Study to evaluate if eculizumab is efficient and safe enough to be used for treatment of adults with atypical hemolytic-uremic syndrome

Adult patients testing eculizumab for atypical hemolytic-uremic syndrome for safety and efficacy

Status

Terminated

Phases

Phase 2

Study type

Interventional

Source

ANZCTR

Registry ID

ACTRN12611000498998

Enrollment

Registered

2011-05-12

Start date

2011-07-01

Completion date

Unknown

Last updated

2026-02-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief summary

Atypical hemolytic-uremic syndrome is a serious, life-threatening rare and chronic disease believed to be caused by genetic mutations. Current treatment for the disease is inadequate. Due to the uncontrolled complement activation seen in aHUS patients and the previously shown activity of eculizumab to selectively inhibit terminal complement activation, it has been decided to look in to the use of eculizumab in the treatment of serverely affected aHUS patients.

Interventions

Eculizumab 900mg administered intravenously weekly for 4 weeks via infusion over 30 minutes then 1200mg intravenous infusion every two weeks for 26 weeks with the possibility of continued treatment for up to 2 years or until marketing approval.

Study design

Allocation

Non-randomised trial

Intervention model

Single group

Primary purpose

Treatment

Masking

Open (masking not used)

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

Patients must be willing to give written informed consent. Patients must be over 18 years of age with a diagnosis of atyipcal hemolytic-uremic syndrome. Patients exhibit thrombocytopenia, hemolysis and elevated serum creatinine.

Exclusion criteria

Chronic dialysis. Prior eculizumab use or hypersensitivity to eculizumab, to murine proteins or to one of the excipients.

Outcome results