Clinical trial NCT02614898: Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
Official title: Evidence - Evaluation of Potential Predictors of Disease Progression in Patients With aHUS, Including Genetics, Biomarkers and Treatment
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Atypical Hemolytic Uremic Syndrome
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2015-11-25
Enrollment: 67
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Participants with aHUS enrolled in the M11-001 aHUS Registry and currently receiving eculizumab with no prior intentional eculizumab discontinuations at any time (for example, not occasional, unplanned, or temporary missed doses of eculizumab, but intended long-term discontinuation).

Brief 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.


Eligibility criteria: 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)

Primary outcomes:
measure: Rate Of Thrombotic Microangiopathy (TMA) Manifestations During Eculizumab Treatment Compared To Off-Treatment
timeFrame: Baseline, 24 Months
Secondary outcomes:
measure: Change From Baseline To 24 Months In Estimated Glomerular Filtration Rate (eGFR)
timeFrame: Baseline, 24 Months
measure: Incidence Of Plasma Exchange And Plasma Infusion (PE/PI)
timeFrame: Baseline, 24 Months
Other outcomes:
Locations:
city: Washington
country: United States
facility: Clinical Trial Site
state: District of Columbia
city: Atlanta
country: United States
facility: Clinical Trial Site
state: Georgia
city: Chicago
country: United States
facility: Clinical Trial Site
state: Illinois
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: Hackensack
country: United States
facility: Clinical Trial Site
state: New Jersey
city: Charlotte
country: United States
facility: Clinical Trial Site
state: North Carolina
city: Columbus
country: United States
facility: Clinical Trial Site
state: Ohio
city: Houston
country: United States
facility: Clinical Trial Site
state: Texas
city: Adelaide
country: Australia
facility: Clinical Trial Site
city: Clayton
country: Australia
facility: Clinical Trial Site
city: Heidelberg
country: Australia
facility: Clinical Trial Site
city: Kingswood
country: Australia
facility: Clinical Trial Site
city: Liverpool
country: Australia
facility: Clinical Trial Site
city: Nedlands
country: Australia
facility: Clinical Trial Site
city: Parkville
country: Australia
facility: Clinical Trial Site
city: Parkville
country: Australia
facility: Clinical Trial Site
city: Perth
country: Australia
facility: Clinical Trial Site
city: Westmead
country: Australia
facility: Clinical Trial Site
city: Woolloongabba
country: Australia
facility: Clinical Trial Site
city: Hannover
country: Germany
facility: Clinical Trial Site
city: Hannöver
country: Germany
facility: Clinical Trial Site
city: Kiel
country: Germany
facility: Clinical Trial Site
city: Luebeck
country: Germany
facility: Clinical Trial Site
city: London
country: United Kingdom
facility: Clinical Trial Site
Study officials:
References: