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
Interventional
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
Condition
Myotonic Dystrophy, Type 1 (DM1)
Intervention
ERX-963
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.
Publications
* 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
Drug
Estimated Enrollment
12
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
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
Gender
All
Ages
18 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Elliot Ehrich, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT03959189
Organization ID
ERX-963-001
Responsible Party
Sponsor
Study Sponsor
Expansion Therapeutics, Inc.
Study Sponsor
Elliot Ehrich, MD, Study Director, Chief Medical Officer
Verification Date
May 2021