- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03959189
Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
Double-Blind, Placebo-Controlled, Dose-Range-Finding, Crossover Trial of Single Day Administration 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.
Study Overview
Status
Intervention / Treatment
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.
Study Type
Enrollment (Actual)
Phase
- Phase 1
Contacts and Locations
Study Locations
-
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California
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Palo Alto, California, United States, 94305
- Stanford Neurosciences Health Center
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Florida
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Miami, Florida, United States, 33126
- Sleep Medicine Specialists of South Florida
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Iowa
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Iowa City, Iowa, United States, 52242
- University of Iowa
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Maryland
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Chevy Chase, Maryland, United States, 20815
- The Center for Sleep & Wake Disorders
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: Double
Arms and Interventions
Participant Group / 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.
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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.
|
Comparator
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
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Adverse Events were collected from screening to the End of Study Visit, up to 57 days
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
From dosing to approximately 2 hours
|
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.
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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.
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Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
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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).
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Administered at the end of the dosing visit day, upon completion of the other outcome measures. Approximately 2 hours after the end of infusion.
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Assess the Effect of ERX-963 on the Psychomotor Vigilance Task (PVT)
Time Frame: From dosing to approximately 2 hours
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Participants will be tested for their response time and number of lapses during the PVT.
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From dosing to approximately 2 hours
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Assess the Effect of ERX-963 on the One-back Task
Time Frame: From dosing to approximately 2 hours
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Participants will be tested for the proportion of correct response to the One-back task.
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From dosing to approximately 2 hours
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Collaborators and Investigators
Sponsor
Investigators
- Study Director: Elliot Ehrich, MD, Chief Medical Officer
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- ERX-963-001
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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