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 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.

Time frame: 15 days

Secondary Outcomes

Change in attention and inhibition symptoms

KiTAP Tests of Attentional Performance for Children, to measure alertness and assessments of visual reactions

Time frame: 15 days

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 eye tracking for social gaze

Data from ClinicalTrials.gov

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