NCT02727647

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

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Chulalongkorn University · Academic / Other
• Sex: All
• Age: 6 Months – 20 Years
• Healthy volunteers: Accepted

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.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
DRUG	FVIII	FVIII concentration 35-40 U/kg/dose 1 time/week for 5 months
DRUG	FVIII	FVIII concentration 15-20 U/kg/dose 1 time/week for 5 months

Timeline

• Start date: 2015-05-01
• Primary completion: 2016-03-01
• Completion: 2016-03-01
• First posted: 2016-04-04
• Last updated: 2016-04-04

Locations

1 site across 1 country: Thailand