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

Expansion Therapeutics, Inc.12 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Myotonic Dystrophy, Type 1 (DM1)Myotonic Dystrophy

Interventions

ERX-963DRUG

Active medicine

PlaceboDRUG

Comparator

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (4)

Stanford Neurosciences Health Center

Palo Alto, California, United States

Sleep Medicine Specialists of South Florida

Miami, Florida, United States

University of Iowa

Iowa City, Iowa, United States

The Center for Sleep & Wake Disorders

Chevy Chase, Maryland, United States

Outcomes

Primary Outcomes

Incidence of Adverse Events, Serious Adverse Events, and Drug-related Adverse Events [Safety and Tolerability] After a Single Dose of ERX-963 vs. Placebo

An adverse event (AE) was defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study intervention. Treatment-emergent AEs were AEs which started between the date and time of study drug dosing and through Study Day 2, within each period. Drug-related AEs were assessed by the investigator to determine the relationship (related or unrelated) between the study intervention and each AE occurrence.

Time frame: Adverse Events were collected from screening to the End of Study Visit, up to 57 days

Secondary Outcomes

Assess the Effect of ERX-963 on the Stanford Sleepiness Scale Score Compared to the Effect of Placebo

Participants will self-report their level of sleepiness by self-rated questionnaire "Stanford Sleepiness Scale" (SSS). This is a single item questionnaire on a 7-point scale (1-7). Higher values indicate worse outcome.

Time frame: From dosing to approximately 2 hours

Assess the Effect of ERX-963 on the Change in Patient Global Impression - Improvement Scale (PGI-I) Compared to Placebo

The PGI-I is a 7-point rating system used by the patient to rate their overall clinical condition after intervention relative to before intervention where 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse, and 7=very much worse.

Time frame: Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.

Assess the Effect of ERX-963 on the Clinical Global Impressment - Improvement (CGI-I) Scale Compared to Placebo

Data from ClinicalTrials.gov

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