Treatment of an Inherited Ventricular Arrhythmia

Summary

The goal of the proposed project is to determine the safety and tolerability as well as the preliminary efficacy of a novel small molecule drug, S48168 (ARM210), for the treatment of Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT1). This disease is associated with fatal changes in heart rhythms leading to sudden death with exercise or excessive excitement. It is due to mutations in the Ryanodine Receptor calcium release channel, which cause leaky channels leading to the disease. S48168 (ARM210) repairs these leaky channels and can be a disease-modifying therapy restoring normal function to the channels. This result would allow patients with CPVT to live normal, active lives. Funding Source- FDA OOPD.

Timeline

Start

2023-08-01

Primary completion

2024-04-01

Completion

2024-04-01

Outcome

Outcome not reported

Stopped (Enrollment): “Due to challenges in recruiting patients.”

Drugs

Indications