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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 : Completed
First Posted : May 22, 2019
Results First Posted : June 23, 2021
Last Update Posted : June 23, 2021
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)
Actual 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
Actual Primary Completion Date : March 31, 2020
Actual Study Completion Date : April 30, 2020


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: 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 Outcome Measures :
  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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

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


Locations
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United States, California
Stanford Neurosciences Health Center
Palo Alto, California, United States, 94305
United States, Florida
Sleep Medicine Specialists of South Florida
Miami, Florida, United States, 33126
United States, Iowa
University of Iowa
Iowa City, Iowa, United States, 52242
United States, Maryland
The Center for Sleep & Wake Disorders
Chevy Chase, Maryland, United States, 20815
Sponsors and Collaborators
Expansion Therapeutics, Inc.
Investigators
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Study Director: Elliot Ehrich, MD Chief Medical Officer
  Study Documents (Full-Text)

Documents provided by Expansion Therapeutics, Inc.:
Study Protocol  [PDF] October 31, 2019
Statistical Analysis Plan  [PDF] April 2, 2020

Additional Information:
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Responsible Party: Expansion Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03959189    
Other Study ID Numbers: ERX-963-001
First Posted: May 22, 2019    Key Record Dates
Results First Posted: June 23, 2021
Last Update Posted: June 23, 2021
Last Verified: May 2021
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
Myotonic Disorders
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn