Atypical Hemolytic Uremic Syndrome(aHUS)NCT06099236
A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Atypical Hemolytic Uremic Syndrome Patients
An observational study of Atypical Hemolytic Uremic Syndrome(aHUS), run by AstraZeneca at 26 sites in China.
Trial summary
This is a China, non-interventional, observational study and will follow the Good Phar-macoepidemiology Practices guidelines. This study will enrol paediatric and adult patients diagnosed with aHUS who will be treated according to routine clinical practice defined by local institutional treatment guidelines/protocol. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Atypical Hemolytic Uremic Syndrome(aHUS)
- How many people
- 367 participants
- Where
- 26 sitesChina
- Study dates
- 15 Jan 2024to 31 Dec 2027
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: paediatric and adult patients diagnosed with aHUS
Who may be able to join
- Participants are eligible to be included in the study. Age
- Patients of any age, who are diagnosed as aHUS by a professional physician (first epi-sode or relapse).
- Type of Patient and Disease Characteristics
- Evidence of TMA, including thrombocytopenia, evidence of hemolysis, and kidney dys-function, based on the following laboratory findings, should be recorded within 2 weeks time frame:
- Platelet count < 150,000 per microliter (μL), and
- Mechanic hemolytic anemia evident by LDH ≥ 1.5 × upper limit of normal (ULN), and hemoglobin ≤ lower limit of normal (LLN) for age and gender and
Show all 9Show fewer
- Serum creatinine level ≥ ULN in adults (≥18 years of age), or ≥ 97.5th percentile for age at screening in children (patients who require dialysis for acute kidney injury are also eligi-ble).
- Gender: Male and/or female. Informed Consent
- Willing and able to give written informed consent and comply with the study visit schedule as described in Section 6.2.1. For patients < 18 years of age, patient's legal guardian must be willing and able to give written informed consent and the patient must be willing to give written informed assent (if applicable as determined by the central.
Who cannot join
- Participants are excluded from the study if any of the following criteria apply:
- Medical Conditions
- Patients who were diagnosed with HUS only due to Shiga toxin-producing Escherichia coli (STEC).
- Patients who were diagnosed with TTP (ADAMTS13 activity <10%). Other Exclusions
- Unable to give written informed consent.
- Any medical or psychological condition that, in the opinion of the Investigator, could increase the risk to the participant by participating in the study or confound the outcome of the study.
Trial locations and contacts
26 sites in 1 country.
26 sites in 1 country
China26 sites
Research Site
Anhui
Research Site
Beijing, Beijing Municipality
Research Site
Beijing
Research Site
Chongqing, Sichuan
Research Site
Chongqing
Research Site
Fujian
Research Site
Guangdong
Research Site
Guangxi
Research Site
Guizhou
Research Site
Hainan
Research Site
Hebei
Research Site
Henan
Research Site
Hong Kong
Research Site
Hubei
Research Site
Hunan
Research Site
Jiangsu
Research Site
Jiangxi
Research Site
Liaoning
Research Site
Shaanxi
Research Site
Shandong
Research Site
Shanghai
Research Site
Shanxi
Research Site
Sichuan
Research Site
Tianjin
Research Site
Xinjiang
Research Site
Zhejiang
About this study
Official title: A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Chinese Atypical Hemolytic Uremic Syndrome Patients
This is a China, non-interventional, observational study and will follow the Good Phar-macoepidemiology Practices guidelines.
This study will enrol paediatric and adult patients diagnosed with aHUS who will be treated according to routine clinical practice defined by local institutional treatment guidelines/protocol. Those aHUS patients who will be treated with a supportive therapy, which does not contain eculizumab, will be monitored for up to 12 months since the ini-tial diagnosis. Patients initiated on eculizumab treatment anytime between aHUS diagno-sis until 12 months will be followed for additional 12 months, starting from the ecu initia-tion. Patient disposition, characteristics, outcomes and safety will be described for all pa-tients enrolled into this study
- Conditions
- Atypical Hemolytic Uremic Syndrome(aHUS)
- Medical subject headings
- Atypical Hemolytic Uremic Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
Event free survival, where event defined as ESRD or death on or after TMA onset.
