Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1

Myotonic Dystrophy Clinical Trial

Official title: Double-Blind, Placebo-Controlled, Dose-Range-Finding, Crossover Trial of Single Day Administration of ERX-963 in Adults With Myotonic Dystrophy Type 1

Status Completed

Clinical Trial Summary

Participants in this study will receive two treatments, placebo and ERX-963, on different days in a randomized fashion.

The primary purpose of this study is to investigate the safety and tolerability of ERX-963 in participants diagnosed with Myotonic Dystrophy, Type 1 (DM1).

The secondary purpose is to evaluate the potential of ERX-963 treatment to reduce excessive daytime sleepiness / hypersomnia and improve cognitive function in DM1 participants compared to placebo treatment.

Clinical Trial Description

This study is evaluating single administration of two dose levels of ERX-963 to explore the relationship between dose, safety, tolerability, exposure and clinical benefit. This is a multi-center, randomized, double-blind, placebo-controlled, two-treatment period crossover study in two cohorts of participants with DM1.

Participants who have consented and meet eligibility criteria will receive two treatments, placebo and ERX-963, in a randomized crossover fashion with a washout period between the treatments. On treatment days, participants will receive treatment followed by repeated blood collection for pharmacokinetic analysis and administration of a battery of outcome measures relevant to sleep and cognition. ;

Related Conditions & MeSH terms

• Myotonic Dystrophy
• Myotonic Dystrophy, Type 1 (DM1)

• NCT number: NCT03959189
• Study type: Interventional
• Source: Expansion Therapeutics, Inc.
• Status: Completed
• Phase: Phase 1
• Start date: June 17, 2019
• Completion date: April 30, 2020