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Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT02614898
Other study ID # ECU-aHUS-403
Secondary ID 2015-003135-35
Status Terminated
Phase
First received
Last updated
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
Health authority
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)

Study Design


Related Conditions & MeSH terms


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
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