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

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ClinicalTrials.gov Identifier: NCT02727647
Recruitment Status : Completed
First Posted : April 4, 2016
Last Update Posted : April 4, 2016
Sponsor:
Information provided by (Responsible Party):
Darintr Sosothikul, Chulalongkorn University

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:

    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.


Condition or disease Intervention/treatment Phase
Hemophilia A Drug: FVIII Phase 4

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Study Type : Interventional  (Clinical Trial)
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 2015
Actual Primary Completion Date : March 2016
Actual Study Completion Date : March 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

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 Name: 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 Name: Hemofil-M




Primary Outcome Measures :
  1. Annualized bleeding rate per year [ Time Frame: 10 months ]

Secondary Outcome Measures :
  1. Hemophilia joint health score [ Time Frame: 10 months ]
  2. Amount of FVIII use [ Time Frame: 10 months ]
  3. Quality of life score [ Time Frame: 10 months ]
    EQ-5D-5L (Thailand version)

  4. Number of hospital stays [ Time Frame: 10 months ]
  5. Number of school days loss [ Time Frame: 10 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02727647


Locations
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Thailand
Division of Hemato/oncology, Department of Pediatrics, Faculty of medicine,Chulalongkorn U
Bangkok, Pathumwan, Thailand, 10330
Sponsors and Collaborators
Chulalongkorn University
Investigators
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Principal Investigator: Darintr Sosothikul, MD Chulalongkorn University

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Responsible Party: Darintr Sosothikul, Associate professor, Chulalongkorn University
ClinicalTrials.gov Identifier: NCT02727647     History of Changes
Other Study ID Numbers: 3125056
First Posted: April 4, 2016    Key Record Dates
Last Update Posted: April 4, 2016
Last Verified: October 2015
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Darintr Sosothikul, Chulalongkorn University:
Moderate hemophilia A
Severe hemophilia A
Intermediate dose prophylaxis
Low dose prophylaxis
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants