Study of SPG601 in Adult Men With Fragile X Syndrome

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

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.

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