Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03959189|
Recruitment Status : Completed
First Posted : May 22, 2019
Last Update Posted : May 13, 2020
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.
|Condition or disease||Intervention/treatment||Phase|
|Myotonic Dystrophy, Type 1 (DM1) Myotonic Dystrophy||Drug: ERX-963 Drug: Placebo||Phase 1|
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||12 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Double (Participant, Investigator)|
|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|
|Actual Study Start Date :||June 17, 2019|
|Actual Primary Completion Date :||March 31, 2020|
|Actual Study Completion Date :||April 30, 2020|
Experimental: ERX-963 then placebo
Participants in this arm will receive ERX-963 followed by a washout period. After the washout period, participants will receive placebo.
Experimental: Placebo then ERX-963
Participants in this arm will receive placebo followed by a washout period. After the washout period, participants will receive ERX-963.
- Incidence of Adverse Events, Serious Adverse Events, and drug-related Adverse Events [Safety and Tolerability] after a single dose of ERX-963 vs. placebo [ Time Frame: up to 57 days ]Percentage of participants with Adverse Events, Serious Adverse Events, and drug-related Adverse Events
- Assess the effect of ERX-963 on the Stanford Sleepiness Scale score [ Time Frame: From dosing to approximately 2 hours ]Participants will self-report their level of sleepiness by self-rated questionnaire "Stanford Sleepiness Scale" (SSS). This is a single item questionnaire on a 7-point scale (1-7). Higher values indicate worse outcome.
- Assess the effect of ERX-963 on the Psychomotor Vigilance Task (PVT) [ Time Frame: From dosing to approximately 2 hours ]Participants will be tested for their response time and number of lapses during the PVT.
- Assess the effect of ERX-963 on the One-back task [ Time Frame: From dosing to approximately 2 hours ]Participants will be tested for the proportion of correct response to the One-back task.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03959189
|United States, California|
|Stanford Neurosciences Health Center|
|Palo Alto, California, United States, 94305|
|United States, Florida|
|Sleep Medicine Specialists of South Florida|
|Miami, Florida, United States, 33126|
|United States, Iowa|
|University of Iowa|
|Iowa City, Iowa, United States, 52242|
|United States, Maryland|
|The Center for Sleep & Wake Disorders|
|Chevy Chase, Maryland, United States, 20815|
|Study Director:||Elliot Ehrich, MD||Chief Medical Officer|