Title

A Study in Adults and Adolescents With Angelman Syndrome (STARS)
A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol
  • Phase

    Phase 2
  • Study Type

    Interventional
  • Intervention/Treatment

    gaboxadol ...
  • Study Participants

    88
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.
Two dosing schedules of OV101 (gaboxadol) giving as once daily or twice daily dose will be assessed against placebo.
Study Started
Jan 31
2016
Primary Completion
Jun 07
2018
Study Completion
Aug 06
2018
Results Posted
Mar 31
2022
Last Update
Mar 31
2022

Drug OV101 Regimen 1

  • Other names: Gaboxadol

Drug OV101 regimen 2

  • Other names: Gaboxadol

Other Placebo

OV101 regimen 1 Experimental

OV101 once daily

OV101 regimen 2 Experimental

OV101 twice daily

Placebo Placebo Comparator

Twice daily

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.

Summary

Placebo

OV101 QD

OV101 BID

All Events

Event Type Organ System Event Term Placebo OV101 QD OV101 BID

Incidence of Adverse Events in Placebo and Active Treatment Groups

Summary of Subjects Reporting at least one Treatment Emergent Adverse Event (TEAEs), Safety Set. The table below summarizes the subjects who experienced TEAEs in the study.

Placebo

Any Life-Threatening TEAE

Any Mild TEAE

Any Moderate TEAE

Any Serious TEAE

Any Severe TEAE

Any TEAE

Any TEAE leading to Dose Change or Interruption

Any TEAE Leading to Study Withdrawal

Drug-related TEAE

OV101 QD

Any Life-Threatening TEAE

Any Mild TEAE

Any Moderate TEAE

Any Serious TEAE

Any Severe TEAE

Any TEAE

Any TEAE leading to Dose Change or Interruption

Any TEAE Leading to Study Withdrawal

Drug-related TEAE

OV101 BID

Any Life-Threatening TEAE

Any Mild TEAE

Any Moderate TEAE

Any Serious TEAE

Any Severe TEAE

Any TEAE

Any TEAE leading to Dose Change or Interruption

Any TEAE Leading to Study Withdrawal

Drug-related TEAE

Incidence of Adverse Events in Placebo and Active Treatment Groups

The Treatment Emergent Adverse Event (TEAEs) Reported by ≥10% of Subjects in Any Treatment Group by Preferred Term, Safety Set.

Placebo

Aggression

At least 1 TEAE

Decreased appetite

Diarrhoea

Irritability

Nasopharyngitis

Nausea

Pyrexia

Rash

Seizure

Somnolence

Upper respiratory tract infection

Vomiting

OV101 QD

Aggression

At least 1 TEAE

Decreased appetite

Diarrhoea

Irritability

Nasopharyngitis

Nausea

Pyrexia

Rash

Seizure

Somnolence

Upper respiratory tract infection

Vomiting

OV101 BID

Aggression

At least 1 TEAE

Decreased appetite

Diarrhoea

Irritability

Nasopharyngitis

Nausea

Pyrexia

Rash

Seizure

Somnolence

Upper respiratory tract infection

Vomiting

Incidence of Adverse Events in Placebo and Active Treatment Groups

Treatment-related TEAEs (Treatment Emergent Adverse Event) in ≥ 2 Subjects in OV101 Combined, Safety Set. The incidence of TEAEs assessed as treatment-related (at least possibly related to study drug, by the Investigator). Preferred Term in the table below.

Placebo

Aggression

Agitation

Anxiety

At least 1 Treatment-related TEAE

Decreased appetite

Enuresis

Fatigue

Insomnia

Irritability

Lethargy

Myoclonic epilepsy

Myoclonus

Nausea

Rash

Sedation

Seizure

Sleep disorder

Somnolence

Tremor

OV101 QD

Aggression

Agitation

Anxiety

At least 1 Treatment-related TEAE

Decreased appetite

Enuresis

Fatigue

Insomnia

Irritability

Lethargy

Myoclonic epilepsy

Myoclonus

Nausea

Rash

Sedation

Seizure

Sleep disorder

Somnolence

Tremor

OV101 BID

Aggression

Agitation

Anxiety

At least 1 Treatment-related TEAE

Decreased appetite

Enuresis

Fatigue

Insomnia

Irritability

Lethargy

Myoclonic epilepsy

Myoclonus

Nausea

Rash

Sedation

Seizure

Sleep disorder

Somnolence

Tremor

Total

87
Participants

Age, Continuous

Ethnicity (NIH/OMB)

Gender

Sex: Female, Male

Overall Study

Placebo

OV101 QD

OV101 BID