A Study of OV101 in Individuals With Angelman Syndrome (AS) (NEPTUNE)

Ovid Therapeutics

Status and phase: Completed; Phase 3

Conditions: Primary Disease or Condition Being Studied: Angelman Syndrome (AS)

Treatments: Drug: Placebo; Drug: Gaboxadol

Study type: Interventional

Funder types: Industry

Identifiers

NCT04106557

OV101-19-001

Details and patient eligibility

About

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

Enrollment: 104 patients

Sex: All

Ages: 2 to 12 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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104 participants in 2 patient groups, including a placebo group

OV101 once daily (weight-based dosing) Other Name:Gaboxadol

Experimental group

Description:

OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration

Treatment:

Drug: Gaboxadol

Placebo once daily

Placebo Comparator group

Description:

Matching placebo,oral, provided once daily at bedtime for 12 week duration

Treatment:

Drug: Placebo