Phase 3
Completed N=104
A Study of OV101 in Individuals With Angelman Syndrome (AS)
Primary Disease or Condition Being Studied: Angelman Syndrome (AS)
Source: ClinicalTrials.gov NCT04106557 ↗
Enrolled (actual)
104
Serious AEs
1.0%
Results posted
Jan 2024
Primary outcomePrimary: Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks — 1; 2; 11; 11 Participants
◆ Published Evidence
Emerging
13citations · ~4 / year
Gaboxadol in angelman syndrome: A double-blind, parallel-group, randomized placebo-controlled phase 3 study.
Summary
The purpose of this study is to assess the efficacy and safety of oral OV101 (gaboxadol) in pediatric subjects with Angelman syndrome.
Linked Publications
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Gaboxadol in angelman syndrome: A double-blind, parallel-group, randomized placebo-controlled phase 3 study.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks |
1; 2; 11; 11; 11; 11 | — |
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:
- Subjects 2 to 3 years old must have a minimum body weight of 9 kg.
- 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.
Data sourced from ClinicalTrials.gov (NCT04106557) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.