Potential Predictors of Disease Progression in aHUS Patients.
Research type
Research Study
Full title
Evaluation of Potential Predictors of Disease Progression in Patients with aHUS including Genetics, Biomarkers, and Treatment.
IRAS ID
205613
Contact name
Daniel Gale
Contact email
Sponsor organisation
ALEXION PHARMACEUTICALS, INC
Eudract number
2015-003135-35
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
NCT02614898, ClinicalTrials.gov; 2015-003135-35, EudraCT number
Duration of Study in the UK
3 years, 11 months, 8 days
Research summary
This is a research study looking at patients with Atypical Haemolytic Uraemic Syndrome (aHUS) who have previously participated in an Alexion- sponsored clinical trial for uHUS. aHUS is a serious life-threatening rare disorder that is seen as an anaemia caused by red blood cells bursting in small arteries (thrombocytic microangiopathy or TMA), low platelets, and ultimately kidney failure. These abnormalities are due to the presence of clots in the small blood vessels in the kidney. aHUS can recur and is a chronic disease and therefore long- term.
The purpose of this study is to assess TMA disease manifestations in patients with aHUS with or without ongoing eculizumab treatment in a real-world setting.
Additional objectives of this study are to determine potential clinical predictors of disease expression and progression, including clinical characteristics and genetic profiling to determine disease activity and relationship to treatment as shown by levels of biomarkers associated with aHUS complement activation, as well as the use of associated supportive interventions.REC name
London - Hampstead Research Ethics Committee
REC reference
16/LO/1436
Date of REC Opinion
25 Nov 2016
REC opinion
Further Information Favourable Opinion