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

Primary Purpose

Hemophilia A

Status

Completed

Phase

Phase 4

Locations

Thailand

Study Type

Interventional

Intervention

FVIII

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring Moderate hemophilia A, Severe hemophilia A, Intermediate dose prophylaxis, Low dose prophylaxis

Eligibility Criteria

6 Months - 20 Years (Child, Adult)All SexesAccepts Healthy Volunteers

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

Sites / Locations

Division of Hemato/oncology, Department of Pediatrics, Faculty of medicine,Chulalongkorn U

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Arm 1

Arm 2

Arm Description

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

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

Outcomes

Primary Outcome Measures

Annualized bleeding rate per year

Secondary Outcome Measures

Hemophilia joint health score

Amount of FVIII use

Quality of life score

EQ-5D-5L (Thailand version)

Number of hospital stays

Number of school days loss

Full Information

NCT ID

NCT02727647

First Posted

March 30, 2016

Last Updated

April 1, 2016

Sponsor

Chulalongkorn University

1. Study Identification

Unique Protocol Identification Number

NCT02727647

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

October 2015

Overall Recruitment Status

Completed

Study Start Date

May 2015 (undefined)

Primary Completion Date

March 2016 (Actual)

Study Completion Date

March 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Chulalongkorn University

4. Oversight

Data Monitoring Committee

5. 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: 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. Observation and measurement: History, interesting clinical data and laboratory data will be recorded in Clinical record Form (CRF) 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 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hemophilia A

Keywords

Moderate hemophilia A, Severe hemophilia A, Intermediate dose prophylaxis, Low dose prophylaxis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

16 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Arm 1

Arm Type

Experimental

Arm Description

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

Arm Title

Arm 2

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

FVIII

Other Intervention Name(s)

Hemofil-M

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

FVIII

Other Intervention Name(s)

Hemofil-M

Intervention Description

FVIII concentration 15-20 U/kg/dose 1 time/week for 5 months

Primary Outcome Measure Information:

Title

Annualized bleeding rate per year

Time Frame

10 months

Secondary Outcome Measure Information:

Title

Hemophilia joint health score

Time Frame

10 months

Title

Amount of FVIII use

Time Frame

10 months

Title

Quality of life score

Description

EQ-5D-5L (Thailand version)

Time Frame

10 months

Title

Number of hospital stays

Time Frame

10 months

Title

Number of school days loss

Time Frame

10 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Months

Maximum Age & Unit of Time

20 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Darintr Sosothikul, MD

Organizational Affiliation

Chulalongkorn University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Division of Hemato/oncology, Department of Pediatrics, Faculty of medicine,Chulalongkorn U

City

Bangkok

State/Province

Pathumwan

ZIP/Postal Code

10330

Country

Thailand

12. IPD Sharing Statement

Plan to Share IPD

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

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