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

Expansion Therapeutics

Status and phase: Completed; Phase 1

Conditions: Myotonic Dystrophy, Type 1 (DM1); Myotonic Dystrophy

Treatments: Drug: Placebo; Drug: ERX-963

Study type: Interventional

Funder types: Industry

Identifiers

NCT03959189

ERX-963-001

Details and patient eligibility

About

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.

Full 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.

Enrollment: 12 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Double Blind

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12 participants in 2 patient groups

ERX-963 then placebo

Experimental group

Description:

Participants in this arm will receive ERX-963 followed by a washout period. After the washout period, participants will receive placebo.

Treatment:

Drug: ERX-963

Placebo then ERX-963

Experimental group

Description:

Participants in this arm will receive placebo followed by a washout period. After the washout period, participants will receive ERX-963.

Treatment:

Drug: Placebo