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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

30 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-02-28

Study Completion Date

2021-01-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors

NCT03154437

Hemophilia A

APPROVED_FOR_MARKETING

A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors

NCT04158648

Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor Hemophilia A

COMPLETED PHASE3

A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors

NCT02622321

Hemophilia A

COMPLETED PHASE3

A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors

NCT03191799

Hemophilia A

COMPLETED PHASE3

Emicizumab in Acquired Hemophilia A

NCT04188639

Hemophilia A, Acquired

COMPLETED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Emicizumab offers patients protection from bleeding with fewer injections, this should reduce treatment burden. While patient expectation is high (as evidenced by the conversations on UK haemophilia social media sites) there are risks that patients may forget to treat and thus experience bleeds. During clinical trials participants have been monitored closely and, through questionnaire completion, have described improvement in quality of life \[Mancuso et al, 2018, Oldenburg et al 2019\].

What is also apparent to clinical teams is the dramatic change to life experience of not just the patient but his family - the ability to travel, to plan attending events knowing that bleeds will not occur, even potentially to have another child because of the reduction in treatment burden, particularly the time needed for treatment and rehabilitation.

This therapy represents a substantial shift in the entire life experience of living with and managing haemophilia with inhibitors. As such, there is a need to look beyond the quantitative data collected in clinical trials and to assess the real impact of therapy on the everyday lives of patients and their families, gathered using qualitative research techniques.

Emicizumab has been available in the UK in clinical trials and as part of an Early Access to Medicines Scheme (EAMS) for non-trial eligible patients since spring 2018. It was recently licensed in the UK and is now available for routine clinical care for any person with haemophilia A and an inhibitor.

We hypothesise that patients and their carers/families will be excited about this new treatment option and that for most, if not all, the promise of improved care and quality of life will be a reality.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hemophilia A With Inhibitor

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

FAMILY_BASED

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

individuals With Haemophilia A and with inhibitors on emicizumab

Qualitative interviews

Qualitative Interview

Intervention Type OTHER

A single one hour semi structured qualitative interview

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Qualitative Interview

A single one hour semi structured qualitative interview

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.

Minimum Eligible Age

8 Years

Maximum Eligible Age

100 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No