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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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5 July 2021 |
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Main ID: |
NCT03959189 |
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Date of registration:
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16/05/2019 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
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Scientific title:
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Double-Blind, Placebo-Controlled, Dose-Range-Finding, Crossover Trial of Single Day Administration of ERX-963 in Adults With Myotonic Dystrophy Type 1 |
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Date of first enrolment:
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June 17, 2019 |
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Target sample size:
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12 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT03959189 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
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Phase:
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Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Elliot Ehrich, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Chief Medical Officer |
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Key inclusion & exclusion criteria
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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
Age minimum:
18 Years
Age maximum:
65 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Myotonic Dystrophy
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Myotonic Dystrophy, Type 1 (DM1)
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Intervention(s)
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Drug: ERX-963
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Drug: Placebo
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Primary Outcome(s)
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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: Adverse Events were collected from screening to the End of Study Visit, up to 57 days]
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Secondary Outcome(s)
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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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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.]
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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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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]
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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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Secondary ID(s)
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ERX-963-001
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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