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

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

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

ANZCTR

Registry ID

ACTRN12611000461998

Enrollment

Registered

2011-05-04

Start date

2011-07-01

Completion date

Unknown

Last updated

2020-01-13

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 severely affected aHUS patients.

Interventions

Eculizumab 30mL vials with a solution concentraion of 10mg/mL. Given by intravenous infusion over 1-4 hours with the dose depending on body weight. Dosing is weekly infusion for 4 weeks and then IV infusion every 2 weeks. There will be 26 weeks of treatment to meet the study defined endpoints. Patients will be allowed to continue to receive the drug for 2 more year or until the product is registred in Australia unless the study is prematurely discontinued.

Study design

Allocation

Non-randomised trial

Intervention model

Single group

Primary purpose

Treatment

Masking

Open (masking not used)

Eligibility

Sex/Gender

All

Age

1 Months to 18 Years

Healthy volunteers

Inclusion criteria

Signed informed consent. Patients from 1 month to 18 years diagnosed with aHUS

Exclusion criteria

Typical hemolytic-uremic syndrome (HUS), malignancy within 5 years of screening, HUS related to infection or bone marrow transplant or vitamin B12 deficiency, systemic lupus erythematosus (SLE), meningococcal/pneumococcal disease history.

Outcome results