A Retrospective Study to Evaluate the Clinical Outcome According to Treatment in aHUS Patients in South Korea

Completed

• Conditions: aHUS

• Registration Number: NCT05405777
• Lead Sponsor: Handok Inc.

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

Study Timeline

• Study First Submitted: 2022-05-13
• Study First Submitted QC: 2022-06-02
• Study First Posted: 2022-06-06
• Study Start: 2022-10-04
• Primary Completion: 2022-12-31
• Study Completion: 2022-12-31
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-24
• Last Update Posted: 2025-01-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 97

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Clinical prognosis (Overall survival) by treatment with or without eculizumab	from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, whichever came first	Overall survival (OS)
Clinical prognosis (Renal survival) by treatment with or without eculizumab	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	Renal survival (end-stage renal disease \[ESRD\]-free survival)

Secondary Outcome Measures

Name	Time	Method
Clinical prognosis (Overall survival) of patients diagnosed with aHUS prior to the initiation of eculizumab reimbursement system in Korea	from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, whichever came first	Overall survival (OS)
Clinical prognosis (Renal survival) of patients diagnosed with aHUS prior to the initiation of eculizumab reimbursement system in Korea	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	Renal survival (ESRD-free survival)
Response rates of each treatment, compared to prior to start treatment	3rd and 6th months	Complete response of TMA
Clinical prognosis (Renal survival) of all aHUS patients in Korea	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	Renal survival (ESRD-free survival)
Clinical prognosis (Overall survival) of all aHUS patients in Korea	from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, whichever came first	Overall survival (OS)

Trial Locations

Locations (1)

Handok; 🇰🇷 Seoul, Korea, Republic of