Atypical Hemolytic Uremic SyndromeNCT04749810
Observational Study of Elizaria® in aHUS Patients
An observational study of Atypical Hemolytic Uremic Syndrome and aHUS, run by AO GENERIUM at 8 sites in Russia.
Trial summary
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 Read more about this study
- Who is it for?
- Ages 2 months and older
- Conditions the study is for
- Atypical Hemolytic Uremic Syndrome, aHUS
- What is being tested?
- Elizaria®
- Type of treatment
- Injection or infusion
- How many people
- 50 participants
- Where
- 8 sitesRussia
- Study dates
- 19 Dec 2019to 30 Apr 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 months and older
- All sexes
- Child, Adult, Older adult
Study population: At least 50 adult and pediatric patients (from 2 months), with a confirmed diagnosis of aHUS, who meet the enrollment criteria.
Who may be able to join
- Written informed consent to study participation.
- Male and female patients aged 2 months and older with documented atypical hemolytic uremic syndrome (aHUS)diagnosis.
- By the time of inclusion in the study, Elizaria® should be prescribed as a pathogenetic therapy for aHUS; Exclusion Criteria
- 1. Intolerance to eculizumab, or other components of the drug.
Who cannot join
None listed.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
Russia8 sites
Federal State Budgetary Educational Institution of Higher Education "Kazan State Medical University" of the Ministry of Health of the Russian Federation
Kazan', 420012
Regional State Budgetary Healthcare Institution "Krasnoyarsk' Regional Clinical Center for Maternity and Childhood Protection"
Krasnoyarsk, 660074
State budgetary healthcare institution of the city of Moscow "Children's City Clinical Hospital of St. Vladimir of the Healthcare Department of the City of Moscow"
Moscow, 107014
Federal State Autonomous Educational Institution of Higher Education "First Moscow State Medical University n.a. I.M. Sechenov" of the Ministry of Health of the Russian Federation
Moscow, 119991
State budgetary healthcare institution of the City of Moscow "City clinical hospital #52 of the Moscow City Healthcare Department"
Moscow, 123182
State budgetary healthcare institution of the city of Moscow "City Clinical Hospital n.a. A.K. Eramishantsev of the Moscow City Healthcare Department"
Moscow, 129327
St. Petersburg's State Budgetary Healthcare Institution "City Mariinsky Hospital"
Saint Petersburg, 191104
St. Petersburg's State Budgetary Healthcare Institution "Children's City Multidisciplinary Clinical Specialized Center of High Medical Technologies"
Saint Petersburg, 198205
About this study
Official title: Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® in Patients With Atypical Hemolytic Uremic Syndrome
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
Read the detailed description
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.
- Conditions
- Atypical Hemolytic Uremic Syndrome, aHUS
- Being tested
- Elizaria®
- Keywords
- Atypical Hemolytic Uremic Syndrome, Azotemia, Hemolytic-Uremic Syndrome, Hemolysis, Syndrome, Disease, Pathologic Processes, Uremia, Kidney Diseases, Urologic Diseases, Anemia, Hemolytic, Anemia, Hematologic Diseases, Thrombotic Microangiopathies, Thrombocytopenia, Blood Platelet Disorders
- Medical subject headings
- Atypical Hemolytic Uremic Syndrome, Azotemia, Hemolytic-Uremic Syndrome, Hemolysis, Syndrome, Disease, Pathologic Processes, Uremia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Elizaria®Drug
Also called Eculizumab
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.
| Study group | Role | What they receive |
|---|---|---|
| Elizaria® | Eculizumab Receives: Drug: Elizaria® |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 52 week
Secondary outcomes (12)
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
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 52 weeks
Proportion of patients with antidrug antibodies
Proportion of patients with antidrug antibodies; titer of antidrug antibodies and their neutralizing activity
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2021
- Study start19 Dec 2019
- Primary completion28 Feb 2022
- Study completion30 Apr 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Atypical Hemolytic Uremic SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- AO GENERIUM
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Oksana A. Markova, MD, Study chair, AO GENERIUM
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04749810
- Sponsor's protocol ID
- ECU-aHUS-N01
- Status last verified
- Feb 2022
- First submitted
- 8 Feb 2021
- Last updated
- 4 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® in Patients With Atypical Hemolytic Uremic Syndrome
- ClinicalTrials.gov ID
- NCT04749810
- Acronym / sponsor ID
- ECU-aHUS-N01
- Conditions
- Atypical Hemolytic Uremic Syndrome, aHUS
- Keywords
- Atypical Hemolytic Uremic Syndrome, Azotemia, Hemolytic-Uremic Syndrome, Hemolysis, Syndrome, Disease, Pathologic Processes, Uremia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- AO GENERIUMIndustry (pharma, biotech, devices)
- Study start
- 19 Dec 2019 (actual)
- Primary completion
- 28 Feb 2022 (actual)
- Study completion
- 30 Apr 2022 (actual)
- First posted
- 11 Feb 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
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.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Elizaria® | – | Drug: Elizaria®Eculizumab |
Who may qualify
- Ages
- 2 months and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- At least 50 adult and pediatric patients (from 2 months), with a confirmed diagnosis of aHUS, who meet the enrollment criteria.
Key inclusion criteria (4 listed)
- Written informed consent to study participation.
- Male and female patients aged 2 months and older with documented atypical hemolytic uremic syndrome (aHUS)diagnosis.
- By the time of inclusion in the study, Elizaria® should be prescribed as a pathogenetic therapy for aHUS; Exclusion Criteria
- 1. Intolerance to eculizumab, or other components of the drug.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Elizaria®Also: EculizumabInduction 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. | Drug | Elizaria® |
Primary outcome
Change in platelet count compared to the screening level
Time frame: 52 week
Change in platelet count at 52 week after treatment with study drug compared to baseline at screening
12 secondary outcomes
Change in lactate dehydrogenase (LDH) levels from baseline at screening
Time frame: 52 week
Proportion of patients with normalized platelet levels
Time frame: 52 week
Proportion of patients with no thrombotic microangiopathy (TMA) events
Time frame: 52 week
Proportion of TMA-related interventions
Time frame: 52 week
Proportion of patients with complete TMA response
Time frame: 52 week
Change in eGFR (ml / min. / 1.73m2) compared with the baseline level 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.
Time frame: 52 week
Proportion of patients with more then 1 stage-improvement in chronic kidney desease (CKD) compared to 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.
Time frame: 52 week
Dynamics of membrane attack complex (MAC) level compared to baseline at Visit 2
Time frame: 52 week
The frequency and severity of adverse events (AEs)
Time frame: 52 weeks
Proportion of patients with antidrug antibodies
Time frame: 52 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Aug 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Oksana A. Markova, MD study chair
AO GENERIUM
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Atypical Hemolytic Uremic Syndrome and aHUS, run by AO GENERIUM at 8 sites in Russia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in platelet count compared to the screening level (time frame: 52 week). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Aug 2022.