The Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
1 other identifier
interventional
2
1 country
2
Brief Summary
Protocol is intended to characterize the overall safety and tolerability of eculizumab in this population.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started May 2012
Shorter than P25 for phase_2
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
May 16, 2012
CompletedFirst Submitted
Initial submission to the registry
December 4, 2012
CompletedFirst Posted
Study publicly available on registry
December 31, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 24, 2013
CompletedStudy Completion
Last participant's last visit for all outcomes
September 25, 2013
CompletedMarch 10, 2017
March 1, 2017
1.4 years
December 4, 2012
March 9, 2017
Conditions
Outcome Measures
Primary Outcomes (1)
Adverse events and serious adverse events and their severity and relationship to the drug
12 weeks
Study Arms (1)
ECULIZUMAB
OTHERInterventions
Eligibility Criteria
You may qualify if:
- 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:
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (2)
Nagano Red Cross Hospital
Nagano, Japan
Tokyo Medical and Dental University Hospital
Tokyo, Japan
Related Publications (1)
Pugh D, O'Sullivan ED, Duthie FA, Masson P, Kavanagh D. Interventions for atypical haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2021 Mar 23;3(3):CD012862. doi: 10.1002/14651858.CD012862.pub2.
PMID: 33783815DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 4, 2012
First Posted
December 31, 2012
Study Start
May 16, 2012
Primary Completion
September 24, 2013
Study Completion
September 25, 2013
Last Updated
March 10, 2017
Record last verified: 2017-03