Back to resultsOutcomes of Prophylaxis With Emicizumab in Children With Severe Hemophilia A in Ivory Coast
Primary Purpose
Hemophilia A
Status
Completed
Phase
Not Applicable
Locations
Côte D'Ivoire
Study Type
Interventional
Intervention
Prophylaxis with Emicizumab
Sponsored by
About this trial
This is an interventional treatment trial for Hemophilia A
Eligibility Criteria
Inclusion Criteria: severe hemophilia A with or without inhibitors -
Exclusion Criteria: parents refusal
-
Sites / Locations
- CHU de Yopougon
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Ivoirian Boys with severe Hemophilia A treated with Emicizumab
Arm Description
All Ivoirian boys with severe Hemophilia A (with and without inhibitors) on prophylaxis with Emicizumab
Outcomes
Primary Outcome Measures
Change in annual bleeding rate
Using the patients' logbook
Change in the scores of HR-QoL tools CHO-KLAT and EQ-5D in children treated with Emicizumab
Using the CHO-KLAT and EQ-5D. The scales range from 0 to 100 with 0 representing the worst quality of life and 100 the best quality of life score
Satisfaction of the parents about the treatment with Emicizumab after 6 month
Usign the PGIC (scale ranging from extremely satisfied to extremely unsatisfied)
Secondary Outcome Measures
Full Information
NCT ID
NCT05279924
First Posted
February 25, 2022
Last Updated
October 24, 2022
Sponsor
Cliniques universitaires Saint-Luc- Université Catholique de Louvain
1. Study Identification
Unique Protocol Identification Number
NCT05279924
Brief Title
Outcomes of Prophylaxis With Emicizumab in Children With Severe Hemophilia A in Ivory Coast
Official Title
Impact of Implementation of Prophylaxis With Emicizumab in Children With Severe Hemophilia A in Ivory Coast
Study Type
Interventional
2. Study Status
Record Verification Date
October 2022
Overall Recruitment Status
Completed
Study Start Date
July 1, 2021 (Actual)
Primary Completion Date
October 20, 2022 (Actual)
Study Completion Date
October 20, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Cliniques universitaires Saint-Luc- Université Catholique de Louvain
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Assessment of the outcomes of prophylaxis with Emicizumab in children with severe hemophilia A in Ivory Coast
Detailed Description
Assessment of the outcomes of prophylaxis with Emicizumab in children with severe hemophilia A in Ivory Coast.
Duration of the study: 1 year after initiation of prophylaxis with Emicizumab
Inclusion criteria: boys aged > 2 years, affected with severe hemophilia A with or without inhibitors
Evaluated outcomes: annual bleeding rate, days of absence from school, the need for additional facteur VIII consumption, satisfaction about the treatment and quality of life.
Tools used to assess quality of life are the Ivoirian version of the Canadian Haemophilia Outcomes-Kids' Life Assessment Tool version 2 (CHO-KLAT) and EQ-5D
The satisfaction of the parents will be assessed using the patients' global impression of change scale (PGIC).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Model Description
outcomes of prophylaxis with Emicizumab in Ivoirian boys with severe hemophilia A
Masking
None (Open Label)
Allocation
N/A
Enrollment
33 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Ivoirian Boys with severe Hemophilia A treated with Emicizumab
Arm Type
Experimental
Arm Description
All Ivoirian boys with severe Hemophilia A (with and without inhibitors) on prophylaxis with Emicizumab
Intervention Type
Drug
Intervention Name(s)
Prophylaxis with Emicizumab
Intervention Description
Prophylaxis with Emicizumab in Ivoirian boys with severe hemophilia A
Primary Outcome Measure Information:
Title
Change in annual bleeding rate
Description
Using the patients' logbook
Time Frame
change in annual bleeding rate from baseline at 6 months and 12 months
Title
Change in the scores of HR-QoL tools CHO-KLAT and EQ-5D in children treated with Emicizumab
Description
Using the CHO-KLAT and EQ-5D. The scales range from 0 to 100 with 0 representing the worst quality of life and 100 the best quality of life score
Time Frame
Change in the scores of HR-QoL tools CHO-KLAT and EQ-5D at 6 months.
Title
Satisfaction of the parents about the treatment with Emicizumab after 6 month
Description
Usign the PGIC (scale ranging from extremely satisfied to extremely unsatisfied)
Time Frame
Assessment at 6 months after initiation of Emicizumab
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: severe hemophilia A with or without inhibitors -
Exclusion Criteria: parents refusal
-
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
N'Dogomo Meité, MD
Organizational Affiliation
CHU de Cocody
Official's Role
Principal Investigator
Facility Information:
Facility Name
CHU de Yopougon
City
Abidjan
Country
Côte D'Ivoire
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Outcomes of Prophylaxis With Emicizumab in Children With Severe Hemophilia A in Ivory Coast
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