Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment

Atypical Hemolytic Uremic Syndrome Clinical Trial

— EVIDENCE  

Official title:

Evidence - Evaluation of Potential Predictors of Disease Progression in Patients With aHUS, Including Genetics, Biomarkers and Treatment

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02614898
• Other study ID #: ECU-aHUS-403
• Secondary ID: 2015-003135-35
• Status: Terminated
• Start date: November 4, 2015
• Est. completion date: October 5, 2017

Study information

• Verified date: December 2019
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This was a prospective, open-label study with no participant randomization. Treatment for aHUS was observational and at the discretion of the treating physician. The purpose of this study was to assess disease manifestations of complement-mediated thrombotic microangiopathy (TMA) and evaluate potential clinical predictors of disease manifestations and progression in participants with aHUS with or without eculizumab treatment in the clinical setting.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 67
• Est. completion date: October 5, 2017
• Est. primary completion date: October 5, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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 lactate dehydrogenase levels at least 4 weeks apart

4. Willing, committed, and able to return for all clinic visits and complete all study-related procedures

5. Participant or participant's parent/legal guardian must have been willing and able to give written informed consent. Participant (if minor) must have been willing to give written informed assent (if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees)

Exclusion Criteria:

1. Any prior eculizumab treatment discontinuation

2. On chronic dialysis (defined as =3 months on dialysis)

3. Currently participating in another complement inhibitor trial

4. Life expectancy of <6 months

5. Participant or participant's parent/legal guardian was unable to give written informed consent. Participant (if minor) was unable to give written informed assent (if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees)

Related Conditions & MeSH terms

• Atypical Hemolytic Uremic Syndrome
• Disease Progression
• Hemolysis
• Hemolytic-Uremic Syndrome
• Syndrome

Intervention

Other:
• Eculizumab
This study was an observational study. Therefore, the study drug was not provided as part of this study, and the dosing regimen was based solely at the discretion of the treating physician.

Locations

Country	Name	City	State
Australia	Clinical Trial Site	Adelaide
Australia	Clinical Trial Site	Clayton
Australia	Clinical Trial Site	Heidelberg
Australia	Clinical Trial Site	Kingswood
Australia	Clinical Trial Site	Liverpool
Australia	Clinical Trial Site	Nedlands
Australia	Clinical Trial Site	Parkville
Australia	Clinical Trial Site	Parkville
Australia	Clinical Trial Site	Perth
Australia	Clinical Trial Site	Westmead
Australia	Clinical Trial Site	Woolloongabba
Germany	Clinical Trial Site	Hannover
Germany	Clinical Trial Site	Hannöver
Germany	Clinical Trial Site	Kiel
Germany	Clinical Trial Site	Luebeck
United Kingdom	Clinical Trial Site	London
United States	Clinical Trial Site	Atlanta	Georgia
United States	Clinical Trial Site	Boston	Massachusetts
United States	Clinical Trial Site	Charlotte	North Carolina
United States	Clinical Trial Site	Chicago	Illinois
United States	Clinical Trial Site	Columbus	Ohio
United States	Clinical Trial Site	Hackensack	New Jersey
United States	Clinical Trial Site	Houston	Texas
United States	Clinical Trial Site	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals

Countries where clinical trial is conducted

United States,  Australia,  Germany,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Rate Of Thrombotic Microangiopathy (TMA) Manifestations During Eculizumab Treatment Compared To Off-Treatment	A TMA manifestation was defined as one of following: Hematologic or renal events due to aHUS; extra-renal clinical signs and symptoms of aHUS; tissue (for example, kidney transplant) biopsy demonstrating TMA due to aHUS.; The study was terminated with only 20% of the planned enrollment and due to the subsequent loss of funding for the program, the samples collected for outcome measure assessment could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.	Baseline, 24 Months
Secondary	Change From Baseline To 24 Months In Estimated Glomerular Filtration Rate (eGFR)	Change in estimated eGFR over time using the chronic kidney disease-epidemiology (CKD-EPI) formula.; The study was terminated with only 20% of the planned enrollment and due to the subsequent loss of funding for the program, the samples collected for outcome measure assessment could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.	Baseline, 24 Months
Secondary	Incidence Of Plasma Exchange And Plasma Infusion (PE/PI)	The number of occurrences of PE/PI per participant-years was calculated and summarized by treatment status.; The study was terminated with only 20% of the planned enrollment and due to the subsequent loss of funding for the program, the samples collected for outcome measure assessment could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.	Baseline, 24 Months