Outcomes of Prophylaxis With Emicizumab in Children With Severe Hemophilia A in Ivory Coast

Sponsor

Cliniques universitaires Saint-Luc- Université Catholique de Louvain (Other)

Overall Status

Recruiting

CT.gov ID

NCT05279924

Collaborator

(none)

Enrollment

Location

Arm

Anticipated Duration (Months)

2.8

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Assessment of the outcomes of prophylaxis with Emicizumab in children with severe hemophilia A in Ivory Coast

Condition or Disease	Intervention/Treatment	Phase
Hemophilia A	Drug: Prophylaxis with Emicizumab	N/A

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).

Study Design

Study Type:

Interventional

Anticipated Enrollment :

33 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Intervention Model Description:

outcomes of prophylaxis with Emicizumab in Ivoirian boys with severe hemophilia Aoutcomes of prophylaxis with Emicizumab in Ivoirian boys with severe hemophilia A

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Impact of Implementation of Prophylaxis With Emicizumab in Children With Severe Hemophilia A in Ivory Coast

Actual Study Start Date :

Jul 1, 2021

Anticipated Primary Completion Date :

Jul 1, 2022

Anticipated Study Completion Date :

Jul 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Ivoirian Boys with severe Hemophilia A treated with Emicizumab All Ivoirian boys with severe Hemophilia A (with and without inhibitors) on prophylaxis with Emicizumab	Drug: Prophylaxis with Emicizumab Prophylaxis with Emicizumab in Ivoirian boys with severe hemophilia A

Arm

Intervention/Treatment

Experimental: Ivoirian Boys with severe Hemophilia A treated with Emicizumab

All Ivoirian boys with severe Hemophilia A (with and without inhibitors) on prophylaxis with Emicizumab

Drug: Prophylaxis with Emicizumab

Prophylaxis with Emicizumab in Ivoirian boys with severe hemophilia A

Outcome Measures

Primary Outcome Measures

Change in annual bleeding rate [change in annual bleeding rate from baseline at 6 months and 12 months]
Using the patients' logbook
Change in the scores of HR-QoL tools CHO-KLAT and EQ-5D in children treated with Emicizumab [Change in the scores of HR-QoL tools CHO-KLAT and EQ-5D at 6 months.]
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 [Assessment at 6 months after initiation of Emicizumab]
Usign the PGIC (scale ranging from extremely satisfied to extremely unsatisfied)

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years to 18 Years

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

Inclusion Criteria: severe hemophilia A with or without inhibitors -

Exclusion Criteria: parents refusal

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	CHU de Yopougon	Abidjan		Côte D'Ivoire

Sponsors and Collaborators

Cliniques universitaires Saint-Luc- Université Catholique de Louvain

Investigators

Principal Investigator: N'Dogomo Meité, MD, CHU de Cocody

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Lambert Catherine, Clinical Professor. Hematology department, Cliniques universitaires Saint-Luc- Université Catholique de Louvain

ClinicalTrials.gov Identifier:

NCT05279924

Other Study ID Numbers:

001-22/MSHPCMU/CNESVS-km

First Posted:

Mar 15, 2022

Last Update Posted:

May 4, 2022

Last Verified:

Mar 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Hemophilia A

Study Results

No Results Posted as of May 4, 2022