Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)

Inclusion Criteria:

1. Subject consents to participate, or if they are not their own legal guardian, offersassent supported by legally authorized representative consent
2. Subject is willing and able to comply with the study procedures as specified in theprotocol and to comply with the study drug administration. Caregiver also commits tothe study requirements prior to any study-related procedures
3. Subject and caregiver are both able to understand the spoken national language clearlyand caregiver can read and write to complete study assessments
4. Males age 18 to 40 years (inclusive)
5. Has FXS with molecular genetic confirmation of the full FMR1 mutation (>200cysteine-guanine-guanine [CGG] repeats). May have been confirmed historically or atScreening
6. Is in general good health as deemed by the Investigator, determined by physicalexamination, medical history, and laboratory tests
7. If receiving serotonin-selective reuptake inhibitor (SSRI), serotonin-norepinephrinereuptake inhibitor (SNRI), or serotonin antagonist and reuptake inhibitor (SARI), ison a stable, well-tolerated dose for the previous 3 months with no further changesanticipated
8. Is not sexually active or can confirm at least one form of contraceptive

Exclusion Criteria:

1. Any chronic major medical comorbid condition deemed by the Investigator as presentingadded risk to the subject, including but not limited to, refractory hypertension,kidney disease, or liver disease
2. Diagnosed with diabetes (Type 1 or II) or receiving any anti-diabetic medication
3. Unstable seizure disorder, defined by any seizure within 6 months prior to baselinevisit and/or a change in any anti-convulsant drug dosing in the 60 days prior to studyconsent
4. Changes in psychotropic or anti-convulsant (where taken for reasons other than seizurecontrol) drug treatment within 30 days prior to Screening
5. Significant changes in any educational, behavioral, and/or dietary interventions themonth prior to Screening
6. Planned initiation of new, or modification of ongoing, interventions during the study
7. Unable or unwilling to take oral medication (whole capsule, despite assistance with aspoonful of applesauce, yoghurt, or equivalent liquid food)
8. Consumption of liver enzyme inducers or inhibitors including and not limited to foods,medicines, herbal remedies and supplements three days prior to any Visit. Foods orbeverages containing CYP3A4/5 inhibitors (e.g., grapefruit, pomegranate, pomelo, andstar fruit) should be avoided before taking study medication and for up to 1 hour postdose throughout the study
9. Has abnormal baseline laboratory assessments including, but not limited to, alanineaminotransferase (ALT) or aspartate aminotransferase (AST) or total bilirubin >1.5 ×the upper limit of normal (ULN), serum creatinine >1.5 x ULN or other clinicallyrelevant laboratory abnormality
10. Has a clinically significant heart rate or blood pressure (BP) at Screening as judgedby the Investigator
11. Has received an investigational drug in any prior clinical study within 30 days or 5half-lives (whichever is longer) prior to Screening