Ravulizumab Outcomes in Polish Patients With aHUS
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Ravulizumab.
- Who it may be relevant to
- Registry conditions: Atypical Hemolytic Uremic Syndrome. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Poland
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Non-interventional Study Evaluating Ravulizumab Treatment Outcomes in Polish Patients With Atypical Hemolytic Uremic Syndrome
Overview
This multicenter, observational cohort study uses retrospective collection of past medical history and prospective follow-up to capture longitudinal data on the management and clinical outcomes of patients with atypical hemolytic uremic syndrome (aHUS) treated with ravulizumab as part of routine clinical practice under Poland's National Drug Program (NDP).
Interventions
- Drug Ravulizumab
Ultomiris
Primary outcome measures
- Proportion of patient attaining Complete Thrombotic Microangiopathy (TMA) Response during observation (naïve) [Time frame: Up to 24 months]
- Proportion of patients attaining/maintaining. Complete TMA Response during observation (switched) [Time frame: Up to 24 months]
Secondary outcome measures (8)
- Time to Complete TMA Response [Time frame: Up to 24 months]
- Proportion of dialysis-free patients [Time frame: Up to 24 months]
- Complete TMA response [Time frame: Up to 24 months]
- Proportion of patients with lab results normalization during observation [Time frame: Up to 24 months]
- Change from baseline in CKD stage, as evaluated by the physician over time [Time frame: Up to 24 months]
- Change from baseline in proteinuria status over time [Time frame: Up to 24 months]
- Change from baseline in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-Fatigue; adults) and Pediatric Functional Assessment of Chronic Illness Therapy - Fatigue (Peds FACIT-F; pediatric patients) score overtime (naïve) [Time frame: Up to 24 months]
- Change form baseline in EQ- 5D-5L (adults) and EQ-5D-Y- 5L (paediatric patients) score overtime (naïve) [Time frame: Up to 24 months]
Eligibility criteria
Inclusion criteria
- Patients of all ages diagnosed with atypical hemolytic uremic syndrome (aHUS) who received treatment with ravulizumab under the National Drug Program (NDP) in Poland.
- Patients who are willing to participate in the study and have provided informed consent by signing the informed consent form (ICF).
Exclusion criteria
- Individuals who intend to participate in a clinical trial for atypical hemolytic uremic syndrome (aHUS) on or after the date of their first ravulizumab infusion through the National Drug Program.
- Patients with cognitive impairments, those who are unwilling to participate, or those facing language barriers that hinder adequate comprehension or cooperation.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Poland · 10 centers
- Research Site — Gdansk
- Research Site — Katowice
- Research Site — Katowice
- Research Site — Krakow
- Research Site — Lodz
- Research Site — Poznan
- Research Site — Poznan
- Research Site — Warsaw
- … and 2 more centers
Identifiers
NCT: NCT07399730 · D928BR00003