Study of SPG601 in Adult Men With Fragile X Syndrome

Fragile X Syndrome Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06413537
• Other study ID #: SPG601-01
• Status: Recruiting
• Phase: Phase 2
• Start date: June 18, 2024
• Est. completion date: February 2025

Study information

• Verified date: June 2024
• Source: Spinogenix
• Contact: Sarah Richter
• Phone: 513-803-3324
• Email: sarah.richter[@]cchmc.org
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: February 2025
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 45 Years

Eligibility

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

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• SPG601
synthetic small molecule
• Placebo
Placebo

Locations

Country	Name	City	State
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio

Sponsors (2)

Lead Sponsor	Collaborator
Spinogenix	Avance Clinical Pty Ltd.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	15 days
Primary	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	15 days
Primary	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	15 days
Primary	Change in auditory response to chirp stimulus	Auditory test will be evaluated for difference in responses to stimuli.	15 days
Secondary	Change in attention and inhibition symptoms	KiTAP Tests of Attentional Performance for Children, to measure alertness and assessments of visual reactions	15 days
Secondary	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.	15 days
Secondary	Change in eye tracking for social gaze	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.	15 days
Secondary	Change in memory and cognitive assessment with RBANS list learning.	RBANS (Repeatable Battery for the Assessment of Neuropsychological Status) is scored from -4 to 4, with higher values indicate higher memory and cognitive assessment	15 days
Secondary	Change in auditory response to steady state auditory stimuli	Auditory test will be evaluated for difference in responses to stimuli.	15 days
Secondary	Change in eye tracking measured by electroretinography	This test will measure the electrical activity in the retina in response to stimuli	15 days
Secondary	Safety and tolerability of SPG601 in patients with Fragile X Syndrome	Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)	15 days