A Study of PRX004 in Subjects With Amyloid Transthyretin (ATTR) Amyloidosis

Summary

A Phase 1, open-label study of intravenous (IV) PRX004 as a single agent in subjects with hereditary amyloid transthyretin (hATTR) amyloidosis. The study will consist of 3 phases and will enroll up to a total of 36 subjects. A 3+3 dose escalation component to determine the safety, tolerability, PK, PD, and MTD. An expansion component in anticipated PRX004 RP2D cohorts selected from the Dose Escalation Phase. An extended dosing component for eligible subjects from the Dose Escalation or Expansion phases.

Timeline

Start

2018-04-05

Primary completion

2020-07-23

Completion

2020-07-23

Outcome

Outcome not reported

Stopped: “Because of the impact of COVID-19 pandemic”

Publications

• Capustin M, Frishman WH. Transthyretin Cardiac Amyloidosis and Novel Therapies to Treat This Not-so-rare Cause of Cardiomyopathy. Cardiol Rev. 2021 Sep-Oct 01;29(5):263-273. doi: 10.1097/CRD.0000000000000387.

Drugs

Indications