HPF burden of illness Observational patient diary study
Research type
Research Study
Full title
Hereditary Periodic Fevers (HPF) Burden of Illness Observational Patient Diary Study (HPF Burden of Illness Study - HEROES)
IRAS ID
198707
Contact name
Helen Jane Lachmann
Contact email
Sponsor organisation
Novartis Pharma AG
Duration of Study in the UK
0 years, 11 months, 3 days
Research summary
This is a multi-national study with adult and teenage patients and caregivers of children affected by hereditary periodic fever syndromes (HPF), specifically: TNF-receptor Associated Periodic Syndrome (TRAPS), Hyper IgD Syndrome (HIDS) and Familial Mediterranean Fever (FMF). HPF are characterised by periodic flares of disease activity, which leads to highly variable quality of life during and outside these flares. Moreover, HPF treatment can impose a high burden on patients and their families. However, due to the rarity of these conditions these elements have not received sufficient attention and analysis to date.
Navigant are conducting this study on behalf of the pharmaceutical company Novartis in order to understand the impact (physical, psychological and financial) of HIDS, TRAPS and FMF on patients and their wider family.
Approximately 110 patients or their caregivers will participate in the study from the three conditions and three patient age cohorts: children (2-12 years), adolescents (13-17 years) and adults (18+ years). Participants will be identified by recruiting clinicians in the U.K., France, Germany, the U.S., Israel and Turkey. Disease burden will be captured through completion of an anonymized electronic diary (e-diary), during and outside of a disease flare. The e-diary includes questions about the patient and their family in order to understand and measure the broad impact of FMF, HIDS or TRAPS.
The recruiting clinician will complete a two-page medical history of the HPF patient.
Study findings will help healthcare providers understand in more detail how symptoms change during a flare and design better treatment strategies for patients with HPF.REC name
East of England - Cambridge South Research Ethics Committee
REC reference
16/EE/0033
Date of REC Opinion
7 Mar 2016
REC opinion
Further Information Favourable Opinion