NCT02727647

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started May 2015

Shorter than P25 for phase_4

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2015

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2016

Completed
29 days until next milestone

First Submitted

Initial submission to the registry

March 30, 2016

Completed
5 days until next milestone

First Posted

Study publicly available on registry

April 4, 2016

Completed
Last Updated

April 4, 2016

Status Verified

October 1, 2015

Enrollment Period

10 months

First QC Date

March 30, 2016

Last Update Submit

April 1, 2016

Conditions

Hemophilia A

Keywords

Moderate hemophilia ASevere hemophilia AIntermediate dose prophylaxisLow dose prophylaxis

Outcome Measures

Primary Outcomes (1)

  • Annualized bleeding rate per year

    10 months

Secondary Outcomes (5)

  • Hemophilia joint health score

    10 months

  • Amount of FVIII use

    10 months

  • Quality of life score

    10 months

  • Number of hospital stays

    10 months

  • Number of school days loss

    10 months

Study Arms (2)

Arm 1

EXPERIMENTAL

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

Drug: FVIII

Arm 2

EXPERIMENTAL

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

Drug: FVIII

Interventions

FVIIIDRUG

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

Also known as: Hemofil-M
Arm 1

Eligibility Criteria

Age6 Months - 20 Years
Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Hemophilia patients with moderate (factor level 1-3%) or severe (factor level \<1%) severity

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

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

Bangkok, Pathumwan, 10330, Thailand

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Study Officials

  • Darintr Sosothikul, MD

    Chulalongkorn University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Associate professor

Study Record Dates

First Submitted

March 30, 2016

First Posted

April 4, 2016

Study Start

May 1, 2015

Primary Completion

March 1, 2016

Study Completion

March 1, 2016

Last Updated

April 4, 2016

Record last verified: 2015-10

Data Sharing

IPD Sharing
Will not share

Locations

TH(1)