A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Atypical Hemolytic Uremic Syndrome Patients

Recruiting

• Conditions: Atypical Hemolytic Uremic Syndrome(aHUS)

• Registration Number: NCT06099236
• Lead Sponsor: AstraZeneca

Brief 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. 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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-09-29
• Study First Submitted QC: 2023-10-23
• Study First Posted: 2023-10-25
• Study Start: 2024-01-15
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-23
• Last Update Posted: 2025-04-24
• Primary Completion: 2028-11-30
• Study Completion: 2028-11-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 1070

Inclusion Criteria

Participants are eligible to be included in the study. Age

1. Patients of any age, who are diagnosed as aHUS by a professional physician (first epi-sode or relapse).

   Type of Patient and Disease Characteristics

2. Evidence of TMA, including thrombocytopenia, evidence of hemolysis, and kidney dys-function, based on the following laboratory findings, should be recorded within 1 week time frame:

   1. Platelet count < 150 per microliter (μL), and
   2. 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
   3. 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).

3. Gender: Male and/or female.. Informed Consent

4. 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.

Exclusion Criteria

Participants are excluded from the study if any of the following criteria apply:

Medical Conditions

1. Patients who were diagnosed with HUS only due to Shiga toxin-producing Escherichia coli (STEC).
2. Patients who were diagnosed with TTP (ADAMTS13 activity <10%). Other Exclusions
3. Unable to give written informed consent.
4. 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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Event free survival, where event defined as ESRD or death.	12 Months

Secondary Outcome Measures

Name	Time	Method
Complete Thrombotic Microangiopathy(TMA ) Response status over time	12 Months
Observed value and change from baseline in estimated glomerular filtration rate (eGFR)	12 Months
Status of Proteinuria over time.	12 Months
Change from Baseline in Platelet Count	12 Months
Thrombotic Microangiopathy(TMA ) relapse Dialysis requirement status over time	12 Months
Chronic kidney disease (CKD) stage, as evaluated by the Investigator and classified as improved, stable (no change), or worsened compared to baseline	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)	12 Months
Observed Value in Platelet Count	12 Months
Change from Baseline in Lactate Dehydrogenase (LDH) (mg/dL)	12 Months
End-stage renal disease(ESRD)	12 Months
Time to Complete Thrombotic Microangiopathy(TMA ) Response	12 Months
Change from Baseline in Hemoglobin (mg/dL)	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	12 Months
Death	12 Months
Albumin-to-creatinine ratio over time.	12 Months
Descriptive data of Serious Adverse Events (SAEs) for treatment period 2.	12 Months
Observed Value in Hemoglobin (mg/dL)	12 Months
Plasma C3, C4, CH50, Factor H and I, soluble C5b-9 (sC5b-9) and CD46 expression over time.	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.	12 Months
Observed Value in Lactate Dehydrogenase (LDH) (mg/dL)	12 Months

Trial Locations

Locations (1)

Research Site; 🇨🇳 Zhengzhou, China