A Study of OV101 in Individuals With Angelman Syndrome (AS)
NCT ID: NCT04106557
Last Updated: 2024-01-08
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
104 participants
INTERVENTIONAL
2019-09-09
2020-11-02
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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OV101 once daily (weight-based dosing) Other Name:Gaboxadol
OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration
Gaboxadol
OV101 versus placebo once daily at bedtime for 12 weeks
Placebo once daily
Matching placebo,oral, provided once daily at bedtime for 12 week duration
Placebo
Matching placebo capsules to OV101 capsules.
Interventions
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Gaboxadol
OV101 versus placebo once daily at bedtime for 12 weeks
Placebo
Matching placebo capsules to OV101 capsules.
Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
2 Years
12 Years
ALL
No
Sponsors
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Healx AI
INDUSTRY
Responsible Party
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Locations
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Ovid Therapeutics Investigative Site
Phoenix, Arizona, United States
Ovid Therapeutics Investigative Site
San Diego, California, United States
Ovid Therapeutics Investigative Site
Chicago, Illinois, United States
Ovid Therapeutics Investigative Site
Boston, Massachusetts, United States
Ovid Therapeutics Investigative Site
Lexington, Massachusetts, United States
Ovid Therapeutics Investigative Site
Cincinnati, Ohio, United States
Ovid Therapeutics Investigative Site
Media, Pennsylvania, United States
Ovid Therapeutics Investigative Site
Nashville, Tennessee, United States
Ovid Therapeutics Investigative Site
Tacoma, Washington, United States
Ovid Therapeutics Investigative Site
Brisbane, Queensland, Australia
Ovid Therapeutics Investigative Site
Heidelberg, Victoria, Australia
Ovid Therapeutics Investigative Site
Munich, , Germany
Ovid Therapeutics Investigative Site
Ramat Gan, , Israel
Ovid Therapeutics Investigative Site
Rotterdam, , Netherlands
Countries
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References
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Keary C, Bird LM, de Wit MC, Hatti S, Heimer G, Heussler H, Kolevzon A, Mathews A, Ochoa-Lubinoff C, Tan WH, Yan Y, Adams M. Gaboxadol in angelman syndrome: A double-blind, parallel-group, randomized placebo-controlled phase 3 study. Eur J Paediatr Neurol. 2023 Nov;47:6-12. doi: 10.1016/j.ejpn.2023.07.008. Epub 2023 Aug 1.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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OV101-19-001
Identifier Type: -
Identifier Source: org_study_id
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