Ravulizumab Outcomes in Polish Patients With aHUS (aHUS-OPTIMUM)

A Non-interventional Study Evaluating Ravulizumab Treatment Outcomes in Polish Patients With Atypical Hemolytic Uremic Syndrome

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 Overview

• Status: Not yet recruiting
• Conditions: Atypical Hemolytic Uremic Syndrome
• Intervention / Treatment: Drug: Ravulizumab

• Study Type: Observational
• Enrollment (Estimated): 80

Contacts and Locations

• Study Contact: Name: AstraZeneca Clinical Study Information Center Study Information Center; Phone Number: +118772409479; Email: information.center@astrazeneca.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Consecutive patients naïve to complement inhibitors (prospective cohort) with a body weight of 10 kg or above (no age restrictions) diagnosed with atypical haemolytic uremic syndrome and meeting all ravulizumab treatment eligibility criteria listed in NDP - no previous exposition to CIs including clinical trials, EAP etc.

Patients who have been switched to ravulizumab from other CI (retrospective cohort) with a body weight of 10 kg or above (no age restrictions) diagnosed with atypical haemolytic uremic syndrome and meeting all ravulizumab treatment eligibility criteria listed in NDP.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Prospective cohort; Group of patients naive to complement inhibitors	Drug: Ravulizumab; Ultomiris
Retrospective cohort; Group of patients who transitioned from other complement inhibitors to ravulizumab.	Drug: Ravulizumab; Ultomiris

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	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	Up to 24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to Complete TMA Response	Time from initiation of ravulizumab to TMA Response: continuous variable (days; specify when complete response criteria are met)	Up to 24 months
Proportion of dialysis-free patients	In order to achieve the secondary objectives, the following variables will be estimated	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),	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	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	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	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.	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)	Up to 24 months

Collaborators and Investigators

• Sponsor: AstraZeneca

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2026
• Primary Completion (Estimated): December 31, 2030
• Study Completion (Estimated): December 31, 2030

Study Registration Dates

• First Submitted: January 27, 2026
• First Submitted That Met QC Criteria: February 3, 2026
• First Posted (Actual): February 10, 2026

Study Record Updates

• Last Update Posted (Actual): February 10, 2026
• Last Update Submitted That Met QC Criteria: February 3, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: Observational; Atypical Hemolytic Uremic Syndrome; Ravulizumab; aHUS
• Additional Relevant MeSH Terms: Urogenital Diseases; Cytopenia; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Hematologic Diseases; Anemia, Hemolytic; Anemia; Blood Platelet Disorders; Thrombotic Microangiopathies; Thrombocytopenia; Uremia; Hemic and Lymphatic Diseases; Hemolytic-Uremic Syndrome; Atypical Hemolytic Uremic Syndrome; ravulizumab
• Other Study ID Numbers: D928BR00003

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

• IPD Sharing Time Frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
• IPD Sharing Access Criteria: When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No