Study of SPG601 in Adult Men With Fragile X Syndrome

Spinogenix10 enrolled

Overview

This Phase 2 study described herein will evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamics of SPG601 in adult men with Fragile X syndrome.

This study is a Phase 2 randomized, double blind, placebo-controlled, cross over, single dose of SPG601 and matching Placebo in patients with Fragile X syndrome. This study will entail 2 in person clinic visits to administer oral doses of SPG601 or matching placebo. Participants will receive a dose of either SPG601 or placebo at first visit, and will cross over to receive the other product at the second visit.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Fragile X Syndrome

Interventions

SPG601DRUG

synthetic small molecule

PlaceboDRUG

Placebo

Eligibility

Sex: MALEMin age: 18 YearsMax age: 45 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Adult males aged 18 to 45 years * Diagnosis of Fragile X as confirmed with genetic testing * Patient must have caregiver * Must be in good health with no significant medical history * Clinical laboratory values within normal range or \< 1.2 times ULN * Contraceptive use by men or women consistent with local regulations * Able and willing to provide written informed consent * Stable dosing of psychotropic drugs for at least 4 weeks Exclusion Criteria: * Any physical or psychological condition that prohibits study completion * Uncontrolled seizures or history of epilepsy with a seizure in the past 6 months. * Auditory or visual impairments that can not be corrected * History of suicidal behavior or suicidal ideation * Screening vital signs that are abnormal per protocol specification * ECG that are clinically significant abnormal * History of substance abuse or dependence within 6 months * Other investigational products within 30 days

Locations (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Outcomes

Primary Outcomes

Clinical Global Impressions Improvement Scale as Determined by the Treating Clinician

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

Time frame: 15 days

Clinical Global Impressions Improvement Scale as Determined by the Caregiver

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

Time frame: 15 days

Visual Analog Scale as Determined by the Patient Caregiver

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

Time frame: 15 days

Change in Auditory Response to Chirp Stimulus

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).

Time frame: 15 days

Secondary Outcomes

Change From Baseline in Attention and Inhibition Symptoms-Go/NoGo Subtest

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.

Data from ClinicalTrials.gov

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Change in Cognitive Outcomes Measured by NIH Cognitive Toolbox

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.

Time frame: 15 days

Change in Memory and Cognitive Assessment With RBANS List Learning.

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

Time frame: 15 days

Change in Auditory Response to Steady State Auditory Stimuli From Baseline

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)

Time frame: 15 days

Safety and Tolerability of SPG601 in Patients With Fragile X Syndrome

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

Time frame: 15 days

Change From Baseline in Attention and Inhibition Symptoms-Flexibility Subtest

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.

Time frame: 15 days

Change From Baseline in Attention and Inhibition Symptoms-Alertness Subtest

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.

Time frame: 15 days

Change From Baseline in Attention and Inhibition Symptoms-Distractability Subtest

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.

Time frame: 15 days

Change in Eye Tracking for Social Gaze

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.

Time frame: 15 days

Change in Eye Tracking Measured by Electroretinography

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

Time frame: 15 days

Change in Eye Tracking Measured by Electroretinography

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

Time frame: 15 days