EVIDENCE - EVALUATION OF POTENTIAL PREDICTORS OF DISEASE PROGRESSION IN PATIENTS WITH aHUS, INCLUDING GENETICS, BIOMARKERS, AND TREATMENT

Phase 1

• Conditions: atypical Hemolytic-Uremic Syndrome (aHUS); MedDRA version: 20.0Level: LLTClassification code 10019515Term: Hemolytic uremic syndromeSystem Organ Class: 100000004851; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2015-003135-35-IT
• Lead Sponsor: ALEXION PHARMACEUTICALS INCORPORATED

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2018-04-20
• Last Update Posted: 14 May 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 330

Inclusion Criteria

1. Currently receiving eculizumab treatment in the M11-001 aHUS Registry
2. Two normal platelet counts at least 4 weeks apart
3. Two normal LDH levels at least 4 weeks apart
4. Willing, committed, and able to return for all clinic visits and complete all study related procedures
5. Patient or patient's parent/legal guardian must be willing and able to give written informed consent. Patient (if minor) must be willing to give written informed assent (if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees [IRB/IEC]).
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 155
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 50

Exclusion Criteria

1. Any prior intentional discontinuation of eculizumab treatment
2. On chronic dialysis (defined as =3 months on dialysis)
3. Currently participating in another complement inhibitor trial
4. With life expectancy of <6 months
5. Patient or patient's parent/legal guardian unable to give written informed consent. Patient (if minor) unable to give written informed assent (if applicable as determined by the central Institutional Review
Boards/Independent Ethics Committees [IRB/IEC]).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess TMA disease manifestations in patients with aHUS with or without ongoing eculizumab treatment in a real-world setting.;Secondary Objective: To evaluate potential clinical predictors of disease manifestation and progression, including clinical characteristics and genetic profiling to evaluate disease activity and relationship to treatment as determined by levels of biomarkers associated with complement activation, and the use of associated supportive interventions such as PE/PI, dialysis, blood transfusions, and renal transplantation.;Primary end point(s): Rate of TMA manifestations, where TMA includes:<br>1. Hematologic or renal events due to aHUS<br>2. Extra-renal clinical signs and symptoms of aHUS<br>3. Tissue (eg, kidney transplant) biopsy demonstrating TMA due to aHUS;Timepoint(s) of evaluation of this end point: From week 2 up to month 47

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): • Change in estimated Glomerular Filtration Rate (eGFR) over time<br>• Rate of TMA manifestations<br>• Incidence of PE/PI;Timepoint(s) of evaluation of this end point: From week 2 up to month 47