Atypical Hemolytic Uremic SyndromeaHUS-OPTIMUMNCT07399730
Ravulizumab Outcomes in Polish Patients With aHUS
An observational study of Atypical Hemolytic Uremic Syndrome, run by AstraZeneca at 10 sites in Poland.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Atypical Hemolytic Uremic Syndrome
- What is being tested?
- Ravulizumab
- How many people
- 80 participants
- Where
- 10 sitesPoland
- Study dates
- 24 Jun 2026to 30 Sep 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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).
Who cannot join
- 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 locations and contacts
10 sites in 1 country. Contact the study team or a site near you to ask about taking part.
AstraZeneca Clinical Study Information Center Study Information Center contact
10 sites in 1 country
Poland10 sites, 10 recruiting
- Not yet recruiting
Research Site
Gdansk
- Not yet recruiting
Research Site
Katowice
- Recruiting
Research Site
Katowice
- Not yet recruiting
Research Site
Krakow
- Recruiting
Research Site
Lodz
- Recruiting
Research Site
Poznan
- Not yet recruiting
Research Site
Warsaw
- Recruiting
Research Site
Warsaw
- Not yet recruiting
Research Site
Wroclaw
- Recruiting
Research Site
Wroclaw
About this study
Official title: 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).
- Conditions
- Atypical Hemolytic Uremic Syndrome
- Being tested
- Ravulizumab
- Keywords
- aHUS, Atypical Hemolytic Uremic Syndrome, Ravulizumab, Observational
- Medical subject headings
- Atypical Hemolytic Uremic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RavulizumabDrug
Ultomiris
| Study group | Role | What they receive |
|---|---|---|
| Prospective cohort | Group of patients naive to complement inhibitors Receives: Drug: Ravulizumab | |
| Retrospective cohort | Group of patients who transitioned from other complement inhibitors to ravulizumab. Receives: Drug: Ravulizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: Up to 24 months
Secondary outcomes (8)
Time to Complete TMA Response
Time from initiation of ravulizumab to TMA Response: continuous variable (days; specify when complete response criteria are met)
Measured: Up to 24 months
Proportion of dialysis-free patients
In order to achieve the secondary objectives, the following variables will be estimated
Measured: 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),
Measured: 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
Measured: 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
Measured: 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
Measured: 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.
Measured: 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)
Measured: Up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2026
- Study start24 Jun 2026
- Primary completion30 Sep 2029 (estimated)
- Study completion30 Sep 2029 (estimated)
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.
- More trials run by AstraZenecaThe sponsor's other registered studies.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07399730
- Sponsor's protocol ID
- D928BR00003
- Acronym
- aHUS-OPTIMUM
- Status last verified
- Aug 2026
- First submitted
- 27 Jan 2026
- Last updated
- 15 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Non-interventional Study Evaluating Ravulizumab Treatment Outcomes in Polish Patients With Atypical Hemolytic Uremic Syndrome
- ClinicalTrials.gov ID
- NCT07399730
- Acronym / sponsor ID
- aHUS-OPTIMUM / D928BR00003
- Conditions
- Atypical Hemolytic Uremic Syndrome
- Keywords
- aHUS, Atypical Hemolytic Uremic Syndrome, Ravulizumab, Observational
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Study start
- 24 Jun 2026 (actual)
- Primary completion
- 30 Sep 2029 (estimated)
- Study completion
- 30 Sep 2029 (estimated)
- First posted
- 10 Feb 2026 (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.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Prospective cohort | – | Drug: RavulizumabGroup of patients naive to complement inhibitors |
| Retrospective cohort | – | Drug: RavulizumabGroup of patients who transitioned from other complement inhibitors to ravulizumab. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- 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).
Key exclusion criteria (2 listed)
- 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.
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 |
|---|---|---|
| RavulizumabUltomiris | Drug | Prospective cohort, Retrospective cohort |
Primary outcome
Proportion of patient attaining Complete Thrombotic Microangiopathy (TMA) Response during observation (naïve)
Time frame: Up to 24 months
In order to achieve the primary objectives, the following variables will be estimated: To assess ravulizumab primary treatment outcome in Polish patients with aHUS
Proportion of patients attaining/maintaining. Complete TMA Response during observation (switched)
Time frame: Up to 24 months
In order to achieve the primary objectives, the following variables will be estimated: To assess ravulizumab primary treatment outcome in Polish patients with aHUS
8 secondary outcomes
Time to Complete TMA Response
Time frame: Up to 24 months
Proportion of dialysis-free patients
Time frame: Up to 24 months
Complete TMA response
Time frame: Up to 24 months
Proportion of patients with lab results normalization during observation
Time frame: Up to 24 months
Change from baseline in CKD stage, as evaluated by the physician over time
Time frame: Up to 24 months
Change from baseline in proteinuria status over time
Time frame: 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)
Time frame: Up to 24 months
Change form baseline in EQ- 5D-5L (adults) and EQ-5D-Y- 5L (paediatric patients) score overtime (naïve)
Time frame: Up to 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2026.
- Individual participant dataSharing planned: Yes. 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.
Related trials for Atypical Hemolytic Uremic Syndrome
Active, not recruitingObservational study
A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Atypical Hemolytic Uremic Syndrome Patients
Atypical Hemolytic Uremic Syndrome(aHUS)
Active, not recruitingPhase 3
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Atypical Hemolytic Uremic Syndrome
Active, not recruitingPhase 3
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Atypical Hemolytic Uremic Syndrome
RecruitingPhase 3
Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
Atypical Hemolytic Uremic Syndrome
RecruitingPhase 3
Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Atypical Hemolytic Uremic Syndrome
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.
AstraZeneca Clinical Study Information Center Study Information Center contact
10 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, run by AstraZeneca at 10 sites in Poland.
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 2 inclusion and 2 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: looking for participants now, as of the sponsor's last update on 15 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in Poland. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact AstraZeneca Clinical Study Information Center Study Information Center (+118772409479, information.center@astrazeneca.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of patient attaining Complete Thrombotic Microangiopathy (TMA) Response during observation (naïve) (time frame: Up to 24 months). The study lists 2 primary outcomes in all. 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 15 Sep 2026.