The Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS)

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01757431
• Date of registration: 04/12/2012
• Prospective Registration: No
• Primary sponsor: Alexion Pharmaceuticals
• Public title: The Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
• Date of first enrolment: May 16, 2012
• Target sample size: 2
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01757431
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Japan

Key inclusion & exclusion criteria

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

Exclusion Criteria:

Shiga-toxin producing E. coli-HUS (STEC-HUS; shiga-toxin and/or STEC positive) History of
malignancy within 5 years of screening Known human immunodeficiency virus (HIV) infection
Identified drug exposure-related HUS Infection-related HUS HUS related to bone marrow
transplant (BMT) HUS related to vitamin B12 deficiency Known Systemic Lupus Erythematosus
(SLE) or antiphospholipid antibody positivity or syndrome Chronic dialysis (defined as
dialysis on a regular basis as renal replacement therapy for end-stage renal disease
(ESRD)) Patients with a confirmed diagnosis of sepsis defined as positive blood cultures
within 7 days of the screening visit and not treated with antibiotics to which the organism
is sensitive Presence or suspicion of active and untreated systemic bacterial infection
that, in the opinion of the Investigator confounds an accurate diagnosis of aHUS or impedes
the ability to manage the aHUS disease Pregnancy or lactation Unresolved systemic
meningococcal disease Any medical or psychological condition that, in the opinion of the
investigator, could increase patient's risk by participating in the study or confound the
outcome of the study Patients receiving chronic intravenous immunoglobulin (IVIG) within 8
weeks unless for unrelated medical condition (e.g., hypogammaglobulinemia) or chronic
rituximab therapy within 12 weeks of the screening visit Patients receiving other
immunosuppressive therapies such as steroids, calcineurin inhibitors (mTOR), (e.g.,
cyclosporine or tacrolimus) are excluded unless: [1] part of an established post-transplant
anti-rejection regimen, or [2] patient has confirmed anti-Complement Factor Antibodies
requiring immunosuppressive therapy or [3] steroids are used for a condition other than
aHUS (e.g., asthma) Prior eculizumab use, hypersensitivity to eculizumab, to murine
proteins or to one of the excipients Inclusion in any other investigational intervention
trial except this study



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Atypical Hemolytic Uremic Syndrome (aHUS)

Intervention(s)

Drug: Eculizumab

Primary Outcome(s)

Adverse events and serious adverse events and their severity and relationship to the drug [Time Frame: 12 weeks]

Secondary ID(s)

C11-005J

Source(s) of Monetary Support

Please refer to primary and secondary sponsors