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Safety, Tolerability and Pharmacokinetics 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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Myotonic Dystrophy, Type 1 (DM1) Myotonic Dystrophy | Drug: ERX-963 Drug: Placebo | Phase 1 |
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 : | 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 |
|---|---|
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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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Drug: ERX-963
Active medicine
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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.
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Drug: Placebo
Comparator
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- 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 57 days ]Percentage of participants with Adverse Events, Serious Adverse Events, and drug-related Adverse Events
- 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.
- 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.
- 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.
| Ages Eligible for Study: | 18 Years to 65 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
| 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 | |
| Study Director: | Elliot Ehrich, MD | Chief Medical Officer |
| Tracking Information | |||||
|---|---|---|---|---|---|
| First Submitted Date ICMJE | May 16, 2019 | ||||
| First Posted Date ICMJE | May 22, 2019 | ||||
| Last Update Posted Date | May 13, 2020 | ||||
| Actual Study Start Date ICMJE | June 17, 2019 | ||||
| Actual Primary Completion Date | March 31, 2020 (Final data collection date for primary outcome measure) | ||||
| Current Primary Outcome Measures ICMJE |
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 57 days ] Percentage of participants with Adverse Events, Serious Adverse Events, and drug-related Adverse Events
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| Original Primary Outcome Measures ICMJE |
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
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| Change History | |||||
| Current Secondary Outcome Measures ICMJE |
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| Original Secondary Outcome Measures ICMJE |
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| Current Other Pre-specified Outcome Measures | Not Provided | ||||
| Original Other Pre-specified Outcome Measures | Not Provided | ||||
| Descriptive Information | |||||
| Brief Title ICMJE | Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1 | ||||
| Official Title ICMJE | Double-Blind, Placebo-Controlled, Dose-Range-Finding, Crossover Trial of Single Day Administration of ERX-963 in Adults With Myotonic Dystrophy Type 1 | ||||
| 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. |
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| 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 ICMJE | Interventional | ||||
| Study Phase ICMJE | Phase 1 | ||||
| Study Design ICMJE | Allocation: Randomized Intervention Model: Crossover Assignment Masking: Double (Participant, Investigator) Primary Purpose: Treatment |
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| Condition ICMJE |
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| Intervention ICMJE |
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| Study Arms ICMJE |
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| Publications * | Not Provided | ||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||
| Recruitment Status ICMJE | Completed | ||||
| Actual Enrollment ICMJE |
12 | ||||
| Original Estimated Enrollment ICMJE | Same as current | ||||
| Actual Study Completion Date ICMJE | April 30, 2020 | ||||
| Actual Primary Completion Date | March 31, 2020 (Final data collection date for primary outcome measure) | ||||
| Eligibility Criteria ICMJE |
Key Inclusion Criteria:
Key Exclusion Criteria:
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| Sex/Gender ICMJE |
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| Ages ICMJE | 18 Years to 65 Years (Adult, Older Adult) | ||||
| Accepts Healthy Volunteers ICMJE | No | ||||
| Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
| Listed Location Countries ICMJE | United States | ||||
| Removed Location Countries | |||||
| Administrative Information | |||||
| NCT Number ICMJE | NCT03959189 | ||||
| Other Study ID Numbers ICMJE | ERX-963-001 | ||||
| Has Data Monitoring Committee | No | ||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement ICMJE |
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| Responsible Party | Expansion Therapeutics, Inc. | ||||
| Study Sponsor ICMJE | Expansion Therapeutics, Inc. | ||||
| Collaborators ICMJE | Not Provided | ||||
| Investigators ICMJE |
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| PRS Account | Expansion Therapeutics, Inc. | ||||
| Verification Date | January 2020 | ||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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