Interviews to Elicit the Burden of PNH & Treatment with Eculizumab.v1
Research type
Research Study
Full title
Interviews to Elicit the Burden of Paroxysmal Nocturnal Haemoglobinuria (PNH) and Treatment with Eculizumab in Patients and Caregivers. Version 1.0.0
IRAS ID
255697
Contact name
Ekaterina Anokhina
Contact email
Sponsor organisation
Alexion Pharma UK
Duration of Study in the UK
0 years, 3 months, 31 days
Research summary
A non-interventional qualitative research study to gain an in-depth understanding of how living with Paroxysmal Nocturnal Haemoglobinuria (PNH) and receiving regular infusions of Soliris (eculizumab) impacts UK patients and caregivers quality of life, daily activities and general wellbeing.
Up to 20 UK adult patients with PNH currently treated with eculizumab and up to 10 of their primary caregivers will undergo an in-depth semi-structured 1-hour interview by telephone. Patients will include adults living in the UK who have a confirmed diagnosis of PNH and are currently receiving regular infusions of eculizumab and have been treated with eculizumab for six months or more. Caregivers include adults who live in the UK and are the primary provider of support and assistance to a patient. Alexion Pharma UK will be funding this research and will be working in collaboration with a UK based patient organisation for PNH (PNH Support) and a health economic and outcomes research agency-BresMed Health Solutions. The study will be recruiting in the UK.
Following the availability of eculizumab as a treatment option for patients with PNH, the burden of living with this ultra-rare disease has been transformed, however the burden of disease or burden of treatment with eculizumab from the perspective of patients and caregivers has not previously been described in the UK. Alexion is currently developing a new therapy for the treatment of PNH which is expected to be indicated for the same population as eculizumab. We hope that exploring how PNH and treatment with eculizumab impacts UK patients and caregivers in terms of quality of life, daily activities and general wellbeing will help to better understand what it is like to live with this ultra-rare disease when receiving currently available treatment regimens.REC name
South West - Cornwall & Plymouth Research Ethics Committee
REC reference
18/SW/0286
Date of REC Opinion
28 Jan 2019
REC opinion
Further Information Favourable Opinion