An observational study in Atypical Hemolytic Uremic Syndrome and aHUS, sponsored by AO GENERIUM. Completed at 8 sites in Russian Federation. Open to participants aged 2 Months and older. Per ClinicalTrials.gov, last updated 2022-08-04.
Sponsored by AO GENERIUM · Observational
It is a multicenter observational non-comparative study of the efficacy and safety of long-term pathogenetic Elizaria® therapy in patients with atypical Hemolytic Uremic Syndrome
After screening, patients meeting all of the inclusion / non-inclusion criteria and vaccinated against meningococcal infections were treated by Elizaria®.
The study is planned to include at least 50 patients receiving Elizaria® for the aHUS treatment.
The study will consist of a screening period of up to 4 weeks, including, if necessary, immunization with meningococcal vaccine, a treatment period of 52 weeks.
Medication will be prescribed in accordance with routine medical practice. Accordingly to minimize the risks and subjectivity of assessments the methods adopted in the routine practice of treating patients with aHUS will be used.
Investigators enroll patients with aHUS diagnosis who have indications for pathogenetic therapy and who are receiving Elizaria® under the government program. Patients will receive medication in accordance with the established requirements of national standards and protocols for the treatment of patients with aHUS. The registration of the amount of the drug used will be carried out on the basis of information in the Patient Diaries, as well as primary documentation.
45 studies on the registry are indexed under Atypical Hemolytic Uremic Syndrome; 14 are open to participants now.
This study's enrollment of 50 is below the median of 80 across 15 observational studies indexed under Atypical Hemolytic Uremic Syndrome.
Browse Atypical Hemolytic Uremic Syndrome studies →AO GENERIUM is the lead sponsor of 31 studies on the registry; 3 are open to participants now.
Counted across the registry records on this site, refreshed daily.
At least 50 adult and pediatric patients (from 2 months), with a confirmed diagnosis of aHUS, who meet the enrollment criteria.
Inclusion Criteria
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Eculizumab
Drug: Elizaria®
Induction cycle: 900 mg (3 vials of 30 mL, 10 mg/mL) intravenous infusion for 30 minutes once a week for 4 weeks. Maintenance therapy: 1200 mg (4 vials of 30 mL, 10 mg/mL) intravenous infusion for 30 minutes in Week 5, followed by 1200 mg every 14 days.
Also known as: Eculizumab
Change in platelet count compared to the screening level
Change in platelet count at 52 week after treatment with study drug compared to baseline at screening
Time frame: 52 week
Change in lactate dehydrogenase (LDH) levels from baseline at screening
Change in LDH levels at 52 week after starting study drug treatment from baseline at screening
Time frame: 52 week
Proportion of patients with normalized platelet levels
Proportion of patients with normal platelet count at 52 week after initiation of study drug treatment
Time frame: 52 week
Proportion of patients with no thrombotic microangiopathy (TMA) events
The absence of TMA-related events is defined as the absence, for at least 12 weeks, of: 1) a decrease in platelet counts greater than 25% from baseline at screening; 2) plasma therapy; 3) hemodialysis.
Time frame: 52 week
Proportion of TMA-related interventions
The proportion of TMA-related interventions is defined as (number of plasma therapy sessions + number of hemodialysis sessions) / number of patient days.
Time frame: 52 week
Proportion of patients with complete TMA response
Complete TMA response is defined as the absence of abnormalities in LDH and platelet levels + improvement in renal function (decrease in creatinine levels by 25% or more compared to the baseline value on screening) when performed at least two consecutive tests within 8 weeks
Time frame: 52 week
Change in eGFR (ml / min. / 1.73m2) compared with the baseline level at screening;
Change in eGFR (mL/min/1.73m2) at 52 week after initiation of study drug treatment from baseline at screening
Time frame: 52 week
Proportion of patients with an improvement in glomerular filtration rate (eGFR) of 15 ml / min / 1.73m2 or more compared to the baseline level at screening.
Proportion of patients with improvement in eGFR of 15 ml/min/1.73m2 or more at 52 week after treatment with study drug compared to baseline at screening
Time frame: 52 week
Proportion of patients with more then 1 stage-improvement in chronic kidney desease (CKD) compared to baseline at screening.
Proportion of patients with \>=1 stage improvement in CKD at 52 week after initiation of study drug treatment compared with baseline at screening
Time frame: 52 week
Proportion of patients with an increase in hemoglobin level of more than 20 g / l compared to the baseline level at screening.
Proportion of patients with an increase in hemoglobin level of more than 20 g/l at 52 week after the start of study drug treatment compared with baseline at screening
Time frame: 52 week
Dynamics of membrane attack complex (MAC) level compared to baseline at Visit 2
Changes in MAC levels at 52 week compared to baseline
Time frame: 52 week
The frequency and severity of adverse events (AEs)
Frequency and severity of adverse events (AEs), including serious adverse events (SAEs) and AEs associated with study drug use
Time frame: 52 weeks
Proportion of patients with antidrug antibodies
Proportion of patients with antidrug antibodies; titer of antidrug antibodies and their neutralizing activity
Time frame: 52 weeks
Plan to share: No
No publications or documents are linked to this record.
This study is completed, as verified in Feb 2022. You cannot join it, but the record below documents what was studied.
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Atypical Hemolytic Uremic Syndrome →