Fragile X Syndrome (FXS)
Conditions
Brief summary
The purpose of this study is to assess the safety, tolerability and efficacy of oral OV101 (gaboxadol) in subjects with Fragile X syndrome.
Interventions
DRUGOV101 (gaboxadol)
OV101 (gaboxadol)
Sponsors
Healx AI
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
MALE
Age
13 Years to 22 Years
Healthy volunteers
No
Inclusion criteria
* Is male and 13 to 22 years old (inclusive) at the time of informed consent. * Has a diagnosis of FXS with a confirmed FMR1 full mutation (≥200 CGG repeats).
Exclusion criteria
* Concomitant disease or condition that are clinically significant and would limit study participation * Clinically significant lab abnormalities or vital signs at the time of screening * History of uncontrollable seizure disorder or seizure episodes within 6 months of screening or change in the anticonvulsant pharmacotherapy in the past 3 months. * Unable or does not have a caregiver able to comply with study requirements. * Enrolled in any clinical trial within the 30 days before screening.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAE) | Week 12 | Number of Participants with Treatment Emergent Adverse Events |
Other
| Measure | Time frame |
|---|---|
| Clinical Global Impressions- Improvement (CGI-I) | Week 12 |
Countries
United States
Participant flow
Recruitment details
Participants were enrolled in 7 study centers in the United States and 1 study center in Israel.
Participants by arm
| Arm | Count |
|---|---|
| OV101 (Gaboxadol) Regimen 1 5 mg/day (5 mg QD) OV101 (gaboxadol) | 7 |
| OV101 (Gaboxadol) Regimen 2 10 mg/day (5 mg BID) OV101 (gaboxadol) | 8 |
| OV101 (Gaboxadol) Regimen 3 15 mg/day (5 mg TID) OV101 (gaboxadol) | 8 |
| Total | 23 |
Baseline characteristics
| Characteristic | OV101 (Gaboxadol) Regimen 2 | OV101 (Gaboxadol) Regimen 3 | OV101 (Gaboxadol) Regimen 1 | Total |
|---|---|---|---|---|
| Age, Customized Age of subjects: Adolescent (13 to 17) | 5 Participants | 4 Participants | 4 Participants | 13 Participants |
| Age, Customized Age of subjects: Adult (18 to 23) | 3 Participants | 4 Participants | 3 Participants | 10 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) White | 7 Participants | 6 Participants | 7 Participants | 20 Participants |
| Region of Enrollment Israel | 0 participants | 0 participants | 4 participants | 4 participants |
| Region of Enrollment United States | 8 participants | 8 participants | 3 participants | 19 participants |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 8 Participants | 8 Participants | 7 Participants | 23 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 7 | 0 / 8 | 0 / 8 |
| other Total, other adverse events | 1 / 7 | 8 / 8 | 8 / 8 |
| serious Total, serious adverse events | 0 / 7 | 0 / 8 | 0 / 8 |
Outcome results
Primary
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Number of Participants with Treatment Emergent Adverse Events
Time frame: Week 12
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| OV101 (Gaboxadol) (5 mg QD) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any TEAE leading to discontinuation | 0 participants |
| OV101 (Gaboxadol) (5 mg QD) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any TEAE leading to dose modification | 0 participants |
| OV101 (Gaboxadol) (5 mg QD) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Severity of TEAE Severe | 0 participants |
| OV101 (Gaboxadol) (5 mg QD) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any serious TEAE | 0 participants |
| OV101 (Gaboxadol) (5 mg QD) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any TEAE with outcome of death | 0 participants |
| OV101 (Gaboxadol) (5 mg QD) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Severity of TEAE Mild | 1 participants |
| OV101 (Gaboxadol) (5 mg QD) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any study medication-related serious TEAE | 0 participants |
| OV101 (Gaboxadol) (5 mg QD) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any study medication-related TEAE | 1 participants |
| OV101 (Gaboxadol) (5 mg QD) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Severity of TEAE Moderate | 0 participants |
| OV101 (Gaboxadol) (5 mg BID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any study medication-related TEAE | 5 participants |
| OV101 (Gaboxadol) (5 mg BID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any study medication-related serious TEAE | 0 participants |
| OV101 (Gaboxadol) (5 mg BID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Severity of TEAE Mild | 6 participants |
| OV101 (Gaboxadol) (5 mg BID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Severity of TEAE Moderate | 2 participants |
| OV101 (Gaboxadol) (5 mg BID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any TEAE leading to dose modification | 1 participants |
| OV101 (Gaboxadol) (5 mg BID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any TEAE with outcome of death | 0 participants |
| OV101 (Gaboxadol) (5 mg BID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any serious TEAE | 0 participants |
| OV101 (Gaboxadol) (5 mg BID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Severity of TEAE Severe | 0 participants |
| OV101 (Gaboxadol) (5 mg BID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any TEAE leading to discontinuation | 1 participants |
| OV101 (Gaboxadol) (5 mg TID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any serious TEAE | 0 participants |
| OV101 (Gaboxadol) (5 mg TID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Severity of TEAE Mild | 6 participants |
| OV101 (Gaboxadol) (5 mg TID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Severity of TEAE Moderate | 0 participants |
| OV101 (Gaboxadol) (5 mg TID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any study medication-related serious TEAE | 0 participants |
| OV101 (Gaboxadol) (5 mg TID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any TEAE leading to discontinuation | 0 participants |
| OV101 (Gaboxadol) (5 mg TID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Severity of TEAE Severe | 0 participants |
| OV101 (Gaboxadol) (5 mg TID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any TEAE with outcome of death | 0 participants |
| OV101 (Gaboxadol) (5 mg TID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any TEAE leading to dose modification | 0 participants |
| OV101 (Gaboxadol) (5 mg TID) | Number of Participants With Treatment Emergent Adverse Events (TEAE) | Any study medication-related TEAE | 2 participants |
Other Pre-specified
Clinical Global Impressions- Improvement (CGI-I)
Time frame: Week 12