Study of SPG601 in Adult Men With Fragile X Syndrome

NCT ID: NCT06413537

Last Updated: 2025-01-14

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-07-05
• Study Completion Date: 2024-12-31

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.

Conditions

Fragile X Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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.

Group Type: ACTIVE_COMPARATOR

SPG601

Intervention Type: DRUG

synthetic small molecule

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.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo

Interventions

SPG601

synthetic small molecule

Intervention Type: DRUG

Placebo

Placebo

Intervention Type: DRUG

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 45 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Avance Clinical Pty Ltd.

INDUSTRY

Sponsor Role: collaborator

Spinogenix

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Craig Erickson, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Locations

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Countries

United States

Other Identifiers

SPG601-01

Identifier Type: -

Identifier Source: org_study_id