Trial Search Results

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

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.

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

Expansion Therapeutics, Inc.

Stanford Investigator(s):


  • Drug: ERX-963
  • Drug: Placebo


Phase 1


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

Ages Eligible for Study

18 Years - 65 Years

Genders Eligible for Study


Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Mitchell Reddan
Not Recruiting