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

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Brief Title

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

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

Brief 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.

Detailed 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.

Study Phase

Phase 1

Study Type


Primary Outcome

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

Secondary Outcome

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


Myotonic Dystrophy, Type 1 (DM1)



Study Arms / Comparison Groups

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


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

June 17, 2019

Completion Date

April 30, 2020

Primary Completion Date

March 31, 2020

Eligibility 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

          -  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




18 Years - 65 Years

Accepts Healthy Volunteers



Elliot Ehrich, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Responsible Party


Study Sponsor

Expansion Therapeutics, Inc.

Study Sponsor

Elliot Ehrich, MD, Study Director, Chief Medical Officer

Verification Date

May 2021