D58.8
Conditions
Interventions
Group 1: Evaluation of the treatment effects of ravulizumab on emotional wellbeing as part of quality of life (QoL), depressiveness and TMA resolution, as well as the impact of aHUS diagnosis on depre
Sponsors
Alexion Pharma Germany GmbH
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Adult patients (18 years and above) with a diagnosis of aHUS (including those with a triggering event) 2. Scheduled to start treatment with ravulizumab according to routine practice and as per SmPC, either as the initial treatment (initiation cohort) or after switch from eculizumab to ravulizumab after at least 3 months of treatment with eculizumab (switch cohort)
Exclusion criteria
Exclusion criteria: 1. Treatment with other complement inhibitors than eculizumab 2. Participation in an interventional trial (with an investigational medicinal product) in parallel or within the last 3 months before the inclusion to this study. 3. Any contraindication as per SmPC of ravulizumab.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| •SF-36 questionnaire physical and mental component scores at baseline and week 26 (or last available measurement) and change from baseline to week 26 (or last available measurement) •PHQ-9 questionnaire score at baseline and week 26 (or last available measurement) and change from baseline to week 26 (or last available measurement) •One Question QoL score at baseline, week 26 (or last available measurement) and change from baseline to week 26 (or last available measurement) •Change of general state of health from baseline to week 26 (or last available measurement) | — |
Secondary
| Measure | Time frame |
|---|---|
| • Cut-off points for SF-36 and PHQ-9 corresponding to patient acceptable symptom state (PASS). • PASS at baseline, week 26 (or last available measurement) and change from baseline to week 26 (or last available measurement). • TMA response by week 26 (or last available measurement), defined as o Thrombocytes normalization (= 150×109/l) and o LDH normalization (LDH = ULN) and o = 25% reduction of serum creatinine (SCr)) at one blood test during the study (initiation cohort only). • Change of components of TMA response from baseline to week 26 (or last available measurement). • Change of Hb from baseline to week 26 (or last available measurement). • Change of schistocytes from baseline to week 26 (or last available measurement). • Change of haptoglobin from baseline to week 26 (or last available measurement). • Change of proteinuria from baseline to week 26 (or last available measurement). • Presence of TMA symptoms, incl. extrarenal manifestations (yes/no), at baseline, week 1, week 2, week 10, week 18 and week 26 (or last available measurement). • Type and incidence of serious adverse events (SAEs) and adverse drug reactions (ADRs). | — |
Countries
Germany
Contacts
Public ContactAnja Gäckler
Universitätsklinikum Essen Klinik für Nephrologie
Outcome results
None listed