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

April 1, 2016 updated by: Darintr Sosothikul, Chulalongkorn University

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:

    1. History, interesting clinical data and laboratory data will be recorded in Clinical record Form (CRF)
    2. Measurement:

    i. 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

  3. 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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

16

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Thailand
    • Pathumwan
      • Bangkok, Pathumwan, Thailand, 10330
        • Division of Hemato/oncology, Department of Pediatrics, Faculty of medicine,Chulalongkorn U

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 20 years (Child, Adult)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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
Drug: FVIII
FVIII concentration 15-20 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 35-40 U/kg/dose 1 time/week for 5 months
Other Names:
  • Hemofil-M
Drug: FVIII
FVIII concentration 15-20 U/kg/dose 1 time/week for 5 months
Other Names:
  • Hemofil-M

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Annualized bleeding rate per year
Time Frame: 10 months
10 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Hemophilia joint health score
Time Frame: 10 months
10 months
Amount of FVIII use
Time Frame: 10 months
10 months
Quality of life score
Time Frame: 10 months
EQ-5D-5L (Thailand version)
10 months
Number of hospital stays
Time Frame: 10 months
10 months
Number of school days loss
Time Frame: 10 months
10 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Chulalongkorn University

Investigators

  • Principal Investigator: Darintr Sosothikul, MD, Chulalongkorn University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2015

Primary Completion (Actual)

March 1, 2016

Study Completion (Actual)

March 1, 2016

Study Registration Dates

First Submitted

March 30, 2016

First Submitted That Met QC Criteria

April 1, 2016

First Posted (Estimate)

April 4, 2016

Study Record Updates

Last Update Posted (Estimate)

April 4, 2016

Last Update Submitted That Met QC Criteria

April 1, 2016

Last Verified

October 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 3125056

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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