Ravulizumab Outcomes in Polish Patients With aHUS

N/ANot Yet RecruitingNCT07399730

AstraZeneca80 enrolled

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).

Study Type

OBSERVATIONAL

Enrollment

Conditions

Atypical Hemolytic Uremic Syndrome

Interventions

RavulizumabDRUG

Ultomiris

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Proportion of patient attaining Complete Thrombotic Microangiopathy (TMA) Response during observation (naïve)

In order to achieve the primary objectives, the following variables will be estimated: To assess ravulizumab primary treatment outcome in Polish patients with aHUS

Time frame: Up to 24 months

Proportion of patients attaining/maintaining. Complete TMA Response during observation (switched)

In order to achieve the primary objectives, the following variables will be estimated: To assess ravulizumab primary treatment outcome in Polish patients with aHUS

Time frame: Up to 24 months

Secondary Outcomes

Time to Complete TMA Response

Time from initiation of ravulizumab to TMA Response: continuous variable (days; specify when complete response criteria are met)

Time frame: Up to 24 months

Proportion of dialysis-free patients

In order to achieve the secondary objectives, the following variables will be estimated

Time frame: Up to 24 months

Complete TMA response

In order to achieve the secondary objectives, the following variables will be estimated: * Platelet count (≥150 x 109/L) * Lactate dehydrogenase (LDH) levels (≤ULN) * Serum creatinine (≤ULN for age or an improvement \> 25% compared to baseline),

Time frame: Up to 24 months

Proportion of patients with lab results normalization during observation

Laboratory Parameters: * platelet count (≥150 x 109/L)\*, * lactate dehydrogenase (LDH) levels (≤ULN), * serum creatinine (≤ULN for age or an improvement \> 25% compared to baseline), * serum creatinine improvement of \>50% compared to baseline * estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73 m2, * increase in haemoglobin of ≥ 20 g/L

Central Contacts

AstraZeneca Clinical Study Information Center Study Information Center

CONTACT

+118772409479 information.center@astrazeneca.com

Data from ClinicalTrials.gov

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Time frame: Up to 24 months

Change from baseline in CKD stage, as evaluated by the physician over time

In order to achieve the secondary objectives, the following variables will be estimated: CKD stage 1 (rather theoretical at baseline, possible following successful treatment): eGFR ≥90 ml/min./1.73m2 CKD stage 2 (rather theoretical at baseline, possible following successful treatment): eGFR 60 - 90 ml/min./1.73m2 CKD stage 3a: eGFR 45 - 59 ml/min./1.73m2 CKD stage 3b: eGFR 30 - 44 ml/min./1.73m2 CKD stage 4: eGFR 15 - 29 ml/min./1.73m2 CKD stage 5: eGFR\< 15 ml/min./1.73m2 CKD stage 5D: need for dialysis independent from eGFR value

Time frame: Up to 24 months

Change from baseline in proteinuria status over time

In order to achieve the secondary objectives, the following variables will be estimated: At least one of the following numbers describing the highest proteinuria and/or albuminuria: * urine protein-to-creatinine ratio \[mg/g\] (preferred) * urine protein loss \[mg/24 hours\] * urine protein concentration \[mg/dl\] * urine albumin-to-creatinine ratio \[mg/g\] (preferred) * urine albumin loss \[mg/24 hours\] Urine protein reduction to ≤ 500 mg/g (500 mg/24 hours) Urine protein reduction by ≥50% from baseline Time to urine protein reduction to ≤ 500 mg/g (500 mg/24 hours) Time to urine protein reduction by ≥50% from baseline

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)

FACIT-Fatigue - scoring ranges from 0 (highest fatigue) to 52 (lowest fatigue), where higher scores indicate lower levels of fatigue. Peds FACIT-F - scoring ranges from 0 (highest fatigue) to 52 (lowest fatigue), where higher scores indicate lower levels of fatigue.

Time frame: Up to 24 months

Change form baseline in EQ- 5D-5L (adults) and EQ-5D-Y- 5L (paediatric patients) score overtime (naïve)

EQ-5D-5L/EQ-5D-Y-5L consists of 2 pages: EQ-5D descriptive system and EQ visual analogue scale (EQ VAS). EQ-5D/EQ-5D-Y comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems, and extreme problems. EQ VAS - scale rating from 0 (worst imaginable health state) to 100 (best imaginable health state)

Time frame: Up to 24 months