A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Ou… (NCT06099236) | Clinical Trial Compass
Active — Not RecruitingNot Applicable
A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Atypical Hemolytic Uremic Syndrome Patients
China367 participantsStarted 2024-01-15
Plain-language 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
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Patients of any age, who are diagnosed as aHUS by a professional physician (first epi-sode or relapse).
. 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
. 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Event free survival, where event defined as ESRD or death on or after TMA onset.
. 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.