Clinical trial NCT04106557: A Study of OV101 in Individuals With Angelman Syndrome (AS)
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Primary Disease or Condition Being Studied: Angelman Syndrome (AS)
Lead sponsor: Ovid Therapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2019-09-27
Enrollment: 104
Sex: ALL
Minimum age (years): 2
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to assess the efficacy and safety of oral OV101 (gaboxadol) in pediatric subjects with Angelman syndrome.


Eligibility criteria: 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.

Primary outcomes:
measure: Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
timeFrame: 12 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Ovid Therapeutics Investigative Site
state: Arizona
city: San Diego
country: United States
facility: Ovid Therapeutics Investigative Site
state: California
city: Chicago
country: United States
facility: Ovid Therapeutics Investigative Site
state: Illinois
city: Boston
country: United States
facility: Ovid Therapeutics Investigative Site
state: Massachusetts
city: Lexington
country: United States
facility: Ovid Therapeutics Investigative Site
state: Massachusetts
city: Cincinnati
country: United States
facility: Ovid Therapeutics Investigative Site
state: Ohio
city: Media
country: United States
facility: Ovid Therapeutics Investigative Site
state: Pennsylvania
city: Nashville
country: United States
facility: Ovid Therapeutics Investigative Site
state: Tennessee
city: Tacoma
country: United States
facility: Ovid Therapeutics Investigative Site
state: Washington
city: Brisbane
country: Australia
facility: Ovid Therapeutics Investigative Site
state: Queensland
city: Heidelberg
country: Australia
facility: Ovid Therapeutics Investigative Site
state: Victoria
city: Munich
country: Germany
facility: Ovid Therapeutics Investigative Site
city: Ramat Gan
country: Israel
facility: Ovid Therapeutics Investigative Site
city: Rotterdam
country: Netherlands
facility: Ovid Therapeutics Investigative Site
Study officials:
References: