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NCT Number: NCT03959189

Safety, Tolerability and Pharmacokinetics 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.

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Key information

Conditions

Myotonic Dystrophy, Type 1 (DM1) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disorders of Excessive Somnolence Dyssomnias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Mental Disorders Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Myotonic Disorders Myotonic Dystrophy Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Sleep Disorders, Intrinsic Sleep Wake Disorders

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Stanford Neurosciences Health Center, Palo Alto, California, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ERX-963

Drug

Active medicine

Placebo

Drug

Comparator

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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).

  4. 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.

  5. 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.

Sponsors and collaborators

Lead sponsor

Expansion Therapeutics, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2020
Study completion
2020
First posted
May 22, 2019
Registry last updated
Jun 23, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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