TMAs are a group of diseases in which thrombosis occurs in small blood vessels as a result of damage to the endothelium (lining) of the vessels. This leads to hemolytic anemia, low platelet count (thrombocytopenia), end organ damage which may result in complications including renal failure, stroke and pulmonary hypertension.
There are no currently approved drugs for the treatment of HSCT-TMA and, untreated, the condition in its more severe forms has a high risk of death. TMA-HSCT is an orphan condition with an estimated fatality rate of more than 80% in pediatric patients with the disease.
A pivotal trial design for pediatric TMA-HSCT patients was agreed with the FDA in which the response to nomacopan of selected, clinically meaningful treatment variables would be the primary endpoint.
- Part A of the trial is a dose confirmation study.
- Part B of the trial is a single arm responder-based efficacy study that will follow an interim analysis of Part A and a meeting with the FDA.
Clinical site activations for this study were initially delayed by the COVID-19 pandemic; however as of Q1 2021, several clinical sites had been opened for recruitment in both the US and Europe.