Phase 1
N=12
Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
Myotonic Dystrophy, Type 1 (DM1) · Myotonic Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT03959189 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Jun 2021
Primary outcome: Primary: Incidence of Adverse Events, Serious Adverse Events, and Drug-related Adverse Events [Safety and Tolerability] After a Single Dose of ERX-963 vs. Placebo — 0; 0; 1; 1 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- ERX-963 (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Expansion Therapeutics, Inc.
- Primary completion
- Mar 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Adverse Events, Serious Adverse Events, and Drug-related Adverse Events [Safety and Tolerability] After a Single Dose of ERX-963 vs. Placebo |
0; 0; 1; 1; 0; 0 | — |
| SECONDARY Assess the Effect of ERX-963 on the Stanford Sleepiness Scale Score Compared to the Effect of Placebo |
3.4; 3.4; 4.0; 4.2; 3.0; 2.7 | 0.7746 |
| SECONDARY Assess the Effect of ERX-963 on the Change in Patient Global Impression - Improvement Scale (PGI-I) Compared to Placebo |
3.4; 3.4; 4.0; 4.2 | — |
| SECONDARY Assess the Effect of ERX-963 on the Clinical Global Impressment - Improvement (CGI-I) Scale Compared to Placebo |
3.7; 3.9; 4.0; 4.4 | — |
| SECONDARY Assess the Effect of ERX-963 on the Psychomotor Vigilance Task (PVT) |
— | — |
| SECONDARY Assess the Effect of ERX-963 on the One-back Task |
— | — |
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.
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 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
Data sourced from ClinicalTrials.gov (NCT03959189). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.