Trial Outcomes & Findings for A Study of OV101 in Individuals With Angelman Syndrome (AS) (NCT NCT04106557)
NCT ID: NCT04106557
Last Updated: 2024-01-08
Results Overview
To evaluate the efficacy of OV101 versus placebo as assessed by the Clinical Global Impressions-Improvement-Angelman syndrome (CGI-I-AS) score at Week 12. CGIA-I-AS scores range from 1 (very much improved), to 4 (no change), to 7 (very much worse).
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
104 participants
Primary outcome timeframe
12 weeks
Results posted on
2024-01-08
Participant Flow
Participants were enrolled at study sites in the United States, Australia, Germany, Israel, and Netherlands.
Participant milestones
| Measure |
OV101 Once Daily (Weight-based Dosing) Age 4 to 12
OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration 4 to 12 years old
|
Placebo Once Daily
Matching placebo,oral, provided once daily at bedtime for 12 week duration
Placebo: Matching placebo capsules to OV101 capsules.
|
OV101 Once Daily (Weight-based Dosing) Age 2 to 3
OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration 2 to 3 years old
|
|---|---|---|---|
|
Overall Study
STARTED
|
47
|
50
|
7
|
|
Overall Study
COMPLETED
|
47
|
48
|
7
|
|
Overall Study
NOT COMPLETED
|
0
|
2
|
0
|
Reasons for withdrawal
| Measure |
OV101 Once Daily (Weight-based Dosing) Age 4 to 12
OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration 4 to 12 years old
|
Placebo Once Daily
Matching placebo,oral, provided once daily at bedtime for 12 week duration
Placebo: Matching placebo capsules to OV101 capsules.
|
OV101 Once Daily (Weight-based Dosing) Age 2 to 3
OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration 2 to 3 years old
|
|---|---|---|---|
|
Overall Study
Physician Decision
|
0
|
1
|
0
|
|
Overall Study
Protocol Violation
|
0
|
1
|
0
|
Baseline Characteristics
Patients actual enroll
Baseline characteristics by cohort
| Measure |
OV101 Once Daily (Weight-based Dosing) Age 4 to 12
n=47 Participants
OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration
Gaboxadol: OV101 versus placebo once daily at bedtime for 12 weeks
|
Placebo Once Daily
n=50 Participants
Matching placebo,oral, provided once daily at bedtime for 12 week duration
Placebo: Matching placebo capsules to OV101 capsules.
|
OV101 Once Daily (Weight-based Dosing) Age 2 to 3
n=7 Participants
OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration
|
Total
n=104 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Customized
4-8
|
23 Participants
n=5 Participants • Patients actual enroll
|
25 Participants
n=7 Participants • Patients actual enroll
|
0 Participants
n=5 Participants • Patients actual enroll
|
48 Participants
n=4 Participants • Patients actual enroll
|
|
Age, Customized
9-12
|
24 Participants
n=5 Participants • Patients actual enroll
|
25 Participants
n=7 Participants • Patients actual enroll
|
0 Participants
n=5 Participants • Patients actual enroll
|
49 Participants
n=4 Participants • Patients actual enroll
|
|
Age, Customized
2-3
|
0 Participants
n=5 Participants • Patients actual enroll
|
0 Participants
n=7 Participants • Patients actual enroll
|
7 Participants
n=5 Participants • Patients actual enroll
|
7 Participants
n=4 Participants • Patients actual enroll
|
|
Sex: Female, Male
Female
|
24 Participants
n=5 Participants
|
20 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
46 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
23 Participants
n=5 Participants
|
30 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
58 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
44 Participants
n=5 Participants
|
46 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
97 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Region of Enrollment
Netherlands
|
3 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
4 participants
n=4 Participants
|
|
Region of Enrollment
United States
|
32 participants
n=5 Participants
|
35 participants
n=7 Participants
|
4 participants
n=5 Participants
|
67 participants
n=4 Participants
|
|
Region of Enrollment
Israel
|
4 participants
n=5 Participants
|
7 participants
n=7 Participants
|
3 participants
n=5 Participants
|
11 participants
n=4 Participants
|
|
Region of Enrollment
Australia
|
4 participants
n=5 Participants
|
4 participants
n=7 Participants
|
0 participants
n=5 Participants
|
8 participants
n=4 Participants
|
|
Region of Enrollment
Germany
|
4 participants
n=5 Participants
|
3 participants
n=7 Participants
|
0 participants
n=5 Participants
|
7 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 12 weeksPopulation: Total of 97 subjects, ages 4-12 years, inclusive. Participants aged 2 to 3 (n=7) did not participate in the scoring.
To evaluate the efficacy of OV101 versus placebo as assessed by the Clinical Global Impressions-Improvement-Angelman syndrome (CGI-I-AS) score at Week 12. CGIA-I-AS scores range from 1 (very much improved), to 4 (no change), to 7 (very much worse).
