Study of SPG601 in Adult Men With Fragile X Syndrome

Change in clinician rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation

Change in caregiver rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation

Change from baseline in notation on Visual Analog scale as determined by the patient caregiver. Rater will indicate on linear measurement scale of 1 to 100 centimeters, with higher score indicating more severe symptoms

Auditory test will be evaluated for difference in responses to stimuli, as demonstrated by the Inter-Trial Coherence (ITC). ITC is a measure of phase consistency across trials, expressed as a dimensionless value ranging from 0 to 1. A value of 0 indicates completely random phase across trials, while 1 denotes a perfectly phase-locked response across trials. Higher ITC values reflect greater phase consistency of neural oscillations. ITC is a unit of measure as frequency (40Hz) vs time (msec).

KiTAP Tests of Attentional Performance for Children, to measure alertness and assessments of visual reactions. The Go-NoGo subtest measures impulsivity by requiring subjects to tap a button when the target stimulus is presented, while refraining from hitting the button for the non-target stimulus. Summary statistics presented for change in reaction time from baseline.

National Institute of Health (NIH)- Toolbox Cognitive Battery (TCB):

A series of tests will be conducted to assess reading, vocabulary, and speed matching. These tests are scored from zero to 100. A higher score indicate better performance.

RBANS (Repeatable Battery for the Assessment of Neuropsychological Status) is scored from -4 to 4, with higher values indicate higher memory and cognitive assessment

Auditory test will be evaluated for difference in responses to stimuli. Auditory Steady State Response (ASSR) is measured following a short stream of click trains with a 500 msec inter-train interval (duration), at standard tone and at 40Hz tone. The magnitude of ITC represents the phase consistency of oscillatory activities. ITC is a measure of phase consistency across trials, expressed as a dimensionless value ranging from 0 to 1. A value of 0 indicates completely random phase across trials, while 1 denotes a perfectly phase-locked response across trials. Higher ITC values reflect greater phase consistency of neural oscillations. ITC as a unit of measure is expressed as frequency (40Hz) vs time (msec)

Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)

The KiTAP is a computerized executive function battery that consists of eight nonverbal subtests measuring different basal as well as higher-order components of attention and executive functioning. The alertness subtest requires subjects to tap a button every time a stimulus appears on the screen. Mean reaction time was measured over 90 seconds and change from baseline was calculated. A lower reaction time (net decrease in reaction time) indicates better performance.

KiTAP Tests of Attentional Performance for Children, to measure alertness and assessments of visual reactions. The alertness subtest records the number of times a subject taps a button when a stimulus appears on screen. Change in Baseline summary statistics for reaction time presented below. A lower number indicates lower reaction time and better performance.

The KiTAP is a computerized executive function battery that consists of eight nonverbal subtests measuring components of attention and executive functioning. The distractibility subtest measures how easily the subject is distracted by extraneous stimuli. This subtest requires subjects to tap a button when a target stimulus appears on the screen while ignoring distractions. Mean number of correct responses were recorded, and change from baseline was calculated, and a higher mean number of correct responses indicates better performance.

This test will measure eye tracking and movement in response to stimuli, and social gaze version, with a Likert scale of 1-5 for behavior rating.

This test will measure the electrical activity in the retina in response to stimuli, evaluating change in stimuli response post dose minus stimuli pre dose-RIGHT EYE

This test will measure the electrical activity in the retina in response to stimuli, evaluating change in stimuli response post dose minus stimuli pre dose-LEFT EYE