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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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15 January 2024 |
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Main ID: |
NCT04106557 |
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Date of registration:
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25/09/2019 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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A Study of OV101 in Individuals With Angelman Syndrome (AS)
NEPTUNE |
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Scientific title:
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A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 3 Study to Evaluate the Efficacy and Safety of OV101 in Pediatric Individuals With Angelman Syndrome |
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Date of first enrolment:
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September 9, 2019 |
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Target sample size:
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104 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT04106557 |
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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: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
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Phase:
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Phase 3
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Countries of recruitment
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Australia
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Germany
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Israel
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Netherlands
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United States
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Male or female and 2 to 12 years old (inclusive) at the time of informed consent
- Confirmed molecular diagnosis of AS
- Has a CGI-S-AS score of 3 or more at baseline.
- Meets the following age-appropriate body weight criterion:
1. Subjects 2 to 3 years old must have a minimum body weight of 9 kg.
2. Subjects 4 years and older must be between 17 kg and 64 kg (inclusive).
- Stable concomitant mediations for at least 4 weeks before study start
Exclusion Criteria:
- Any condition that would limit study participation
- Clinically significant lab or vital sign abnormalities at the time of screening
- Poorly controlled seizures (weekly seizures of any frequency with a duration more than
3 minutes, weekly seizures occurring more than 3 times per week, each with a duration
of less than 3 minutes, or as defined by investigator assessment)
- Use of prescription medications for sleep, minocycline, or levodopa within the 4 weeks
prior to Day 1 or during the study. Benzodiazepines chronically administered for
seizure control are permitted.
- Cannot comply with protocol study assessments during screening or caregiver unable to
comply with study requirements.
- Enrolled in any clinical trial or used any investigational agent within the 30 days
before screening or concurrently with this study.
Age minimum:
2 Years
Age maximum:
12 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Primary Disease or Condition Being Studied: Angelman Syndrome (AS)
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Intervention(s)
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Drug: Gaboxadol
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Drug: Placebo
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Primary Outcome(s)
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Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
[Time Frame: 12 weeks]
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Secondary ID(s)
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OV101-19-001
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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