Clinical trial NCT03959189: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Myotonic Dystrophy, Type 1 (DM1)
Myotonic Dystrophy
Keywords: Excessive Daytime Sleepiness
hypersomnia
Lead sponsor: Expansion Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2019-05-22
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Incidence of Adverse Events, Serious Adverse Events, and Drug-related Adverse Events [Safety and Tolerability] After a Single Dose of ERX-963 vs. Placebo
timeFrame: Adverse Events were collected from screening to the End of Study Visit, up to 57 days
Secondary outcomes:
measure: Assess the Effect of ERX-963 on the Stanford Sleepiness Scale Score Compared to the Effect of Placebo
timeFrame: From dosing to approximately 2 hours
measure: Assess the Effect of ERX-963 on the Change in Patient Global Impression - Improvement Scale (PGI-I) Compared to Placebo
timeFrame: Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
measure: Assess the Effect of ERX-963 on the Clinical Global Impressment - Improvement (CGI-I) Scale Compared to Placebo
timeFrame: Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
measure: Assess the Effect of ERX-963 on the Psychomotor Vigilance Task (PVT)
timeFrame: From dosing to approximately 2 hours
measure: Assess the Effect of ERX-963 on the One-back Task
timeFrame: From dosing to approximately 2 hours
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford Neurosciences Health Center
state: California
city: Miami
country: United States
facility: Sleep Medicine Specialists of South Florida
state: Florida
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Chevy Chase
country: United States
facility: The Center for Sleep & Wake Disorders
state: Maryland
Study officials:
affiliation: Chief Medical Officer
name: Elliot Ehrich, MD
role: STUDY_DIRECTOR
References: