An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families

Hemophilia A With Inhibitor Clinical Trial

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Official title:

An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04723693
• Other study ID #: v4 4Nov2019
• Status: Completed
• Start date: February 28, 2020
• Est. completion date: January 31, 2021

Study information

• Verified date: January 2021
• Source: Haemnet
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This study aims to examine the real-life experience and impact of using emicizumab in a cohort of patients with haemophilia and inhibitors, who were prescribed emicizumab as part of the early access to medicine schema (EAMS),those who have been in clinical trials and those now receiving emicizumab as part of routine haemophilia care.

The Investigators also intend to capture the impact of emicizumab use on the lives of close family members (parents/carers/children/partners/siblings). Each participant and his family members will be deemed a study 'dyad'.

This is a prospective, observational cohort qualitative research study to be conducted among patients using emicizumab in routine clinical practice.

The study is designed to allow English-speaking patients and their families to tell their own life stories through narrative accounts. The narratives represent a true sharing of experiences and therefore offers insight into how these patients and families cope with haemophilia.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: January 31, 2021
• Est. primary completion date: January 31, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 8 Years to 100 Years

Eligibility

Inclusion Criteria:

• Males

• A diagnosis of Haemophilia A with inhibitors

• Using emicizumab (prescribed by treating clinicians) in usual clinical care.

Exclusion Criteria:

• No history of a Factor VIII inhibitor

• Not currently being treated with emicizumab,

• Does not speak English (for the interviews)

• Does not consent to take part.

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia A With Inhibitor

Intervention

Other:
• Qualitative Interview
A single one hour semi structured qualitative interview

Locations

Country	Name	City	State
United Kingdom	Oxford University Hospitals NHS Foundation Trust	Oxford	Oxfordshire

Sponsors (2)

Lead Sponsor	Collaborator
Haemnet	Roche Chugai

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Real life experience	To capture the individual participant's and his family's experience of using emicizumab for haemophilia inhibitor therapy.	Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & analysed using a grounded theory approach.
Secondary	Treatment satisfaction	To describe patient satisfaction with injections including frequency and numbers of injections, how to remember treatment dates, treatment comfort, bleed rate post switching.	Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & analysed using a grounded theory approach.
Secondary	Treatment expectations	To understand user's expectations of emicizumab and how they see future haemophilia care (less frequent injections, impact on home storage, number of bleeds, reduced hospitalisations etc).	Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & analysed using a grounded theory approach.
Secondary	Treatment impact	To describe the impact of the change in treatment on the extended family	Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & analysed using a grounded theory approach.