Phase 2
Completed N=10
Study of SPG601 in Adult Men With Fragile X Syndrome
Source: ClinicalTrials.gov NCT06413537 ↗Enrolled (actual)
10
Serious AEs
0.0%
Results posted
May 2026
Primary outcomePrimary: Clinical Global Impressions Improvement Scale as Determined by the Treating Clinician — 3.7; 3.8 score on a scale
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.
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 | — |
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
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. Informational only — not medical advice.