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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified March 2012 by Alexion Pharmaceuticals.
Recruitment status was  Recruiting
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01757431
First received: December 4, 2012
Last updated: December 21, 2012
Last verified: March 2012
  Purpose

Protocol is intended to characterize the overall safety and tolerability of eculizumab in this population.


Condition Intervention Phase
Atypical Hemolytic Uremic Syndrome (aHUS)
Drug: Eculizumab
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • Adverse events and serious adverse events and their severity and relationship to the drug [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 5
Study Start Date: May 2012
Estimated Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
ECULIZUMAB Drug: Eculizumab

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01757431

Locations
Japan
Nagano Red Cross Hospital Recruiting
Nagano, Japan
Contact: Alexion Pharmaceuticals, Inc       clinicaltrials@alxn.com   
Tokyo Medical and Dental University Hospital Recruiting
Tokyo, Japan
Contact: Alexion Pharmaceuticals, Inc       clinicaltrials@alxn.com   
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

No publications provided

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01757431     History of Changes
Other Study ID Numbers: C11-005J
Study First Received: December 4, 2012
Last Updated: December 21, 2012
Health Authority: Japan: Pharmaceuticals and Medical Devices Agency

Additional relevant MeSH terms:
Azotemia
Hemolysis
Hemolytic-Uremic Syndrome
Syndrome
Anemia
Anemia, Hemolytic
Blood Platelet Disorders
Disease
Hematologic Diseases
Kidney Diseases
Pathologic Processes
Thrombocytopenia
Thrombotic Microangiopathies
Uremia
Urologic Diseases

ClinicalTrials.gov processed this record on October 29, 2014