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Not yet recruiting NCT07439510

A Study to Investigate the Effects and Safety of SPG601 for the Treatment of Fragile X Syndrome in Male Participants

Phase II / Phase III Interventional Fragile X Syndrome (FXS)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: SPG601, Placebo.
Who it may be relevant to
Registry conditions: Fragile X Syndrome (FXS). Basic parameters: 18 years — 45 years · Male.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Phase 2b/3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of SPG601 in Male Participants With Fragile X Syndrome

Overview

This Phase 2b/3, randomized, double-blind, placebo-controlled, 2-part study will evaluate the efficacy, safety and tolerability of different dose regimens of SPG601 in adult male participants with Fragile X syndrome.

Detailed description

This is a Phase 2b/3, randomized, double-blind, placebo-controlled, study designed to evaluate the efficacy, safety and tolerability of multiple dose regimens of SPG601 in male participants with FXS.

The study consists of two parts, Phase 2b, dose-regimen finding, 4-arm parallel design: Eligible participants will be randomized to receive SPG601 or matched placebo. Participants will receive study intervention over 4 weeks and will be followed up for 4 weeks after last dose of study intervention.

Phase 3, 2-arm parallel design: Eligible participants will be randomized 1:1 to receive SPG601 or matched placebo. The dose of SPG601 administered in the Phase 3 will be defined based on analysis of Phase 2b. Participants will receive study intervention over 12 weeks and will be followed up for 4 weeks after last dose of study intervention.

Interventions

  • Drug SPG601
    synthetic small molecule
  • Drug Placebo
    Placebo

Primary outcome measures

  • Phase 2b: Change from baseline in Total Cognition Composite Change Sensitive Score from the NIH-TCB [Time frame: 8 weeks]
  • Phase 2b: Change from baseline in EEG resting state relative power bands during rest [Time frame: 8 weeks]
  • Phase 3:Change from baseline in Total Cognition Composite score from the NIH-TCB [Time frame: 12 weeks]
Secondary outcome measures (12)
  • Phase 2b: Change from baseline in Fluid Reasoning Composite Change Sensitive Scores from the NIH-TCB [Time frame: 8 weeks]
  • Phase 2b: Change from baseline in Flanker Scores from the NIH-TCB [Time frame: 8 weeks]
  • Phase 2b: Incidence, nature, and severity of adverse events (AEs) and serious adverse events (SAEs) [Time frame: 8 weeks]
  • Phase 2b: Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS) throughout the study [Time frame: 8 weeks]
  • Phase 2b: The PK parameters of SPG601 concentrations in plasma-Cmax [Time frame: 4 weeks]
  • Phase 2b: The PK parameters of SPG601 concentrations in plasma-T 1/2 [Time frame: 4 weeks]
  • Phase 3: Incidence, nature, and severity of adverse events (AEs) and serious adverse events (SAEs) [Time frame: 16 weeks]
  • Phase 3: Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS) throughout the study [Time frame: 8 weeks]
  • Phase 3: Change from baseline in Flanker Scores from the NIH-TCB [Time frame: 12 weeks]
  • Phase 3: Change from baseline in Fluid Reasoning Composite Change Sensitive Scores from the NIH-TCB [Time frame: 12 weeks]
  • Phase 3: Change from baseline to week 4 and week 8 in Total Cognition Composite score from the NIH-TCB [Time frame: 8 weeks]
  • Phase 3: Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the Aberrant Behavior Checklist (ABC) Total Score in Stratum [Time frame: 12 weeks]

Eligibility criteria

Inclusion criteria

  • Adult males aged 18 to 45 years, inclusive
  • Diagnosis of Fragile X as confirmed with genetic testing
  • Patient must have caregiver
  • Must be in good health with no significant medical history

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 cannot 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
  • Unable to swallow capsules

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07439510 · SPG601-102 (CLARITY)

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