Phase 3
N=104
A Study of OV101 in Individuals With Angelman Syndrome (AS)
Primary Disease or Condition Being Studied: Angelman Syndrome (AS)
Bottom Line
View on ClinicalTrials.gov: NCT04106557 ↗Enrolled (actual)
104
Serious AEs
1.0%
Results posted
Jan 2024
Primary outcome: Primary: Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks — 1; 2; 11; 11 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Gaboxadol (Drug); Placebo (Drug)
- Age
- Pediatric · 2+ yrs
- Sex
- All
- Sponsor
- Healx AI
- Primary completion
- Nov 2020
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 | — |
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:
- 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). 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.