- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01757431
The Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
-
-
-
Nagano, Japan
- Nagano Red Cross Hospital
-
Tokyo, Japan
- Tokyo Medical and Dental University Hospital
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- All patients with diagnosis of aHUS who have been receiving eculizumab by personal importation (specific eligibility criteria below do not apply) Or,
Patients with current clinical manifestations of aHUS who meet the following criteria:
- 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
- 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)
- Patient at least 1 month of age and body weight ≥5 kg
- Platelet count at screening < lower limit of normal (LLN)
- 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
- Serum Creatinine (SrCr) level ≥ ULN at screening (patient requiring dialysis for acute renal failure also eligible)
- 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
- 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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Other: ECULIZUMAB
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Adverse events and serious adverse events and their severity and relationship to the drug
Time Frame: 12 weeks
|
12 weeks
|
Collaborators and Investigators
Sponsor
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Kidney Diseases
- Urologic Diseases
- Disease
- Hematologic Diseases
- Anemia
- Thrombocytopenia
- Blood Platelet Disorders
- Anemia, Hemolytic
- Thrombotic Microangiopathies
- Uremia
- Syndrome
- Hemolysis
- Hemolytic-Uremic Syndrome
- Atypical Hemolytic Uremic Syndrome
- Physiological Effects of Drugs
- Immunosuppressive Agents
- Immunologic Factors
- Complement Inactivating Agents
- Eculizumab
Other Study ID Numbers
- C11-005J
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Atypical Hemolytic Uremic Syndrome (aHUS)
-
Alexion PharmaceuticalsCompletedATYPICAL HEMOLYTIC UREMIC SYNDROME (AHUS)Japan
-
AstraZenecaRecruitingAtypical Hemolytic Uremic Syndrome(aHUS)China
-
Alexion Pharmaceuticals, Inc.CompletedAtypical Hemolytic Uremic Syndrome (aHUS)United States, France, Spain, Taiwan, United Kingdom, Australia, Belgium, Germany, Italy, Japan, Korea, Republic of, Russian Federation, Austria, Canada
-
AlexionCompletedAtypical Hemolytic Uremic Syndrome (aHUS)United States, France, Germany, Spain, Switzerland, Netherlands, Canada, Austria
-
Alexion Pharmaceuticals, Inc.CompletedAtypical Hemolytic Uremic Syndrome (aHUS)Italy, United States, Korea, Republic of, Spain, Germany, Belgium, United Kingdom
-
AO GENERIUMCompletedAtypical Hemolytic Uremic Syndrome | aHUSRussian Federation
-
NovelMed TherapeuticsNot yet recruitingaHUS - Atypical Hemolytic Uremic Syndrome
-
AlexionCompletedAtypical Hemolytic-Uremic SyndromeUnited Kingdom, United States, France, Italy, Germany, Spain, Belgium
-
Alexion PharmaceuticalsCompletedAtypical Hemolytic-Uremic SyndromeUnited Kingdom, Belgium, United States, Germany, Italy, France, Netherlands, Australia, Canada
-
University Hospital, ToursHospices Civils de Lyon; University Hospital, Strasbourg, France; University... and other collaboratorsActive, not recruitingHemolytic-Uremic Syndrome, AtypicalFrance
Clinical Trials on Eculizumab
-
Samsung Bioepis Co., Ltd.CompletedParoxysmal Nocturnal HemoglobinuriaKorea, Republic of, Taiwan, Malaysia, Thailand, Romania, India, Mexico, Ukraine
-
AlexionCompletedAtypical Hemolytic Uremic SyndromeCanada, United Kingdom, France, Germany, Netherlands, Sweden, Italy
-
Alexion PharmaceuticalsCompletedHemoglobinuria, ParoxysmalUnited States, Australia, Belgium, Canada, Germany, Italy, United Kingdom, France, Ireland, Netherlands, Sweden, Switzerland
-
Nantes University HospitalCompletedAtypical Hemolytic Uremic SyndromeFrance
-
AlexionApproved for marketingHemoglobinuria, ParoxysmalUnited States
-
AlexionCompletedAllergic AsthmaCanada
-
Alexion PharmaceuticalsCompletedNeuromyelitis Optica | Neuromyelitis Optica Spectrum DisorderHong Kong, Korea, Republic of, United States, Croatia, Australia, Canada, Malaysia, Japan, Italy, Turkey, Spain, Taiwan, Argentina, Colombia, Czechia, Denmark, Germany, Russian Federation, Thailand, United Kingdom
-
Alexion PharmaceuticalsNo longer availableCOVID-19 | Pneumonia, Viral | Acute Lung Injury/Acute Respiratory Distress Syndrome (ARDS)United States, France
-
Alexion PharmaceuticalsCompletedAtypical Hemolytic Uremic SyndromeCanada, United Kingdom, France, Germany, Sweden, Netherlands, Italy
-
Alexion PharmaceuticalsCompletedHemoglobinuria, ParoxysmalUnited States, United Kingdom, Australia, Belgium, Canada, France, Germany, Ireland, Italy, Netherlands, Spain, Sweden, Switzerland