Status:
WITHDRAWN
A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome
Lead Sponsor:
Alnylam Pharmaceuticals
Conditions:
Atypical Hemolytic Uremic Syndrome
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with aHUS.
Eligibility Criteria
Inclusion
- Willing to provide written informed consent and to comply with the study requirements
- Age 18 years or older
- Clinical diagnosis of primary aHUS
- Clinical thrombotic microangiopathy (TMA) activity
- Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception
- Previously vaccinated with meningococcal group ACWY conjugate vaccine and meningococcal group B vaccine or willingness to receive these vaccinations
- ADAMTS13 \>10% or other proven aHUS-associated mutation
Exclusion
- Clinically significant abnormal laboratory results
- Positive Shiga toxin producing Escherichia coli test at Screening
- Suspected secondary aHUS, in the opinion of the Investigator (unless there is a documented aHUS-associated genetic mutation)
- Positive direct Coombs test
- Patients who have received hemodialysis for \>3 months
- Bone marrow transplant recipients
- Organ transplant recipients, except for kidney transplant recipients with primary aHUS (confirmed by known genetic mutation and kidney biopsy)
- Known history or evidence of systemic lupus erythematosus or antiphospholipid antibody syndrome
- History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc
- Malignancy (except for non-melanoma skin cancers, cervical in-situ carcinoma, breast ductal carcinoma in situ, or stage 1 prostate carcinoma) within the last 5 years
- Patients with a poor prognosis that is expected to limit their life expectancy to less than 3 months, in the opinion of the Investigator
Key Trial Info
Start Date :
September 19 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 12 2018
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT03303313
Start Date
September 19 2017
End Date
September 12 2018
Last Update
October 1 2018
Active Locations (12)
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1
Clinical Trial Site
Sarajevo, Bosnia and Herzegovina
2
Clinical Trial Site
Calgary, Canada, T2N 2T9
3
Clinical Trial Site
Tallinn, Estonia
4
Clinical Trial Site
Tartu, Estonia