Measured: 12 Months
Secondary outcomes (21)
Death
Measured: 12 Months
End-stage renal disease(ESRD)
Measured: 12 Months
Complete TMA Response during observation the as evidenced by simultaneous normalization of hemato-logical parameters (platelet count and LDH) and ≥ 25% improvement in serum creatinine from baseline
Measured: 12 Months
Complete Thrombotic Microangiopathy(TMA ) Response status over time
Measured: 12 Months
Time to Complete Thrombotic Microangiopathy(TMA ) Response
Measured: 12 Months
Thrombotic Microangiopathy(TMA ) relapse
Measured: 12 Months
Observed value and change from baseline in estimated glomerular filtration rate (eGFR)
Measured: 12 Months
Chronic kidney disease (CKD) stage, as evaluated by the Investigator and classified as improved, stable (no change), or worsened compared to baseline
Measured: 12 Months
Proteinuria over time.
Measured: 12 Months
Urine protein-to-creatinine ratio over time.
Measured: 12 Months
Plasma C3, C4, CH50, Factor H and I, soluble C5b-9 (sC5b-9) and CD46 expression over time.
Measured: 12 Months
The proportion of patients with normal platelet count (≥150 x 109/L),LDH levels ≤upper limit of normal , serum creatinine < up-per limit of normal for age or an improvement > 25% compared to baseline,eGFR≥ 60 mL/min/1.73 m2, proteinuria negative.
Measured: 12 Months
Descriptive data of events of interest (serious infec-tions (Aspergillus infections and infections due to en-capsulated bacteria such as Neisseria meningitidis), pregnancy, lactation, and follow-up-data on neonates for 3 months after delivery)
Measured: 12 Months
Descriptive data of Serious Adverse Events (SAEs) for treatment period 2.
Measured: 12 Months
Observed Value in Platelet Count
Measured: 12 Months
Observed Value in Lactate Dehydrogenase (LDH) (mg/dL)
Measured: 12 Months
Observed Value in Hemoglobin (mg/dL)
Measured: 12 Months
Change from Baseline in Platelet Count
Measured: 12 Months
Change from Baseline in Lactate Dehydrogenase (LDH) (mg/dL)
Measured: 12 Months
Change from Baseline in Hemoglobin (mg/dL)
Measured: 12 Months
The number of patients still on dialysis during each follow-up visit
Measured: 12 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Oct 2023
- Study start15 Jan 2024
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (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 Syndrome(aHUS)Studies 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
- Other
- Time perspective
- Prospective
- Enrolment
- 367 participants (actual)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06099236
- Sponsor's protocol ID
- D7413R00001
- Status last verified
- Oct 2026
- First submitted
- 29 Sep 2023
- Last updated
- 26 Oct 2026
- Results posted
- 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 approved.
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Chinese Atypical Hemolytic Uremic Syndrome Patients
- ClinicalTrials.gov ID
- NCT06099236
- Acronym / sponsor ID
- D7413R00001
- Conditions
- Atypical Hemolytic Uremic Syndrome(aHUS)
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 367 participants (actual)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Study start
- 15 Jan 2024 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 25 Oct 2023 (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 is a China, non-interventional, observational study and will follow the Good Phar-macoepidemiology Practices guidelines.
This study will enrol paediatric and adult patients diagnosed with aHUS who will be treated according to routine clinical practice defined by local institutional treatment guidelines/protocol. Those aHUS patients who will be treated with a supportive therapy, which does not contain eculizumab, will be monitored for up to 12 months since the ini-tial diagnosis. Patients initiated on eculizumab treatment anytime between aHUS diagno-sis until 12 months will be followed for additional 12 months, starting from the ecu initia-tion. Patient disposition, characteristics, outcomes and safety will be described for all pa-tients enrolled into this study
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- paediatric and adult patients diagnosed with aHUS
Key inclusion criteria (9 listed)
- Participants are eligible to be included in the study. Age
- Patients of any age, who are diagnosed as aHUS by a professional physician (first epi-sode or relapse).
