Atypical Hemolytic Uremic SyndromeEVIDENCENCT02614898
Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
An observational study of Atypical Hemolytic Uremic Syndrome, run by Alexion Pharmaceuticals, Inc. at 24 sites in 4 countries.
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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Atypical Hemolytic Uremic Syndrome
- What is being tested?
- Eculizumab
- How many people
- 67 participants
- Where
- 24 sitesin 4 countries
- Study dates
- 4 Nov 2015to 5 Oct 2017
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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).
Who may be able to join
- Currently receiving eculizumab treatment in the M11-001 aHUS Registry
- Two normal platelet counts at least 4 weeks apart
- Two normal lactate dehydrogenase levels at least 4 weeks apart
- Willing, committed, and able to return for all clinic visits and complete all study-related procedures
- 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)
Who cannot join
- Any prior eculizumab treatment discontinuation
- On chronic dialysis (defined as ≥3 months on dialysis)
- Currently participating in another complement inhibitor trial
- Life expectancy of <6 months
- 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)
Trial locations and contacts
24 sites in 4 countries.
24 sites in 4 countries
Australia11 sites
Clinical Trial Site
Adelaide, 5000
Clinical Trial Site
Clayton, 3168
Clinical Trial Site
Heidelberg, 3084
Clinical Trial Site
Kingswood, 2747
Clinical Trial Site
Liverpool, 2170
Clinical Trial Site
Nedlands, 6109
Clinical Trial Site
Parkville, 3050
Clinical Trial Site
Parkville, 3052
Clinical Trial Site
Perth, 6008
Clinical Trial Site
Westmead, 2145
Clinical Trial Site
Woolloongabba, 4102
United States8 sites
Clinical Trial Site
Atlanta, Georgia, 30322
Clinical Trial Site
Boston, Massachusetts, 02115
Clinical Trial Site
Charlotte, North Carolina, 28203
Clinical Trial Site
Chicago, Illinois, 60611
Clinical Trial Site
Columbus, Ohio, 43210
Clinical Trial Site
Hackensack, New Jersey, 07601
Clinical Trial Site
Houston, Texas, 77030
Clinical Trial Site
Washington D.C., District of Columbia, 20007
Germany4 sites
Clinical Trial Site
Hannover, 30625
Clinical Trial Site
Hanover, 30625
Clinical Trial Site
Kiel, 24105
Clinical Trial Site
Lübeck, 23538
About this study
Official title: Evidence - Evaluation of Potential Predictors of Disease Progression in Patients With aHUS, Including Genetics, Biomarkers and Treatment
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.
- Conditions
- Atypical Hemolytic Uremic Syndrome
- Being tested
- Eculizumab
- Medical subject headings
- Atypical Hemolytic Uremic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EculizumabOther
Also called Soliris®
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.
| Study group | Role | What they receive |
|---|---|---|
| Eculizumab | The targeted population consists of pediatric and adult participants with aHUS who received eculizumab at the discretion of the treating physician. Receives: Other: Eculizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, 24 Months
Secondary outcomes (2)
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.
Measured: Baseline, 24 Months
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.
Measured: Baseline, 24 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2015 (estimated)
- Study start4 Nov 2015
- Primary completion5 Oct 2017
- Study completion5 Oct 2017
Why it stopped: Terminated for administrative reasons before enrollment was complete.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Atypical Hemolytic Uremic SyndromeStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 67 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02614898
- Sponsor's protocol ID
- ECU-aHUS-403
- Other IDs
- 2015-003135-35, 2015
- Acronym
- EVIDENCE
- Status last verified
- Dec 2019
- First submitted
- 24 Nov 2015
- Last updated
- 2 Jan 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evidence - Evaluation of Potential Predictors of Disease Progression in Patients With aHUS, Including Genetics, Biomarkers and Treatment
- ClinicalTrials.gov ID
- NCT02614898
- Acronym / sponsor ID
- EVIDENCE / ECU-aHUS-403
- Conditions
- Atypical Hemolytic Uremic Syndrome
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Terminated for administrative reasons before enrollment was complete.
- Enrolment
- 67 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Nov 2015 (actual)
- Primary completion
- 5 Oct 2017 (actual)
- Study completion
- 5 Oct 2017 (actual)
- First posted
- 25 Nov 2015 (estimated)
- Results
- Posted 2 Jan 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eculizumab | – | Other: EculizumabThe targeted population consists of pediatric and adult participants with aHUS who received eculizumab at the discretion of the treating physician. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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).
Key inclusion criteria (5 listed)
- Currently receiving eculizumab treatment in the M11-001 aHUS Registry
- Two normal platelet counts at least 4 weeks apart
- Two normal lactate dehydrogenase levels at least 4 weeks apart
- Willing, committed, and able to return for all clinic visits and complete all study-related procedures
- 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)
Key exclusion criteria (5 listed)
- Any prior eculizumab treatment discontinuation
- On chronic dialysis (defined as ≥3 months on dialysis)
- Currently participating in another complement inhibitor trial
- Life expectancy of <6 months
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EculizumabAlso: Soliris®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. | Other | Eculizumab |
Primary outcome
Rate Of Thrombotic Microangiopathy (TMA) Manifestations During Eculizumab Treatment Compared To Off-Treatment
Time frame: Baseline, 24 Months
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.
2 secondary outcomes
Change From Baseline To 24 Months In Estimated Glomerular Filtration Rate (eGFR)
Time frame: Baseline, 24 Months
Incidence Of Plasma Exchange And Plasma Infusion (PE/PI)
Time frame: Baseline, 24 Months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jan 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
24 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Atypical Hemolytic Uremic Syndrome, run by Alexion Pharmaceuticals, Inc. at 24 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 2 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in Australia, United States, Germany, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Jan 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate Of Thrombotic Microangiopathy (TMA) Manifestations During Eculizumab Treatment Compared To Off-Treatment (time frame: Baseline, 24 Months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jan 2020.