Outcome measures
| Measure |
OV101 (Ages 4-12 Years, Inclusive)
n=47 Participants
ages 4-12 years, inclusive taking OV101
|
Placebo
n=50 Participants
ages 4-12 years, taking Placebo
|
|---|---|---|
|
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
1-Very much improved
|
1 Participants
|
2 Participants
|
|
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
2-Much improved
|
11 Participants
|
11 Participants
|
|
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
3-Minimally improved
|
11 Participants
|
11 Participants
|
|
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
4-No change
|
22 Participants
|
21 Participants
|
|
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
5-Minimally worse
|
1 Participants
|
5 Participants
|
|
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
6-Much worse
|
1 Participants
|
0 Participants
|
|
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
7-Very much worse
|
0 Participants
|
0 Participants
|
Adverse Events
OV101 Once Daily (Weight-based Dosing) Other Name:Gaboxadol
Serious events: 1 serious events
Other events: 31 other events
Deaths: 0 deaths
Placebo Once Daily
Serious events: 0 serious events
Other events: 22 other events
Deaths: 0 deaths
OV101 Once Daily (Weight-based Dosing) Age 2-3
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
OV101 Once Daily (Weight-based Dosing) Other Name:Gaboxadol
n=47 participants at risk
OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration
Gaboxadol: OV101 versus placebo once daily at bedtime for 12 weeks Age (Actual) 4 to 12 Years at Screening
|
Placebo Once Daily
n=50 participants at risk
Matching placebo,oral, provided once daily at bedtime for 12 week duration
Placebo: Matching placebo capsules to OV101 capsules.
|
OV101 Once Daily (Weight-based Dosing) Age 2-3
n=7 participants at risk
OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration Age (Actual) 2 to 3 Years at Screening
|
|---|---|---|---|
|
Nervous system disorders
Seizure
|
2.1%
1/47 • Number of events 1 • 12 Weeks
|
0.00%
0/50 • 12 Weeks
|
0.00%
0/7 • 12 Weeks
|
Other adverse events
| Measure |
OV101 Once Daily (Weight-based Dosing) Other Name:Gaboxadol
n=47 participants at risk
OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration
Gaboxadol: OV101 versus placebo once daily at bedtime for 12 weeks Age (Actual) 4 to 12 Years at Screening
|
Placebo Once Daily
n=50 participants at risk
Matching placebo,oral, provided once daily at bedtime for 12 week duration
Placebo: Matching placebo capsules to OV101 capsules.
|
OV101 Once Daily (Weight-based Dosing) Age 2-3
n=7 participants at risk
OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration Age (Actual) 2 to 3 Years at Screening
|
|---|---|---|---|
|
General disorders
Fatigue
|
6.4%
3/47 • 12 Weeks
|
2.0%
1/50 • 12 Weeks
|
0.00%
0/7 • 12 Weeks
|
|
General disorders
Generalized tonic-clonic seizure
|
6.4%
3/47 • 12 Weeks
|
0.00%
0/50 • 12 Weeks
|
0.00%
0/7 • 12 Weeks
|
|
General disorders
Irritability
|
6.4%
3/47 • 12 Weeks
|
4.0%
2/50 • 12 Weeks
|
0.00%
0/7 • 12 Weeks
|
|
General disorders
Somnolence
|
6.4%
3/47 • 12 Weeks
|
0.00%
0/50 • 12 Weeks
|
14.3%
1/7 • 12 Weeks
|
|
Nervous system disorders
Drooling
|
4.3%
2/47 • 12 Weeks
|
0.00%
0/50 • 12 Weeks
|
0.00%
0/7 • 12 Weeks
|
|
General disorders
Pyrexia
|
4.3%
2/47 • 12 Weeks
|
0.00%
0/50 • 12 Weeks
|
14.3%
1/7 • 12 Weeks
|
|
Infections and infestations
URI
|
4.3%
2/47 • 12 Weeks
|
2.0%
1/50 • 12 Weeks
|
14.3%
1/7 • 12 Weeks
|
|
Nervous system disorders
Contusion
|
0.00%
0/47 • 12 Weeks
|
2.0%
1/50 • 12 Weeks
|
14.3%
1/7 • 12 Weeks
|
|
Ear and labyrinth disorders
Ear Infection
|
0.00%
0/47 • 12 Weeks
|
2.0%
1/50 • 12 Weeks
|
14.3%
1/7 • 12 Weeks
|
|
Nervous system disorders
Insomnia
|
2.1%
1/47 • 12 Weeks
|
4.0%
2/50 • 12 Weeks
|
14.3%
1/7 • 12 Weeks
|
|
Blood and lymphatic system disorders
Lethargy
|
0.00%
0/47 • 12 Weeks
|
2.0%
1/50 • 12 Weeks
|
14.3%
1/7 • 12 Weeks
|
|
Investigations
Prolonged aPTT
|
10.6%
5/47 • 12 Weeks
|
0.00%
0/50 • 12 Weeks
|
0.00%
0/7 • 12 Weeks
|
|
Infections and infestations
Nasopharyngitis
|
10.6%
5/47 • 12 Weeks
|
8.0%
4/50 • 12 Weeks
|
0.00%
0/7 • 12 Weeks
|
|
Nervous system disorders
Seizure
|
10.6%
5/47 • 12 Weeks
|
4.0%
2/50 • 12 Weeks
|
0.00%
0/7 • 12 Weeks
|
|
Gastrointestinal disorders
Vomiting
|
10.6%
5/47 • 12 Weeks
|
8.0%
4/50 • 12 Weeks
|
14.3%
1/7 • 12 Weeks
|
|
Gastrointestinal disorders
Diarrhea
|
8.5%
4/47 • 12 Weeks
|
6.0%
3/50 • 12 Weeks
|
14.3%
1/7 • 12 Weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place