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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 : October 24, 2019
Sponsor:
Information provided by (Responsible Party):
Expansion Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE May 16, 2019
First Posted Date  ICMJE May 22, 2019
Last Update Posted Date October 24, 2019
Actual Study Start Date  ICMJE June 17, 2019
Estimated Primary Completion Date December 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 18, 2019)
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
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03959189 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 21, 2019)
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: May 18, 2019)
  • 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.
  • 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.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
Official Title  ICMJE 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE
  • Myotonic Dystrophy, Type 1 (DM1)
  • Myotonic Dystrophy
Intervention  ICMJE
  • Drug: ERX-963
    Active medicine
  • Drug: Placebo
    Comparator
Study Arms  ICMJE
  • 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.
    Intervention: Drug: ERX-963
  • 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.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 18, 2019)
12
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2019
Estimated Primary Completion Date December 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • 18 to 60 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Anita Seto, PhD 858-764-4290 info@expansionrx.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03959189
Other Study ID Numbers  ICMJE ERX-963-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Expansion Therapeutics, Inc.
Study Sponsor  ICMJE Expansion Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Elliot Ehrich, MD Chief Medical Officer
PRS Account Expansion Therapeutics, Inc.
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP