Clinical trial NCT06099236: A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Atypical Hemolytic Uremic Syndrome Patients
Official title: A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Chinese Atypical Hemolytic Uremic Syndrome Patients
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Atypical Hemolytic Uremic Syndrome(aHUS)
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
First posted: 2023-10-25
Enrollment: 1070
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: paediatric and adult patients diagnosed with aHUS

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


Eligibility criteria: 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.

Primary outcomes:
measure: Event free survival, where event defined as ESRD or death.
timeFrame: 12 Months
Secondary outcomes:
measure: Death
timeFrame: 12 Months
measure: End-stage renal disease(ESRD)
timeFrame: 12 Months
measure: 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
timeFrame: 12 Months
measure: Complete Thrombotic Microangiopathy(TMA ) Response status over time
timeFrame: 12 Months
measure: Time to Complete Thrombotic Microangiopathy(TMA ) Response
timeFrame: 12 Months
measure: Thrombotic Microangiopathy(TMA ) relapse Dialysis requirement status over time
timeFrame: 12 Months
measure: Observed value and change from baseline in estimated glomerular filtration rate (eGFR)
timeFrame: 12 Months
measure: Chronic kidney disease (CKD) stage, as evaluated by the Investigator and classified as improved, stable (no change), or worsened compared to baseline
timeFrame: 12 Months
measure: Status of Proteinuria over time.
timeFrame: 12 Months
measure: Albumin-to-creatinine ratio over time.
timeFrame: 12 Months
measure: Plasma C3, C4, CH50, Factor H and I, soluble C5b-9 (sC5b-9) and CD46 expression over time.
timeFrame: 12 Months
measure: 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.
timeFrame: 12 Months
measure: 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)
timeFrame: 12 Months
measure: Descriptive data of Serious Adverse Events (SAEs) for treatment period 2.
timeFrame: 12 Months
measure: Observed Value in Platelet Count
timeFrame: 12 Months
measure: Observed Value in Lactate Dehydrogenase (LDH) (mg/dL)
timeFrame: 12 Months
measure: Observed Value in Hemoglobin (mg/dL)
timeFrame: 12 Months
measure: Change from Baseline in Platelet Count
timeFrame: 12 Months
measure: Change from Baseline in Lactate Dehydrogenase (LDH) (mg/dL)
timeFrame: 12 Months
measure: Change from Baseline in Hemoglobin (mg/dL)
timeFrame: 12 Months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Research Site
state: Beijing
city: Shijiazhuang
country: China
facility: Research Site
state: Heibei
city: Luzhou
country: China
facility: Research Site
state: Sichuan
city: Baoding
country: China
facility: Research Site
city: Beijing
country: China
facility: Research Site
city: Beijing
country: China
facility: Research Site
city: Changsha
country: China
facility: Research Site
city: Chengdu
country: China
facility: Research Site
city: Chongqing
country: China
facility: Research Site
city: Foshan
country: China
facility: Research Site
city: Fuzhou
country: China
facility: Research Site
city: Guangzhou
country: China
facility: Research Site
city: Guizhou
country: China
facility: Research Site
city: Haikou
country: China
facility: Research Site
city: Hangzhou
country: China
facility: Research Site
city: Hangzhou
country: China
facility: Research Site
city: Hefei
country: China
facility: Research Site
city: Hefei
country: China
facility: Research Site
city: Huai'an
country: China
facility: Research Site
city: Jinan
country: China
facility: Research Site
city: Luoyang
country: China
facility: Research Site
city: Nanchang
country: China
facility: Research Site
city: Nanchong
country: China
facility: Research Site
city: Nanjing
country: China
facility: Research Site
city: Nanning
country: China
facility: Research Site
city: Nanning
country: China
facility: Research Site
city: Ningbo
country: China
facility: Research Site
city: Qingdao
country: China
facility: Research Site
city: Shanghai
country: China
facility: Research Site
city: Shanghai
country: China
facility: Research Site
city: Shenyang
country: China
facility: Research Site
city: Shenzhen
country: China
facility: Research Site
city: Shijiazhuang
country: China
facility: Research Site
city: Shijiazhuang
country: China
facility: Research Site
city: Suzhou
country: China
facility: Research Site
city: Taiyuan
country: China
facility: Research Site
city: Taiyuan
country: China
facility: Research Site
city: Tianjin
country: China
facility: Research Site
city: Tianjin
country: China
facility: Research Site
city: Wuhan
country: China
facility: Research Site
city: Wulumuqi
country: China
facility: Research Site
city: Xiamen
country: China
facility: Research Site
city: Xian
country: China
facility: Research Site
city: Xuzhou
country: China
facility: Research Site
city: Zhengzhou
country: China
facility: Research Site
Study officials:
References: