Phase 2 N=10 Randomized Quadruple-blind Treatment

Study of SPG601 in Adult Men With Fragile X Syndrome

Fragile X Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT06413537 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

May 2026

Primary outcome: Primary: Clinical Global Impressions Improvement Scale as Determined by the Treating Clinician — 3.7; 3.8 score on a scale

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: SPG601 (Drug); Placebo (Drug)
Age: Adult · 18+ yrs
Sex: Male
Sponsor: Spinogenix
Primary completion: Dec 2024

Outcome Measures

Outcome	Result	p-value
PRIMARY Clinical Global Impressions Improvement Scale as Determined by the Treating Clinician	3.7; 3.8	—
PRIMARY Clinical Global Impressions Improvement Scale as Determined by the Caregiver	2.9; 2.8	—
PRIMARY Visual Analog Scale as Determined by the Patient Caregiver	-6.5; -6	—
PRIMARY Change in Auditory Response to Chirp Stimulus	-0.012; 0.014	—
SECONDARY Change From Baseline in Attention and Inhibition Symptoms-Go/NoGo Subtest	67.8; -17.6	—
SECONDARY Change in Cognitive Outcomes Measured by NIH Cognitive Toolbox	15.5; 1.7	—
SECONDARY Change in Memory and Cognitive Assessment With RBANS List Learning.	1.7; 3.8	—
SECONDARY Change in Auditory Response to Steady State Auditory Stimuli From Baseline	-0.012; 0.014	—
SECONDARY Safety and Tolerability of SPG601 in Patients With Fragile X Syndrome	1; 0	—
SECONDARY Change From Baseline in Attention and Inhibition Symptoms-Flexibility Subtest	-61.8; -103.4	—
SECONDARY Change From Baseline in Attention and Inhibition Symptoms-Alertness Subtest	129.4; -133	—
SECONDARY Change From Baseline in Attention and Inhibition Symptoms-Distractability Subtest	-54.7; 45.94	—
SECONDARY Change in Eye Tracking for Social Gaze	—	—
SECONDARY Change in Eye Tracking Measured by Electroretinography	-2.16; .03	—
SECONDARY Change in Eye Tracking Measured by Electroretinography	-2.16; .03	—

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.

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT06413537). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.