Trial Outcomes & Findings for A Study in Adults and Adolescents With Angelman Syndrome (STARS) (NCT NCT02996305)
NCT ID: NCT02996305
Last Updated: 2025-08-03
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
88 participants
Primary outcome timeframe
Baseline and Week 12
Results posted on
2025-08-03
Participant Flow
Participant milestones
| Measure |
Placebo
Giving twice daily
|
OV101 QD
OV101, once daily (15mg night)
|
OV101 BID
OV101, twice daily (10mg morning, 15mg night)
|
|---|---|---|---|
|
Overall Study
STARTED
|
29
|
29
|
30
|
|
Overall Study
COMPLETED
|
26
|
27
|
25
|
|
Overall Study
NOT COMPLETED
|
3
|
2
|
5
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
Baseline characteristics by cohort
| Measure |
Placebo
n=29 Participants
Placebo BID Evening and Morning
|
OV101 QD
n=29 Participants
OV101 15 mg Evening Placebo in the Morning
|
OV101 BID
n=29 Participants
OV101 15 mg Evening OV101 10 mg Morning
|
Total
n=87 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
Age at Inform Consent
|
22.0 years
STANDARD_DEVIATION 6.70 • n=5 Participants
|
23.1 years
STANDARD_DEVIATION 7.76 • n=7 Participants
|
22.8 years
STANDARD_DEVIATION 6.51 • n=5 Participants
|
22.6 years
STANDARD_DEVIATION 6.95 • n=4 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
34 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=5 Participants
|
20 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
53 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
14 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
26 Participants
n=5 Participants
|
23 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
72 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Gender
Male
|
15 Participants
n=5 Participants
|
20 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
53 Participants
n=4 Participants
|
|
Gender
Famale
|
14 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
34 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 12Summary 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.
Outcome measures
| Measure |
Placebo
n=29 Participants
Twice daily
|
OV101 QD
n=29 Participants
OV101, once daily (15mg night)
|
OV101 BID
n=29 Participants
OV101, twice daily (10mg morning, 15mg night)
|
|---|---|---|---|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Any TEAE leading to Dose Change or Interruption
|
5 Participants
|
5 Participants
|
8 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Any TEAE Leading to Study Withdrawal
|
1 Participants
|
0 Participants
|
3 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Any TEAE
|
25 Participants
|
27 Participants
|
25 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Any Mild TEAE
|
23 Participants
|
23 Participants
|
23 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Any Moderate TEAE
|
9 Participants
|
15 Participants
|
9 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Any Severe TEAE
|
0 Participants
|
1 Participants
|
4 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Any Life-Threatening TEAE
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Drug-related TEAE
|
13 Participants
|
18 Participants
|
19 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Any Serious TEAE
|
0 Participants
|
1 Participants
|
1 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 12The Treatment Emergent Adverse Event (TEAEs) Reported by ≥10% of Subjects in Any Treatment Group by Preferred Term, Safety Set.
Outcome measures
| Measure |
Placebo
n=29 Participants
Twice daily
|
OV101 QD
n=29 Participants
OV101, once daily (15mg night)
|
OV101 BID
n=29 Participants
OV101, twice daily (10mg morning, 15mg night)
|
|---|---|---|---|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
At least 1 TEAE
|
25 Participants
|
27 Participants
|
25 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Vomiting
|
9 Participants
|
5 Participants
|
5 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Somnolence
|
5 Participants
|
5 Participants
|
3 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Irritability
|
4 Participants
|
3 Participants
|
5 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Pyrexia
|
2 Participants
|
7 Participants
|
1 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Upper respiratory tract infection
|
4 Participants
|
5 Participants
|
1 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Aggression
|
5 Participants
|
4 Participants
|
1 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Rash
|
1 Participants
|
3 Participants
|
2 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Seizure
|
0 Participants
|
2 Participants
|
3 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Decreased appetite
|
4 Participants
|
2 Participants
|
2 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Diarrhoea
|
3 Participants
|
1 Participants
|
3 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Nausea
|
3 Participants
|
0 Participants
|
4 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Nasopharyngitis
|
5 Participants
|
2 Participants
|
1 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 12Treatment-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.
