Study of SPG601 in Adult Men With Fragile X Syndrome

NCT06413537 | Completed Phase 2 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 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.

  15 days

Secondary Outcomes (7)

• Change in attention and inhibition symptoms

  15 days

• Change in cognitive outcomes measured by NIH Cognitive Toolbox

  15 days

• Change in eye tracking for social gaze

  15 days

• Change in memory and cognitive assessment with RBANS list learning.

  15 days

• Change in auditory response to steady state auditory stimuli

  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

Study Officials

• Craig Erickson, MD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

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: January 14, 2025

Record last verified: 2025-01

Locations

US (1)