Clinical trial NCT05405777: A Retrospective Study to Evaluate the Clinical Outcome According to Treatment in aHUS Patients in South Korea
Official title: A Retrospective Study to Evaluate the Clinical Outcome According to Treatment in Atypical Hemolytic Uremic Syndrome (aHUS) Patients in South Korea
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: aHUS
Keywords: aHUS
eculizumab
Lead sponsor: Handok Inc.
Sponsor class: INDUSTRY
First posted: 2022-06-06
Enrollment: 97
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients who are clinically diagnosed with the atypical hemolytic uremic syndrome (aHUS).

Brief summary: Primary objective

- To determine the clinical prognosis by identifying the disease course and severity by treatment with or without eculizumab in patients with aHUS in Korea.

Secondary objectives

* To determine the clinical prognosis of the disease in all patients with aHUS in Korea.
* To determine the clinical prognosis of the disease in patients diagnosed with aHUS prior to the initiation of eculizumab reimbursement system in Korea.
* To determine the treatment responses by treatment options in patients with aHUS in Korea.
* To identify risk factors that affect mortality in all patients with aHUS in Korea.
* To investigate the recurrence and clinical prognosis in patients with aHUS in Korea when eculizumab is discontinued

Detailed description: As a retrospective, non-interventional, multi-center study

Eligibility criteria: Inclusion Criteria:

* Patients who are clinically diagnosed with the aHUS

Exclusion Criteria:

* Patients who are diagnosed with Shiga toxin-producing E. coli hemolytic-uremic syndrome (STEC-HUS).
* Patients with less than 10% in a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS-13) activity test

Primary outcomes:
measure: Clinical prognosis (Renal survival) by treatment with or without eculizumab
timeFrame: from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, or date of ESRD diagnosis, whichever came first
measure: Clinical prognosis (Overall survival) by treatment with or without eculizumab
timeFrame: from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, whichever came first
Secondary outcomes:
measure: Clinical prognosis (Renal survival) of all aHUS patients in Korea
timeFrame: from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, or date of ESRD diagnosis, whichever came first
measure: Clinical prognosis (Overall survival) of all aHUS patients in Korea
timeFrame: from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, whichever came first
measure: Clinical prognosis (Renal survival) of patients diagnosed with aHUS prior to the initiation of eculizumab reimbursement system in Korea
timeFrame: from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, or date of ESRD diagnosis, whichever came first
measure: Clinical prognosis (Overall survival) of patients diagnosed with aHUS prior to the initiation of eculizumab reimbursement system in Korea
timeFrame: from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, whichever came first
measure: Response rates of each treatment, compared to prior to start treatment
timeFrame: 3rd and 6th months
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Handok
Study officials:
References: