Study of SPG601 in Adult Men With Fragile X Syndrome

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

This Phase 2 study described herein will evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamics of SPG601 in adult men with Fragile X syndrome.

Study Overview

• Status: Not yet recruiting
• Conditions: Fragile X Syndrome
• Intervention / Treatment: Drug: Placebo; Drug: SPG601

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.

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Craig Erickson, MD; Email: Craig.Erickson@cchmc.org
• Study Contact Backup: Name: Sarah Richter; Phone Number: 513-803-3324; Email: sarah.richter@cchmc.org

Study Locations

• United States
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
      • Sub-Investigator: Ernest Pedapati, MD
      • Contact: Ashley Dapore; Email: ashley.dapore@cchmc.org
      • Principal Investigator: Craig A Erikson, MD
      • Contact: Sarah Richter; Phone Number: 513-803-3324; Email: sarah.richter@cchmc.org,

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Experimental: Active SPG601 to be administered to participants with Fragile X Syndrome; 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; synthetic small molecule
Placebo Comparator: Placebo Comparator: Placebo comparator to be administered to participants with Fragile X Syndrome; 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; Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
Change in attention and inhibition symptoms	KiTAP Tests of Attentional Performance for Children, to measure alertness and assessments of visual reactions	15 days
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
Change in eye tracking measured by electroretinography	This test will measure the electrical activity in the retina in response to stimuli	15 days
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
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
Change in auditory response to steady state auditory stimuli	Auditory test will be evaluated for difference in responses to stimuli.	15 days

Collaborators and Investigators

• Sponsor: Spinogenix
• Collaborators: Avance Clinical Pty Ltd.
• Investigators: Principal Investigator: Craig Erickson, MD, Children's Hospital Medical Center, Cincinnati

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2024
• Primary Completion (Estimated): December 1, 2024
• Study Completion (Estimated): February 1, 2025

Study Registration Dates

• First Submitted: May 9, 2024
• First Submitted That Met QC Criteria: May 9, 2024
• First Posted (Actual): May 14, 2024

Study Record Updates

• Last Update Posted (Actual): May 21, 2024
• Last Update Submitted That Met QC Criteria: May 20, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Keywords: Fragile X Syndrome; Fragile X Chromosome; cognitive outcomes
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Chromosome Disorders; Sex Chromosome Disorders; Syndrome; Fragile X Syndrome
• Other Study ID Numbers: SPG601-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No