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

E

Expansion Therapeutics

Status and phase

Completed
Phase 1

Conditions

Myotonic Dystrophy, Type 1 (DM1)
Myotonic Dystrophy

Treatments

Drug: Placebo
Drug: ERX-963

Study type

Interventional

Funder types

Industry

Identifiers

NCT03959189
ERX-963-001

Details and patient eligibility

About

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.

Full 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.

Enrollment

12 patients

Sex

All

Ages

18 to 65 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Crossover Assignment

Masking

Double Blind

12 participants in 2 patient groups

ERX-963 then placebo
Experimental group
Description:
Participants in this arm will receive ERX-963 followed by a washout period. After the washout period, participants will receive placebo.
Treatment:
Drug: ERX-963
Placebo then ERX-963
Experimental group
Description:
Participants in this arm will receive placebo followed by a washout period. After the washout period, participants will receive ERX-963.
Treatment:
Drug: Placebo

Trial documents
2

Trial contacts and locations

4

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Data sourced from clinicaltrials.gov

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