Study of SPG601 in Adult Men With Fragile X Syndrome
- Registration Number
- NCT06413537
- Lead Sponsor
- Spinogenix
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 10
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description Placebo Comparator: Placebo comparator to be administered to participants with Fragile X Syndrome Placebo 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. Experimental: Active SPG601 to be administered to participants with Fragile X Syndrome SPG601 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.
- Primary Outcome Measures
Name Time Method Clinical Global Impressions Improvement scale as determined by the treating clinician 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 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 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
Change in auditory response to chirp stimulus 15 days Auditory test will be evaluated for difference in responses to stimuli.
- Secondary Outcome Measures
Name Time Method Change in memory and cognitive assessment with RBANS list learning. 15 days RBANS (Repeatable Battery for the Assessment of Neuropsychological Status) is scored from -4 to 4, with higher values indicate higher memory and cognitive assessment
Change in attention and inhibition symptoms 15 days KiTAP Tests of Attentional Performance for Children, to measure alertness and assessments of visual reactions
Change in cognitive outcomes measured by NIH Cognitive Toolbox 15 days 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.Change in eye tracking for social gaze 15 days 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.
Change in auditory response to steady state auditory stimuli 15 days Auditory test will be evaluated for difference in responses to stimuli.
Change in eye tracking measured by electroretinography 15 days This test will measure the electrical activity in the retina in response to stimuli
Safety and tolerability of SPG601 in patients with Fragile X Syndrome 15 days Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Trial Locations
- Locations (1)
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States