Trial Outcomes & Findings for A Study of OV101 in Individuals With Fragile X Syndrome (NCT NCT03697161)
NCT ID: NCT03697161
Last Updated: 2024-01-23
Results Overview
Number of Participants with Treatment Emergent Adverse Events
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
36 participants
Primary outcome timeframe
Week 12
Results posted on
2024-01-23
Participant Flow
Participants were enrolled in 7 study centers in the United States and 1 study center in Israel.
Participant milestones
| Measure |
OV101 (Gaboxadol) Regimen 1
5 mg/day (5 mg QD) OV101 (gaboxadol)
|
OV101 (Gaboxadol) Regimen 2
10 mg/day (5 mg BID) OV101 (gaboxadol)
|
OV101 (Gaboxadol) Regimen 3
15 mg/day (5 mg TID) OV101 (gaboxadol)
|
|---|---|---|---|
|
Overall Study
STARTED
|
7
|
8
|
8
|
|
Overall Study
COMPLETED
|
6
|
7
|
7
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
23 Participants
Baseline characteristics by cohort
| Measure |
OV101 (Gaboxadol) Regimen 1
n=7 Participants
5 mg/day (5 mg QD) OV101 (gaboxadol)
|
OV101 (Gaboxadol) Regimen 2
n=8 Participants
10 mg/day (5 mg BID) OV101 (gaboxadol)
|
OV101 (Gaboxadol) Regimen 3
n=8 Participants
15 mg/day (5 mg TID) OV101 (gaboxadol)
|
Total
n=23 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Customized
Age of subjects: Adolescent (13 to 17)
|
4 Participants
n=5 Participants • 23 Participants
|
5 Participants
n=7 Participants • 23 Participants
|
4 Participants
n=5 Participants • 23 Participants
|
13 Participants
n=4 Participants • 23 Participants
|
|
Age, Customized
Age of subjects: Adult (18 to 23)
|
3 Participants
n=5 Participants • 23 Participants
|
3 Participants
n=7 Participants • 23 Participants
|
4 Participants
n=5 Participants • 23 Participants
|
10 Participants
n=4 Participants • 23 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
23 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
20 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (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
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
8 participants
n=7 Participants
|
8 participants
n=5 Participants
|
19 participants
n=4 Participants
|
|
Region of Enrollment
Israel
|
4 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
4 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Week 12Number of Participants with Treatment Emergent Adverse Events
Outcome measures
| Measure |
OV101 (Gaboxadol) (5 mg QD)
n=7 Participants
5 mg/day (5 mg QD) OV101 (gaboxadol)
|
OV101 (Gaboxadol) (5 mg BID)
n=8 Participants
10 mg/day (5 mg BID) OV101 (gaboxadol)
|
OV101 (Gaboxadol) (5 mg TID)
n=8 Participants
15 mg/day (5 mg TID) OV101 (gaboxadol)
|
|---|---|---|---|
|
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Any study medication-related serious TEAE
|
0 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Severity of TEAE Mild
|
1 participants
|
6 participants
|
6 participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Severity of TEAE Moderate
|
0 participants
|
2 participants
|
0 participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Severity of TEAE Severe
|
0 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Any TEAE leading to discontinuation
|
0 participants
|
1 participants
|
0 participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Any TEAE leading to dose modification
|
0 participants
|
1 participants
|
0 participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Any TEAE with outcome of death
|
0 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Any serious TEAE
|
0 participants
|
0 participants
|
0 participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Any study medication-related TEAE
|
1 participants
|
5 participants
|
2 participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Week 12Outcome measures
Outcome data not reported
Adverse Events
OV101 (5 mg QD)
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
OV101 (5 mg BID)
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
OV101 (5 mg TID)
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
OV101 (5 mg QD)
n=7 participants at risk
mg/day (5 mg QD) OV101
|
OV101 (5 mg BID)
n=8 participants at risk
10 mg/day (5 mg BID) OV101
|
OV101 (5 mg TID)
n=8 participants at risk
15 mg/day (5 mg TID) OV101
|
|---|---|---|---|
|
Infections and infestations
NASOPHARYNGITIS
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 4 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 6 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Psychiatric disorders
IRRITABILITY
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Gastrointestinal disorders
DIARRHOEA
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
25.0%
2/8 • Number of events 2 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
37.5%
3/8 • Number of events 3 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Psychiatric disorders
STEREOTYPY
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Gastrointestinal disorders
FOOD POISONING
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Gastrointestinal disorders
VOMITING
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Infections and infestations
VIRAL RHINITIS
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Psychiatric disorders
AGGRESSION
|
14.3%
1/7 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Psychiatric disorders
COMPULSIONS
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Nervous system disorders
DIZZINESS
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Nervous system disorders
HEADACHE
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
37.5%
3/8 • Number of events 3 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Respiratory, thoracic and mediastinal disorders
NASAL CONGESTION
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Respiratory, thoracic and mediastinal disorders
OROPHARYNGEAL PAIN
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Respiratory, thoracic and mediastinal disorders
RHINORRHOEA
|
14.3%
1/7 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Musculoskeletal and connective tissue disorders
ARTHRALGIA
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Musculoskeletal and connective tissue disorders
PAIN IN EXTREMITY
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
General disorders
FEELING ABNORMAL
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Metabolism and nutrition disorders
DEHYDRATION
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
|
Skin and subcutaneous tissue disorders
RASH
|
0.00%
0/7 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
12.5%
1/8 • Number of events 1 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
0.00%
0/8 • 12 weeks
All adverse events collected as part of the study are reported in the Adverse Event module
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place