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: 19.1 Level: LLT Classification code 10019515 Term: Hemolytic uremic syndrome System Organ Class: 100000004851; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2015-003135-35-GB
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-06-28
• Study Completion: 2017-10-05
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 67

Inclusion Criteria

1. Currently receiving eculizuab 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: 125
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