A Phase 2a, Randomized, Double-Blinded, Study Evaluating the Neurophysiologic vs Clinical Effects of Single-Dose SPG601 and Placebo in Adult Men With Fragile X Syndrome
Overview
- Phase
- Phase 2
- Intervention
- SPG601
- Conditions
- Fragile X Syndrome
- Sponsor
- Spinogenix
- Enrollment
- 10
- Locations
- 1
- Primary Endpoint
- Change in auditory response to chirp stimulus
- Status
- Completed
- Last Updated
- last year
Overview
Brief Summary
This Phase 2 study described herein will evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamics of SPG601 in adult men with Fragile X syndrome.
Detailed Description
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.
Investigators
Eligibility Criteria
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
Arms & Interventions
Experimental: Active SPG601 to be administered to participants with Fragile X Syndrome
Participants with Fragile X Syndrome will be randomized to receive SPG601 or placebo (8 capsules of 100 mg) one time on days 1 and 8 of the study.
Intervention: SPG601
Placebo Comparator: Placebo comparator to be administered to participants with Fragile X Syndrome
Participants with Fragile X Syndrome will be randomized to receive SPG601 or placebo (8 capsules of 100 mg) one time on days 1 and 8 of the study.
Intervention: Placebo
Outcomes
Primary Outcomes
Change in auditory response to chirp stimulus
Time Frame: 15 days
Auditory test will be evaluated for difference in responses to stimuli.
Clinical Global Impressions Improvement scale as determined by the treating clinician
Time Frame: 15 days
Change in clinician rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation
Clinical Global Impressions Improvement scale as determined by the caregiver
Time Frame: 15 days
Change in caregiver rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation
Visual Analog scale as determined by the patient caregiver
Time Frame: 15 days
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
Secondary Outcomes
- Change in memory and cognitive assessment with RBANS list learning.(15 days)
- Change in attention and inhibition symptoms(15 days)
- Change in cognitive outcomes measured by NIH Cognitive Toolbox(15 days)
- Change in eye tracking for social gaze(15 days)
- Change in auditory response to steady state auditory stimuli(15 days)
- Change in eye tracking measured by electroretinography(15 days)
- Safety and tolerability of SPG601 in patients with Fragile X Syndrome(15 days)