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

Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02727647
• Other study ID #: 3125056
• Status: Completed
• Phase: Phase 4
• First received: March 30, 2016
• Last updated: April 1, 2016
• Start date: May 2015
• Est. completion date: March 2016

Study information

• Verified date: October 2015
• Source: Chulalongkorn University
• Contact: n/a
• Is FDA regulated: No
• Health authority: Thailand: Institutional Review Board
• Study type: Interventional

Clinical Trial 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:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. completion date: March 2016
• Est. primary completion date: March 2016
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 6 Months to 20 Years

Eligibility

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 Design

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Drug:
• FVIII
FVIII concentration 35-40 U/kg/dose 1 time/week for 5 months
• FVIII
FVIII concentration 15-20 U/kg/dose 1 time/week for 5 months

Locations

Country	Name	City	State
Thailand	Division of Hemato/oncology, Department of Pediatrics, Faculty of medicine,Chulalongkorn U	Bangkok	Pathumwan

Sponsors (1)

Lead Sponsor	Collaborator
Chulalongkorn University

Country where clinical trial is conducted

Thailand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Annualized bleeding rate per year		10 months	No
Secondary	Hemophilia joint health score		10 months	No
Secondary	Amount of FVIII use		10 months	No
Secondary	Quality of life score	EQ-5D-5L (Thailand version)	10 months	No
Secondary	Number of hospital stays		10 months	No
Secondary	Number of school days loss		10 months	No