Ravulizumab Outcomes in Polish Patients With aHUS
ConditionAtypical Hemolytic Uremic Syndrome
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorAstraZeneca
About this trial
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).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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).
Disqualifiers
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.
Trial 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.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Ravulizumab
DrugUltomiris
Treatment groups
80 Participants
are divided into 2 treatment groupsGroup A: Prospective cohort1 intervention
Group B: Retrospective cohort1 intervention
Trial outcomes
Primary outcomes
1
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 months2
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 monthsSecondary outcomes
1
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 months2
Proportion of dialysis-free patients
In order to achieve the secondary objectives, the following variables will be estimated
Time frame
Up to 24 months3
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 months4
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
Time frame
Up to 24 monthsSponsors and contacts
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Source ClinicalTrials.gov