Overview

Complement Inhibition in aHUS Dialysis Patients

Status:
Terminated

Trial end date:
2017-07-13

Target enrollment:
0

Participant gender:
All

Summary

This study evaluates the effect of CCX168, a C5aR Antagonist, Oral Administration on Ex Vivo Thrombus Formation and Disease Activity in ten patients with diagnosis of Atypical Hemolytic Uremic Syndrome with or without genetic abnormalities in the complement system or thrombomodulin, on stable chronic extracorporeal or peritoneal dialysis therapy since at least 6 months.

Phase:

Phase 2

Accepts Healthy Volunteers?

Accepts Healthy Volunteers

Details

Lead Sponsor:

Mario Negri Institute for Pharmacological Research

Collaborator:

ChemoCentryx

Criteria

Inclusion Criteria:

- Age >18 years;

- Diagnosis of aHUS with or without identified genetic abnormalities in the complement
system or thrombomodulin;

- Stable chronic extracorporeal or peritoneal dialysis therapy since at least 6 months;

- Written informed consent.

Exclusion Criteria:

- Women of childbearing potential or women who are breastfeeding;

- Shiga toxin-associated HUS or secondary forms of thrombotic microangiopathy;

- ADAMTS13 activity <10 % or circulating anti ADAMTS13 autoantibodies consistent with
the diagnosis of thrombotic thrombocytopenic purpura;

- Need for specific intervention with plasma therapy and/or complement inhibitors as
deemed clinically appropriate;

- Plasma therapy or treatment with complement inhibitors or antiplatelet and
antithrombotic agents over the last two weeks;

- Liver function impairment (serum liver enzymes or bilirubin levels >3 x upper limit of
normal);

- Neutrophil count < 2000/μL or lymphocyte count < 1000/μL;

- Infection requiring antibiotic treatment within the previous 4 weeks prior to
screening;

- Participated in any clinical study of an investigational product within 30 days prior
to screening or within 5 half-lives after taking the last dose;

- History or presence of any medical condition or disease which, in the opinion of the
Investigator may place the subject at unacceptable risk for study participation;

- Inability to understand the potential risks and benefits of the study;

- Legal incapacity.