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

• Status: Completed
• Trial end date: 2020-04-30
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Expansion Therapeutics, Inc.

Criteria

Key Inclusion Criteria:

• 18 to 65 years of age

• DM1 defined by genetic testing or clinical-confirmation

• Epworth Sleepiness Scale (ESS) of > 11 or participants who have long sleep periods of an average of > 10 hours a day

• Age of onset of DM1 greater than 16 years

Key Exclusion Criteria:

• Significant respiratory compromise

• Significant cardiac disease

• Diagnosis of symptomatic Restless Leg Syndrome or significant untreated nocturnal hypoxias

• Significant moderate to severe hepatic insufficiency

• Clinically active depression, anxiety, or other medical condition that, in the investigator's opinion, would interfere with the safety and efficacy assessments

• History of seizures

• History of panic disorders