- Type of Patient and Disease Characteristics
- Evidence of TMA, including thrombocytopenia, evidence of hemolysis, and kidney dys-function, based on the following laboratory findings, should be recorded within 2 weeks time frame:
- Gender: Male and/or female. Informed Consent
Key exclusion criteria (6 listed)
- Medical Conditions
- Patients who were diagnosed with HUS only due to Shiga toxin-producing Escherichia coli (STEC).
- Patients who were diagnosed with TTP (ADAMTS13 activity <10%). Other Exclusions
- Unable to give written informed consent.
- Any medical or psychological condition that, in the opinion of the Investigator, could increase the risk to the participant by participating in the study or confound the outcome of the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Event free survival, where event defined as ESRD or death on or after TMA onset.
Time frame: 12 Months
21 secondary outcomes
Death
Time frame: 12 Months
End-stage renal disease(ESRD)
Time frame: 12 Months
Complete TMA Response during observation the as evidenced by simultaneous normalization of hemato-logical parameters (platelet count and LDH) and ≥ 25% improvement in serum creatinine from baseline
Time frame: 12 Months
Complete Thrombotic Microangiopathy(TMA ) Response status over time
Time frame: 12 Months
Time to Complete Thrombotic Microangiopathy(TMA ) Response
Time frame: 12 Months
Thrombotic Microangiopathy(TMA ) relapse
Time frame: 12 Months
Observed value and change from baseline in estimated glomerular filtration rate (eGFR)
Time frame: 12 Months
Chronic kidney disease (CKD) stage, as evaluated by the Investigator and classified as improved, stable (no change), or worsened compared to baseline
Time frame: 12 Months
Proteinuria over time.
Time frame: 12 Months
Urine protein-to-creatinine ratio over time.
Time frame: 12 Months
Plasma C3, C4, CH50, Factor H and I, soluble C5b-9 (sC5b-9) and CD46 expression over time.
Time frame: 12 Months
The proportion of patients with normal platelet count (≥150 x 109/L),LDH levels ≤upper limit of normal , serum creatinine < up-per limit of normal for age or an improvement > 25% compared to baseline,eGFR≥ 60 mL/min/1.73 m2, proteinuria negative.
Time frame: 12 Months
Descriptive data of events of interest (serious infec-tions (Aspergillus infections and infections due to en-capsulated bacteria such as Neisseria meningitidis), pregnancy, lactation, and follow-up-data on neonates for 3 months after delivery)
Time frame: 12 Months
Descriptive data of Serious Adverse Events (SAEs) for treatment period 2.
Time frame: 12 Months
Observed Value in Platelet Count
Time frame: 12 Months
Observed Value in Lactate Dehydrogenase (LDH) (mg/dL)
Time frame: 12 Months
Observed Value in Hemoglobin (mg/dL)
Time frame: 12 Months
Change from Baseline in Platelet Count
Time frame: 12 Months
Change from Baseline in Lactate Dehydrogenase (LDH) (mg/dL)
Time frame: 12 Months
Change from Baseline in Hemoglobin (mg/dL)
Time frame: 12 Months
The number of patients still on dialysis during each follow-up visit
Time frame: 12 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 26 Oct 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 approved.
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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.
The record lists no contact. Use the official record to reach the sponsor.
26 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(aHUS), run by AstraZeneca at 26 sites in China.
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 9 inclusion and 6 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: under way, not looking for participants, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in China. 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?
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?
Event free survival, where event defined as ESRD or death on or after TMA onset. (time frame: 12 Months). 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 26 Oct 2026.