Primary Disease or Condition Being Studied: Angelman Syndrome (AS) Clinical Trial
— NEPTUNEOfficial 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
| Verified date | January 2024 |
| Source | Ovid Therapeutics Inc. |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
The purpose of this study is to assess the efficacy and safety of oral OV101 (gaboxadol) in pediatric subjects with Angelman syndrome.
| Status | Completed |
| Enrollment | 104 |
| Est. completion date | November 2, 2020 |
| Est. primary completion date | November 2, 2020 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 2 Years to 12 Years |
| Eligibility | 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. |
| Country | Name | City | State |
|---|---|---|---|
| Australia | Ovid Therapeutics Investigative Site | Brisbane | Queensland |
| Australia | Ovid Therapeutics Investigative Site | Heidelberg | Victoria |
| Germany | Ovid Therapeutics Investigative Site | Munich | |
| Israel | Ovid Therapeutics Investigative Site | Ramat Gan | |
| Netherlands | Ovid Therapeutics Investigative Site | Rotterdam | |
| United States | Ovid Therapeutics Investigative Site | Boston | Massachusetts |
| United States | Ovid Therapeutics Investigative Site | Chicago | Illinois |
| United States | Ovid Therapeutics Investigative Site | Cincinnati | Ohio |
| United States | Ovid Therapeutics Investigative Site | Lexington | Massachusetts |
| United States | Ovid Therapeutics Investigative Site | Media | Pennsylvania |
| United States | Ovid Therapeutics Investigative Site | Nashville | Tennessee |
| United States | Ovid Therapeutics Investigative Site | Phoenix | Arizona |
| United States | Ovid Therapeutics Investigative Site | San Diego | California |
| United States | Ovid Therapeutics Investigative Site | Tacoma | Washington |
| Lead Sponsor | Collaborator |
|---|---|
| Ovid Therapeutics Inc. |
United States, Australia, Germany, Israel, Netherlands,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks | To evaluate the efficacy of OV101 versus placebo as assessed by the Clinical Global Impressions-Improvement-Angelman syndrome (CGI-I-AS) score at Week 12. CGIA-I-AS scores range from 1 (very much improved), to 4 (no change), to 7 (very much worse). | 12 weeks |