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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-06-17

Study Completion Date

2020-04-30

Brief Summary

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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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Detailed Description

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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.

Conditions

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Myotonic Dystrophy, Type 1 (DM1) Myotonic Dystrophy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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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.

Group Type EXPERIMENTAL

ERX-963

Intervention Type DRUG

Active medicine

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.

Group Type EXPERIMENTAL

Placebo

Intervention Type DRUG

Comparator

Interventions

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ERX-963

Active medicine

Intervention Type DRUG

Placebo

Comparator

Intervention Type DRUG

Eligibility Criteria

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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

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

Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No