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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 9, 2019
Sponsor:
Information provided by (Responsible Party):
Expansion Therapeutics, Inc.

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.


Condition or disease Intervention/treatment Phase
Myotonic Dystrophy, Type 1 (DM1) Myotonic Dystrophy Drug: ERX-963 Drug: Placebo Phase 1

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Double-Blind, Placebo-Controlled, Dose-Range-Finding, Crossover Trial of Single Day Administration of ERX-963 in Adults With Myotonic Dystrophy Type 1
Actual Study Start Date : June 17, 2019
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019


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.
Drug: ERX-963
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.
Drug: Placebo
Comparator




Primary Outcome Measures :
  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: up to 40 days ]
    Percentage of participants with Adverse Events, Serious Adverse Events, and drug-related Adverse Events


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.



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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03959189


Contacts
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Contact: Anita Seto, PhD 858-764-4290 info@expansionrx.com

Locations
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United States, California
Stanford Neurosciences Health Center Recruiting
Palo Alto, California, United States, 94305
Contact: Mitchell Reddan    650-725-4341    neuromuscularresearch@stanford.edu   
Principal Investigator: Jacinda Sampson, MD, PhD         
United States, Florida
Sleep Medicine Specialists of South Florida Recruiting
Miami, Florida, United States, 33126
Contact: Maria Almanzar, Study Coordinator    305-994-1825    malmanzar@southfloridasleeps.com   
Principal Investigator: Edward Mezerhane, MD, FACP         
United States, Iowa
University of Iowa Recruiting
Iowa City, Iowa, United States, 52242
Contact: Nicole Kressin         
Sponsors and Collaborators
Expansion Therapeutics, Inc.
Investigators
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Study Director: Elliot Ehrich, MD Chief Medical Officer

Additional Information:
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Responsible Party: Expansion Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03959189     History of Changes
Other Study ID Numbers: ERX-963-001
First Posted: May 22, 2019    Key Record Dates
Last Update Posted: October 9, 2019
Last Verified: August 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Expansion Therapeutics, Inc.:
Excessive Daytime Sleepiness
hypersomnia
Additional relevant MeSH terms:
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Myotonic Dystrophy
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Myotonic Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn