Clinical Trials

The aim of this project is to evaluate whether kidney biopsy tissue will stain positive for C3d under immunofluorescence in patients who had renal thrombotic microangiopathy (TMA) caused by atypical hemolytic uremic syndrome (aHUS) and to study if that could offer a diagnostic value.

This is a Phase 2, uncontrolled, three-stage, ascending-dose-escalation study in subjects with one of three forms of TMA: atypical hemolytic uremic syndrome (aHUS), thrombotic thrombocytopenic purpura (TTP), or hematopoietic stem cell transplant (HSCT)-associated TMA. In the first stage, OMS721 will be administered to escalating dose cohorts of three subjects per cohort to identify the optimal dosing regimen. After enrollment and treatment of each cohort there will be a safety review to determine whether dose escalation should proceed. In the second stage, the dose selected in the first stage will be administered to expanded cohorts of 40 subjects per cohort with ...

The goal of this study is to validate testing to be performed in the clinical immunology lab and have a source of positive patient sera.

These tests include:

1. Auto antibodies to Factor H
2. C3 Nephritic Factor
3. Auto antibodies to C1q
4. Auto antibodies to C1INH

The purpose of this study is to develop and validate full gene-sequencing assays or known mutation sequencing assays for the evaluation of patients with suspected alternative pathway complement defects.