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

NCT04723693 | Completed Results

• 1 other identifier: v4 4Nov2019
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Hemophilia A With Inhibitor

Keywords

Haemophilia, Quality of Life

Outcome Measures

Primary Outcomes (1)

• Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities)

  To capture the individual participant's and his family's experience of using emicizumab for haemophilia inhibitor therapy and see if there is any reduction in the burden of the condition.

  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 & thematically analysed and reported.

Secondary Outcomes (3)

• Treatment Satisfaction (Do the Participants and Their Family Feel That the Change in Treatment Improved Their Condition Control and in What Ways)

  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 & thematically analysed and reported.

• Treatment Expectations

  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 & thematically analysed and reported.

• Treatment Impact

  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 & thematically analysed and reported.

Study Arms (1)

individuals With Haemophilia A and with inhibitors on emicizumab

Qualitative interviews

Other: Qualitative Interview

Interventions

Qualitative Interview OTHER

A single one hour semi structured qualitative interview

individuals With Haemophilia A and with inhibitors on emicizumab

Eligibility Criteria

• Age: 8 Years - 100 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Male participants with Hameophilia A and inhibitors currently on emicizumab prophylaxis and a member of their family

You may qualify if:

• Males
• A diagnosis of Haemophilia A with inhibitors
• Using emicizumab (prescribed by treating clinicians) in usual clinical care.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Haemnet: lead
• Roche Chugai: collaborator

Study Sites (1)

Oxford University Hospitals NHS Foundation Trust

Oxford, Oxfordshire, OX3 7LE, United Kingdom

Location

Related Publications (1)

• Fletcher S, Jenner K, Holland M, Khair K. The lived experience of a novel disruptive therapy in a group of men and boys with haemophilia A with inhibitors: Emi & Me. Health Expect. 2022 Feb;25(1):443-454. doi: 10.1111/hex.13404. Epub 2021 Dec 8.

  PMID: 34878209 BACKGROUND

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Simon Fletcher
• Organization: Oxford university Hospitals NHS Foundation Trust

simon.fletcher@ouh.nhs.uk

01865 225316

Study Officials

• Simon P Fletcher, MA

  Researcher

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: FAMILY BASED
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 14, 2021
• First Posted: January 26, 2021
• Study Start: February 28, 2020
• Primary Completion: January 31, 2021
• Study Completion: January 31, 2021
• Last Updated: August 19, 2024
• Results First Posted: August 19, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)