ow dose Prophylaxis study in children with haemaophilia A
Not Applicable
- Conditions
- Health Condition 1: null- HAEMOPHILIA A
- Registration Number
- CTRI/2014/03/004494
- Lead Sponsor
- BAYER AWARD
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Closed to Recruitment of Participants
- Sex
- Not specified
- Target Recruitment
- 30
Inclusion Criteria
1. Severe hemophilia, defined as factor assay showing <1% activity, between 3-7 years of age
2. No single joint should have an HJHS score of more than 5.
3. Not have detectable inhibitors by Bethesda assay ( <0.6 BU) at recruitment
4. Be willing to come for evaluation once in 9-15 months for at least 2 (?4) years. (in case
the study is extended)
Exclusion Criteria
PATIENTS WITH DOCUMENTED INHIBITORS.
NOT WILLING TO COME FOR FOLLOW UP FOR LONG TERM (ATLEAST 4 YEARS)
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To evaluate the feasibility and impact of lower doses of prophylactic clotting factor concentrate (CFC) replacement therapy on the bleeding frequency and musculoskeletal outcome of children with severe hemophilia. <br/ ><br>1.1.2 To correlate this outcome with the dose of CFC used and evaluate if there is a dose-response relationship at these doses.Timepoint: CLINICAL ASSESSMENTS, RADIOLOGICAL ASSESSMENTS, ORTHOPAEDIC ASSESSMENTS WILL BE DONE ONCE IN A YEAR AT YEAR-1,YEAR-2, YEAR-3 AND YEAR-4
- Secondary Outcome Measures
Name Time Method 1.2.1 To familiarize and promote the use of prophylaxis with CFC for children with hemophilia in developing countries. <br/ ><br>1.2.2 To encourage and standardize the assessment and documentation of musculoskeletal outcome in the participating centers for further dissemination to other centers in the country. <br/ ><br>1.2.3 To create models for prophylactic CFC replacement therapy which are practical in developing countries during their evolution towards optimal hemophilia care.Timepoint: ANNUAL VISIT FOR 4 YEARS