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Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients

Sponsor
Chulalongkorn University (Other)
Overall Status
Completed
CT.gov ID
NCT02727647
Collaborator
(none)
16
Enrollment
1
Location
2
Arms
10
Duration (Months)
1.6
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Title: Comparison of different prophylaxis regimens for hemophilia A pediatric patients Principal Investigator: Assistant Professor Darintr Sosothikul, MD Research Question: Does different factor VIII dosage effect outcome of hemophilia treatment in term of breakthrough bleeding, number of hospital stay and day-off from school? Type Research: Clinical research

Study design: Single center clinical trials

Concise methodology:

  1. Study Population: Children with hemophilia A who received treatment at KCMH from May 2015 to March 2016 will be enrolled in this study. The consent will be obtained before the study.

  2. Observation and measurement:

  3. History, interesting clinical data and laboratory data will be recorded in Clinical record Form (CRF)

  4. Measurement:

  1. Complete blood count (CBC), Factor VIII level, Factor VIII inhibitor level ii. Number of breakthrough bleedings, number of hospital stay and day-off from school iii. Joint score from Hemophilia Joint Health Score 2.1 iv. EQ-5D-5L quality of life assessment score
  1. Data analysis: The p-value of less than 0.05 will be considered statistically significant. Mann-Whitney test will be used to test correlation of these variables (CBC, Factor VIII level, Factor VIII inhibitor level, Number of breakthrough bleedings, number of hospital stay, day-off from school, Hemophilia Joint Health Score 2.1, EQ-5D-5L quality of life assessment score) Sample size: 16 patients
Potential impacts:

The outcomes of different factor VIII concentrate dose between 15-20 U/kg/dose 2 times/week and 35-40 U/kg/dose 1 time/week will be revealed. These outcomes include number of breakthrough bleeding, number of hospital stay, day-off from school, joint health and quality of life. The result of this study will guide further study on optimal dose and duration of factor VIII treatment of hemophilia A patients in the future.

Condition or Disease Intervention/Treatment Phase
Phase 4

Study Design

Study Type:
Interventional
Actual Enrollment :
16 participants
Allocation:
Non-Randomized
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients
Study Start Date :
May 1, 2015
Actual Primary Completion Date :
Mar 1, 2016
Actual Study Completion Date :
Mar 1, 2016

Arms and Interventions

Arm Intervention/Treatment
Experimental: Arm 1

FVIII concentration at 35-40 U/kg/dose 1 time/week for 5 months

Drug: FVIII
FVIII concentration 35-40 U/kg/dose 1 time/week for 5 months
Other Names:
  • Hemofil-M

    		•
    Experimental: Arm 2

    FVIII concentration at 15-20 U/kg/dose 2 time/week for 5 months

    Drug: FVIII
    FVIII concentration 15-20 U/kg/dose 1 time/week for 5 months
    Other Names:
  • Hemofil-M

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Annualized bleeding rate per year [10 months]

    Secondary Outcome Measures

    1. Hemophilia joint health score [10 months]

    2. Amount of FVIII use [10 months]

    3. Quality of life score [10 months]

      EQ-5D-5L (Thailand version)

    4. Number of hospital stays [10 months]

    5. Number of school days loss [10 months]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    6 Months to 20 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    Yes
    Inclusion Criteria:
    • Hemophilia patients with moderate (factor level 1-3%) or severe (factor level <1%) severity
    Exclusion Criteria:

    • Platelet count less than 100,000 /mm3 or other bleeding tendency

    • Hemophilia patients who have FVIII inhibitor >0.6 BU (modified Nijmogen method)

    • Hemophilia patients who have no bleeding symptoms

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Division of Hemato/oncology, Department of Pediatrics, Faculty of medicine,Chulalongkorn U Bangkok Pathumwan Thailand 10330

    Sponsors and Collaborators

    • Chulalongkorn University

    Investigators

    • Principal Investigator: Darintr Sosothikul, MD, Chulalongkorn University

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Darintr Sosothikul, Associate professor, Chulalongkorn University
    ClinicalTrials.gov Identifier:
    NCT02727647
    Other Study ID Numbers:
    • 3125056
    First Posted:
    Apr 4, 2016
    Last Update Posted:
    Apr 4, 2016
    Last Verified:
    Oct 1, 2015
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Keywords provided by Darintr Sosothikul, Associate professor, Chulalongkorn University
    Moderate hemophilia A
    Severe hemophilia A
    Intermediate dose prophylaxis
    Low dose prophylaxis
    Additional relevant MeSH terms:
    Hemophilia A
    Factor VIII

    Study Results

    No Results Posted as of Apr 4, 2016