Phase 2 N=88 Randomized Quadruple-blind Treatment

A Study in Adults and Adolescents With Angelman Syndrome (STARS)

Angelman Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT02996305 ↗

Enrolled (actual)

Serious AEs

2.3%

Results posted

Mar 2022

Primary outcome: Primary: Incidence of Adverse Events in Placebo and Active Treatment Groups — 25; 27; 25; 23 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: OV101 Regimen 1 (Drug); OV101 regimen 2 (Drug); Placebo (Other)
Age: Pediatric, Adult · 13+ yrs
Sex: All
Sponsor: Healx AI
Primary completion: Jun 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Incidence of Adverse Events in Placebo and Active Treatment Groups	13; 18; 19; 2; 0; 2	—
PRIMARY Incidence of Adverse Events in Placebo and Active Treatment Groups	13; 18; 19; 2; 0; 2	—
PRIMARY Incidence of Adverse Events in Placebo and Active Treatment Groups	13; 18; 19; 2; 0; 2	—

Summary

The purpose of the study is to assess the safety and tolerability of oral OV101 (gaboxadol) in adult and adolescent subjects with Angelman syndrome. In addition, several exploratory efficacy outcome measures will be investigated.

Eligibility Criteria

Inclusion Criteria

Age 13- 49 years
Diagnosis of Angelman syndrome
Receiving a stable regimen of concomitant medications for at least 4 weeks prior to Baseline, and able to maintain these throughout the duration of the study
Has a caregiver capable of providing informed consent on behalf of the subject and able to attend scheduled study visits
Able to ingest study medication
Caregivers must agree not to post any subject or study information on social media

Exclusion Criteria

Unable to perform the study related safety and exploratory efficacy assessments, such as motor function
Poorly controlled seizure activity
Concomitant cardiovascular, respiratory, liver, renal, or hematologic diseases of a degree that would limit participation in the study
Pregnancy or women of child-bearing potential who are not using and acceptable method of contraception
Concomitant use of minocycline, levodopa, zolpidem, zaleplon, eszopiclone, ramelteon, and cannabinoid derivatives, or any other use of any investigational agent, device, and/or investigational procedure 4 weeks prior to Baseline and during the study
Allergy to OV101 or any excipients
At increased risk of harming self and/or others based on investigator assessment
Any condition or reason that in the opinion of the investigator makes the subject unsuitable for enrollment
Inability of subject or caregiver to comply with study requirements

Other protocol-defined inclusion/exclusion criteria may apply.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02996305). 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.