Clinical trial NCT06413537: Study of SPG601 in Adult Men With Fragile X Syndrome
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Fragile X Syndrome
Keywords: Fragile X Syndrome
Fragile X Chromosome
cognitive outcomes
Lead sponsor: Spinogenix
Sponsor class: INDUSTRY
Collaborators: name: Avance Clinical Pty Ltd.
First posted: 2024-05-14
Enrollment: 10
Sex: MALE
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Clinical Global Impressions Improvement scale as determined by the treating clinician
timeFrame: 15 days
measure: Clinical Global Impressions Improvement scale as determined by the caregiver
timeFrame: 15 days
measure: Visual Analog scale as determined by the patient caregiver
timeFrame: 15 days
measure: Change in auditory response to chirp stimulus
timeFrame: 15 days
Secondary outcomes:
measure: Change in attention and inhibition symptoms
timeFrame: 15 days
measure: Change in cognitive outcomes measured by NIH Cognitive Toolbox
timeFrame: 15 days
measure: Change in eye tracking for social gaze
timeFrame: 15 days
measure: Change in memory and cognitive assessment with RBANS list learning.
timeFrame: 15 days
measure: Change in auditory response to steady state auditory stimuli
timeFrame: 15 days
measure: Change in eye tracking measured by electroretinography
timeFrame: 15 days
measure: Safety and tolerability of SPG601 in patients with Fragile X Syndrome
timeFrame: 15 days
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Craig Erickson, MD
role: PRINCIPAL_INVESTIGATOR
References: