Study of SPG601 in Adult Men With Fragile X Syndrome

NCT06413537 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: SPG601-01
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Fragile X Syndrome

Keywords

Fragile X Syndrome; Fragile X Chromosome; cognitive outcomes

Outcome Measures

Primary Outcomes (4)

• 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

• 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

• Visual Analog Scale as Determined by the Patient Caregiver

  Change from baseline 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

• Change in Auditory Response to Chirp Stimulus

  Auditory test will be evaluated for difference in responses to stimuli, as demonstrated by the Inter-Trial Coherence (ITC). ITC is a measure of phase consistency across trials, expressed as a dimensionless value ranging from 0 to 1. A value of 0 indicates completely random phase across trials, while 1 denotes a perfectly phase-locked response across trials. Higher ITC values reflect greater phase consistency of neural oscillations. ITC is a unit of measure as frequency (40Hz) vs time (msec).

  15 days

Secondary Outcomes (11)

• Change From Baseline in Attention and Inhibition Symptoms-Go/NoGo Subtest

  15 days

• Change in Cognitive Outcomes Measured by NIH Cognitive Toolbox

  15 days

• Change in Memory and Cognitive Assessment With RBANS List Learning.

  15 days

• Change in Auditory Response to Steady State Auditory Stimuli From Baseline

  15 days

• Safety and Tolerability of SPG601 in Patients With Fragile X Syndrome

  15 days

Study Arms (2)

Experimental: Active SPG601 to be administered to participants with Fragile X Syndrome

ACTIVE COMPARATOR

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.

Drug: SPG601

Placebo Comparator: Placebo comparator to be administered to participants with Fragile X Syndrome

PLACEBO COMPARATOR

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.

Drug: Placebo

Interventions

SPG601 DRUG

synthetic small molecule

Experimental: Active SPG601 to be administered to participants with Fragile X Syndrome

Placebo DRUG

Placebo

Placebo Comparator: Placebo comparator to be administered to participants with Fragile X Syndrome

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Spinogenix: lead
• Avance Clinical Pty Ltd.: collaborator

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Fragile X Syndrome

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System

Results Point of Contact

• Title: Chief Development Officer
• Organization: Spinogenix, Inc

sharron.gargosky@spinogenix.com

5039151400

Study Officials

• Craig Erickson, MD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-blinded
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 9, 2024
• First Posted: May 14, 2024
• Study Start: July 5, 2024
• Primary Completion: December 31, 2024
• Study Completion: December 31, 2024
• Last Updated: May 26, 2026
• Results First Posted: May 26, 2026

Record last verified: 2026-02

Locations

US (1)