Back to resultsIndividualized Prophylaxis for Severe Hemophilia A Children
Primary Purpose
Hemophilia
Status
Unknown status
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
according to the efficacy of AJBRs
Sponsored by
About this trial
This is an interventional treatment trial for Hemophilia
Eligibility Criteria
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
tertiary prophylaxis prophy on Hemophilia A patiets
Arm Description
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.
Outcomes
Primary Outcome Measures
AJBRs (annualized Joint bleeding rates)
Secondary Outcome Measures
Full Information
NCT ID
NCT02116855
First Posted
April 15, 2014
Last Updated
April 15, 2014
Sponsor
Beijing Children's Hospital
1. Study Identification
Unique Protocol Identification Number
NCT02116855
Brief Title
Individualized Prophylaxis for Severe Hemophilia A Children
Official Title
Individualized Tertiary Low Dose Prophylaxis for Severe Hemophilia A Children With Arthropathy in China
Study Type
Interventional
2. Study Status
Record Verification Date
April 2014
Overall Recruitment Status
Unknown status
Study Start Date
June 2014 (undefined)
Primary Completion Date
June 2016 (Anticipated)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Beijing Children's Hospital
4. Oversight
5. Study Description
Brief 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 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
Outcomes Assessor
Allocation
N/A
Enrollment
50 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
tertiary prophylaxis prophy on Hemophilia A patiets
Arm Type
Experimental
Arm Description
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.
Intervention Type
Other
Intervention Name(s)
according to the efficacy of AJBRs
Primary Outcome Measure Information:
Title
AJBRs (annualized Joint bleeding rates)
Time Frame
three months
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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
Exclusion Criteria:
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Hui R Wu, MD
Phone
8613911383480
Email
runhuiwu@hotmail.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
hui R Wu, MD
Organizational Affiliation
Beijing Children's Hospital
Official's Role
Study Chair
12. IPD Sharing Statement
Learn more about this trial
Individualized Prophylaxis for Severe Hemophilia A Children
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