Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
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|ClinicalTrials.gov Identifier: NCT03959189|
Recruitment Status : Recruiting
First Posted : May 22, 2019
Last Update Posted : June 3, 2019
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)|
|Estimated 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|
|Estimated Study Start Date :||May 2019|
|Estimated Primary Completion Date :||December 2019|
|Estimated Study Completion Date :||December 2019|
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 40 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03959189
|Contact: Anita Seto, PhDemail@example.com|
|United States, California|
|Stanford Neurosciences Health Center||Recruiting|
|Palo Alto, California, United States, 94305|
|Contact: Mitchell Reddan 650-725-4341 firstname.lastname@example.org|
|Principal Investigator: Jacinda Sampson, MD, PhD|
|United States, Iowa|
|University of Iowa||Not yet recruiting|
|Iowa City, Iowa, United States, 52242|
|Contact: Nicole Kressin|
|Study Director:||Elliot Ehrich, MD||Chief Medical Officer|