIII inhibitor at screening (titer greater than 0.6 BU)
- Chronic renal failure (serum creatinine greater than 2.0 mg /dL)
- Chronic Liver disease (ALT greater than 200 U/L))
- Patients with clinically documented immunodeficiencies
- Patients anticipated to require Major surgery
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 |
|---|---|---|
| according to the efficacy of AJBRs | Other | tertiary prophylaxis prophy on Hemophilia A patiets |
Primary outcome
AJBRs (annualized Joint bleeding rates)
Time frame: three months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Apr 2014.
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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.
hui R Wu, MD study chair
Beijing Children's Hospital
Questions about this trial
What is this clinical trial studying?
A study testing according to the efficacy of AJBRs in people with Hemophilia, run by Beijing Children's Hospital.
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 5 inclusion and 7 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 17 Apr 2014. Status can change between updates, so confirm with the study contact before making plans.
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.
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 Hui R Wu, MD (8613911383480, runhuiwu@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
AJBRs (annualized Joint bleeding rates) (time frame: three months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Apr 2014.