Outcome measures
| Measure |
Placebo
n=29 Participants
Twice daily
|
OV101 QD
n=29 Participants
OV101, once daily (15mg night)
|
OV101 BID
n=29 Participants
OV101, twice daily (10mg morning, 15mg night)
|
|---|---|---|---|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
At least 1 Treatment-related TEAE
|
13 Participants
|
18 Participants
|
19 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Nausea
|
2 Participants
|
0 Participants
|
2 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Fatigue
|
2 Participants
|
1 Participants
|
2 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Decreased appetite
|
2 Participants
|
0 Participants
|
2 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Somnolence
|
5 Participants
|
5 Participants
|
2 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Myoclonic epilepsy
|
0 Participants
|
1 Participants
|
2 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Myoclonus
|
0 Participants
|
1 Participants
|
2 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Seizure
|
0 Participants
|
1 Participants
|
2 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Lethargy
|
0 Participants
|
0 Participants
|
2 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Sedation
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Tremor
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Irritability
|
3 Participants
|
2 Participants
|
4 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Aggression
|
2 Participants
|
4 Participants
|
1 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Insomnia
|
1 Participants
|
2 Participants
|
1 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Agitation
|
0 Participants
|
1 Participants
|
2 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Anxiety
|
0 Participants
|
0 Participants
|
2 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Enuresis
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Sleep disorder
|
0 Participants
|
0 Participants
|
2 Participants
|
|
Incidence of Adverse Events in Placebo and Active Treatment Groups
Rash
|
0 Participants
|
2 Participants
|
0 Participants
|
Adverse Events
Placebo
Serious events: 0 serious events
Other events: 13 other events
Deaths: 0 deaths
OV101 QD
Serious events: 1 serious events
Other events: 18 other events
Deaths: 0 deaths
OV101 BID
Serious events: 1 serious events
Other events: 19 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=29 participants at risk
Placebo Morning and Evening
|
OV101 QD
n=29 participants at risk
OV101 15mg Evening
|
OV101 BID
n=29 participants at risk
OV101 15 mg Evening OV101 10 mg Morning
|
|---|---|---|---|
|
Nervous system disorders
Seizure
|
0.00%
0/29 • 12 Weeks
|
3.4%
1/29 • 12 Weeks
|
3.4%
1/29 • 12 Weeks
|
Other adverse events
| Measure |
Placebo
n=29 participants at risk
Placebo Morning and Evening
|
OV101 QD
n=29 participants at risk
OV101 15mg Evening
|
OV101 BID
n=29 participants at risk
OV101 15 mg Evening OV101 10 mg Morning
|
|---|---|---|---|
|
Gastrointestinal disorders
Nausea
|
6.9%
2/29 • 12 Weeks
|
0.00%
0/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
|
Nervous system disorders
General disorders and administration site conditions
|
17.2%
5/29 • 12 Weeks
|
10.3%
3/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
|
General disorders
Fatigue
|
6.9%
2/29 • 12 Weeks
|
3.4%
1/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
|
Metabolism and nutrition disorders
Decreased appetite
|
6.9%
2/29 • 12 Weeks
|
0.00%
0/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
|
Nervous system disorders
Somnolence
|
17.2%
5/29 • 12 Weeks
|
17.2%
5/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
|
Nervous system disorders
Myoclonus
|
0.00%
0/29 • 12 Weeks
|
3.4%
1/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
|
Nervous system disorders
Seizure
|
0.00%
0/29 • 12 Weeks
|
3.4%
1/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
|
Nervous system disorders
Lethargy
|
0.00%
0/29 • 12 Weeks
|
0.00%
0/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
|
Psychiatric disorders
Irritability
|
10.3%
3/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
13.8%
4/29 • 12 Weeks
|
|
Psychiatric disorders
Aggression
|
6.9%
2/29 • 12 Weeks
|
13.8%
4/29 • 12 Weeks
|
3.4%
1/29 • 12 Weeks
|
|
Psychiatric disorders
Insomnia
|
3.4%
1/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
3.4%
1/29 • 12 Weeks
|
|
Psychiatric disorders
Agitation
|
0.00%
0/29 • 12 Weeks
|
3.4%
1/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/29 • 12 Weeks
|
0.00%
0/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
|
Psychiatric disorders
Sleep disorder
|
0.00%
0/29 • 12 Weeks
|
0.00%
0/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
0.00%
0/29 • 12 Weeks
|
|
Nervous system disorders
Myoclonic epilepsy
|
0.00%
0/29 • 12 Weeks
|
3.4%
1/29 • 12 Weeks
|
6.9%
2/29 • 12 Weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Publication 1. Institution/Investigator will submit to Sponsor any paper relating to the Study 60 days prior to disclosure. If Sponsor wants a patent to be filed on Inventions, then the period shall be extended by 90 days 2. Multi-center Studies.Institution/Investigator may publish a paper in its performance of Study after the later of (a) publication of the multi-center publication; (b) confirmation there will be no multi-center publication; or(c) 36 months after the completion of the Study
- Publication restrictions are in place
Restriction type: OTHER