Observational, Prospective, Natural History Study in patients with PNH
Research type
Research Study
Full title
A Single-centre, Observational, Prospective, Natural History Study in Patients with Paroxysmal Nocturnal Haemoglobinuria.
IRAS ID
231321
Contact name
Peter Hillmen
Contact email
Sponsor organisation
Apellis Pharmaceuticals Inc
Clinicaltrials.gov Identifier
HM17/99874, Leeds Teaching Hospitals R&I number
Duration of Study in the UK
1 years, 0 months, 17 days
Research summary
This is an observational, prospective, natural history research study intended enhance the understanding the clinical course of the Paroxysmal nocturnal haemoglobinuria (PNH). PNH is a rare, life-threatening, acquired chronic disease characterised by haemolysis (rupture of red blood cells) and thrombophilia (increased tendency of blood to clot). The study is designed to collect and track clinical endpoints in patients with PNH by using the Revon digital platform. Revon is a software application that is used by the patients and treating physician to collect information about PNH. The aim of this study is to evaluate the Revon application for data collection and to further understand the disease progression in patients with PNH.
This sponsor of this study is Apellis Pharmaceuticals. This study will run in the UK and male and female PNH patients over 18 years old who meet the inclusion criteria will be eligible to take part. Approximately 48 patients with PNH will be enrolled in this study for 6 months and followed up for a further 6 months. The study is split into 2 phases. The first phase will enrol 8 patients into 2 groups to evaluate the Revon app for data collection. After 2 months, a committee with members from the Sponsor, Revon and the clinical team will evaluate the Revon app and based on their decision, the second phase of the study will commence. The second phase will enrol 40 patients across 4 groups. In both phases, patients will be allocated to the appropriate group based on their current treatment regimen. Patients activity will be recoded and they will be asked to record information about their health on the Revon app. The treating physician will record information gathered at routine standard of care visits on the Revon app. There will be no study mandated visits to the site.REC name
West Midlands - Black Country Research Ethics Committee
REC reference
17/WM/0302
Date of REC Opinion
23 Aug 2017
REC opinion
Further Information Favourable Opinion