Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1

June 22, 2021 updated by: Expansion Therapeutics, Inc.

Double-Blind, Placebo-Controlled, Dose-Range-Finding, Crossover Trial of Single Day Administration of ERX-963 in Adults With Myotonic Dystrophy Type 1

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.

Study Overview

Status

Completed

Intervention / 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

Interventional

Enrollment (Actual)

12

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • California
      • Palo Alto, California, United States, 94305
        • Stanford Neurosciences Health Center
    • Florida
      • Miami, Florida, United States, 33126
        • Sleep Medicine Specialists of South Florida
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa
    • Maryland
      • Chevy Chase, Maryland, United States, 20815
        • The Center for Sleep & Wake Disorders

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.
Active medicine
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.
Comparator

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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: Adverse Events were collected from screening to the End of Study Visit, up to 57 days
An adverse event (AE) was defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study intervention. Treatment-emergent AEs were AEs which started between the date and time of study drug dosing and through Study Day 2, within each period. Drug-related AEs were assessed by the investigator to determine the relationship (related or unrelated) between the study intervention and each AE occurrence.
Adverse Events were collected from screening to the End of Study Visit, up to 57 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assess the Effect of ERX-963 on the Stanford Sleepiness Scale Score Compared to the Effect of Placebo
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.
From dosing to approximately 2 hours
Assess the Effect of ERX-963 on the Change in Patient Global Impression - Improvement Scale (PGI-I) Compared to Placebo
Time Frame: Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
The PGI-I is a 7-point rating system used by the patient to rate their overall clinical condition after intervention relative to before intervention where 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse, and 7=very much worse.
Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
Assess the Effect of ERX-963 on the Clinical Global Impressment - Improvement (CGI-I) Scale Compared to Placebo
Time Frame: Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
The CGI-I is a 7-point rating system used by the clinician or investigator to compare the patient's overall clinical condition after intervention relative to before intervention where 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse, and 7=very much worse (Guy, 1976; Busner, 2007).
Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
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.
From dosing to approximately 2 hours
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.
From dosing to approximately 2 hours

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Study Director: Elliot Ehrich, MD, Chief Medical Officer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 17, 2019

Primary Completion (Actual)

March 31, 2020

Study Completion (Actual)

April 30, 2020

Study Registration Dates

First Submitted

May 16, 2019

First Submitted That Met QC Criteria

May 18, 2019

First Posted (Actual)

May 22, 2019

Study Record Updates

Last Update Posted (Actual)

June 23, 2021

Last Update Submitted That Met QC Criteria

June 22, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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