Atypical Hemolytic Uremic Syndrome (aHUS)
Conditions
Brief summary
Protocol is intended to characterize the overall safety and tolerability of eculizumab in this population.
Interventions
DRUGEculizumab
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
1. All patients with diagnosis of aHUS who have been receiving eculizumab by personal importation (specific eligibility criteria below do not apply) Or, 2. Patients with current clinical manifestations of aHUS who meet the following criteria: 1. Patient with diagnosis of aHUS with or without an identified complement regulatory protein genetic abnormality or anti-complement factor antibody and for whom other known etiologies of hemolytic uremic syndrome (HUS) have been ruled out as confirmed in the
Exclusion criteria
2. Patient (and legal guardian if patient is not an adult) willing and able to give written informed consent and assent (or verbal assent if patient is unable to read or write) 3. Patient at least 1 month of age and body weight ≥5 kg 4. Platelet count at screening \< lower limit of normal (LLN) 5. Signs or symptoms of hemolysis (i.e., lactate dehydrogenase (LDH) ≥ 1.5x upper limit of normal (ULN) and Hemoglobin ≤ LLN) at start of current aHUS event 6. Serum Creatinine (SrCr) level ≥ ULN at screening (patient requiring dialysis for acute renal failure also eligible) 7. Female patient of childbearing potential practicing an effective, reliable and medically approved contraceptive regimen during the entire duration of the study, including the Follow-up Period. At the time of the last follow-up visit, patient must agree to continue to use adequate contraception methods for up to 5 months following discontinuation of eculizumab treatment 8. Able and willing to comply with study procedures
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse events and serious adverse events and their severity and relationship to the drug | 12 weeks |
Countries
Japan
Outcome results
None listed