Ravulizumab Outcomes in Polish Patients With aHUS

NCT07399730 | Not Yet Recruiting FDA Drug

• 1 other identifier: D928BR00003
• Study Type: observational
• Target: 80
• Locations: 0 countries
• Sites: N/A

Brief Summary

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).

Conditions

Atypical Hemolytic Uremic Syndrome

Keywords

aHUS; Atypical Hemolytic Uremic Syndrome; Ravulizumab; Observational

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (8)

• Time to Complete TMA Response

  Up to 24 months

• Proportion of dialysis-free patients

  Up to 24 months

• Complete TMA response

  Up to 24 months

• Proportion of patients with lab results normalization during observation

  Up to 24 months

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

  Up to 24 months

Study Arms (2)

Prospective cohort

Group of patients naive to complement inhibitors

Drug: Ravulizumab

Retrospective cohort

Group of patients who transitioned from other complement inhibitors to ravulizumab.

Drug: Ravulizumab

Interventions

Ravulizumab DRUG

Ultomiris

Prospective cohort; Retrospective cohort

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• AstraZeneca: lead

MeSH Terms

Conditions

Atypical Hemolytic Uremic Syndrome

Interventions

ravulizumab

Condition Hierarchy (Ancestors)

Hemolytic-Uremic Syndrome; Uremia; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia

Central Study Contacts

AstraZeneca Clinical Study Information Center Study Information Center

CONTACT

+118772409479; information.center@astrazeneca.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 27, 2026
• First Posted: February 10, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): December 31, 2030
• Study Completion (Estimated): December 31, 2030
• Last Updated: February 10, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• 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.
• 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.