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| Name | Class |
|---|---|
| Avance Clinical Pty Ltd. | INDUSTRY |
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This Phase 2 study described herein will evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamics of SPG601 in adult men with Fragile X syndrome.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental: Active SPG601 to be administered to participants with Fragile X Syndrome | Active Comparator | 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. |
|
| Placebo Comparator: Placebo comparator to be administered to participants with Fragile X Syndrome | Placebo Comparator | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SPG601 | Drug | synthetic small molecule |
|
| 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 from baseline 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, as demonstrated by the Inter-Trial Coherence (ITC). ITC is a measure of phase consistency across trials, expressed as a dimensionless value ranging from 0 to 1. A value of 0 indicates completely random phase across trials, while 1 denotes a perfectly phase-locked response across trials. Higher ITC values reflect greater phase consistency of neural oscillations. ITC is a unit of measure as frequency (40Hz) vs time (msec). | 15 days |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Attention and Inhibition Symptoms-Go/NoGo Subtest | KiTAP Tests of Attentional Performance for Children, to measure alertness and assessments of visual reactions. The Go-NoGo subtest measures impulsivity by requiring subjects to tap a button when the target stimulus is presented, while refraining from hitting the button for the non-target stimulus. Summary statistics presented for change in reaction time from baseline. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Craig Erickson, MD | Children's Hospital Medical Center, Cincinnati | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Sequence 1: SPG601 First, Placebo Second | Participants with Fragile X Syndrome will be randomized to receive SPG601 800 mg on day one, followed by eight placebo capsules on day eight of the study. SPG601: synthetic small molecule |
| FG001 | Sequence 2: Placebo First SPG601 Second | Participants with Fragile X Syndrome will be randomized to receive eight placebo capsules on day one of the study, followed by SPG601 800 mg on day eight of the study. SPG601: synthetic small molecule |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Adult men with Fragile X syndrome; cross-over study and all study participants were randomized to receive active drug or placebo at first study visit, and received other treatment allocation at subsequent study visit
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| ID | Title | Description |
|---|---|---|
| BG000 | The Study Was a Crossover Trial of Two Treatments Administered at Day 1 and Day 8 of the Study. | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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 | cross-over study, with all 10 participants receiving active drug or placebo at one study visit, and receive the other treatment arm at subsequent study visit | Posted | Mean | 95% Confidence Interval | score on a scale | 15 days |
|
Adverse event data was collected at study visits that happened weekly for 3 weeks.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. SPG601: synthetic small molecule |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| exacerbation of tic | Psychiatric disorders | MedDRA 27.0 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Development Officer | Spinogenix, Inc | 5039151400 | sharron.gargosky@spinogenix.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jun 17, 2024 | Oct 28, 2025 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D005600 | Fragile X Syndrome |
| ID | Term |
|---|---|
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
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Double-blinded
| Placebo | Drug | Placebo |
|
| 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 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 Auditory Response to Steady State Auditory Stimuli From Baseline | Auditory test will be evaluated for difference in responses to stimuli. Auditory Steady State Response (ASSR) is measured following a short stream of click trains with a 500 msec inter-train interval (duration), at standard tone and at 40Hz tone. The magnitude of ITC represents the phase consistency of oscillatory activities. ITC is a measure of phase consistency across trials, expressed as a dimensionless value ranging from 0 to 1. A value of 0 indicates completely random phase across trials, while 1 denotes a perfectly phase-locked response across trials. Higher ITC values reflect greater phase consistency of neural oscillations. ITC as a unit of measure is expressed as frequency (40Hz) vs time (msec) | 15 days |
| 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 From Baseline in Attention and Inhibition Symptoms-Flexibility Subtest | The KiTAP is a computerized executive function battery that consists of eight nonverbal subtests measuring different basal as well as higher-order components of attention and executive functioning. The alertness subtest requires subjects to tap a button every time a stimulus appears on the screen. Mean reaction time was measured over 90 seconds and change from baseline was calculated. A lower reaction time (net decrease in reaction time) indicates better performance. | 15 days |
| Change From Baseline in Attention and Inhibition Symptoms-Alertness Subtest | KiTAP Tests of Attentional Performance for Children, to measure alertness and assessments of visual reactions. The alertness subtest records the number of times a subject taps a button when a stimulus appears on screen. Change in Baseline summary statistics for reaction time presented below. A lower number indicates lower reaction time and better performance. | 15 days |
| Change From Baseline in Attention and Inhibition Symptoms-Distractability Subtest | The KiTAP is a computerized executive function battery that consists of eight nonverbal subtests measuring components of attention and executive functioning. The distractibility subtest measures how easily the subject is distracted by extraneous stimuli. This subtest requires subjects to tap a button when a target stimulus appears on the screen while ignoring distractions. Mean number of correct responses were recorded, and change from baseline was calculated, and a higher mean number of correct responses indicates 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 Eye Tracking Measured by Electroretinography | This test will measure the electrical activity in the retina in response to stimuli, evaluating change in stimuli response post dose minus stimuli pre dose-RIGHT EYE | 15 days |
| Change in Eye Tracking Measured by Electroretinography | This test will measure the electrical activity in the retina in response to stimuli, evaluating change in stimuli response post dose minus stimuli pre dose-LEFT EYE | 15 days |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
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. Placebo: Placebo |
|
|
| Primary | 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 | This assessment not completed for 2 participants | Posted | Mean | 95% Confidence Interval | score on a scale | 15 days |
|
|
|
| Primary | Visual Analog Scale as Determined by the Patient Caregiver | Change from baseline 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 | Posted | Mean | 95% Confidence Interval | score on a scale | 15 days |
|
|
|
| Primary | Change in Auditory Response to Chirp Stimulus | Auditory test will be evaluated for difference in responses to stimuli, as demonstrated by the Inter-Trial Coherence (ITC). ITC is a measure of phase consistency across trials, expressed as a dimensionless value ranging from 0 to 1. A value of 0 indicates completely random phase across trials, while 1 denotes a perfectly phase-locked response across trials. Higher ITC values reflect greater phase consistency of neural oscillations. ITC is a unit of measure as frequency (40Hz) vs time (msec). | Posted | Mean | Standard Deviation | Inter Trial Coherence | 15 days |
|
|
|
| Secondary | Change From Baseline in Attention and Inhibition Symptoms-Go/NoGo Subtest | KiTAP Tests of Attentional Performance for Children, to measure alertness and assessments of visual reactions. The Go-NoGo subtest measures impulsivity by requiring subjects to tap a button when the target stimulus is presented, while refraining from hitting the button for the non-target stimulus. Summary statistics presented for change in reaction time from baseline. | Posted | Mean | 95% Confidence Interval | milliseconds | 15 days |
|
|
|
| Secondary | 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. | Posted | Mean | 95% Confidence Interval | score on a scale | 15 days |
|
|
|
| Secondary | 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 | not completed for one participant | Posted | Mean | 95% Confidence Interval | score on a scale | 15 days |
|
|
|
| Secondary | Change in Auditory Response to Steady State Auditory Stimuli From Baseline | Auditory test will be evaluated for difference in responses to stimuli. Auditory Steady State Response (ASSR) is measured following a short stream of click trains with a 500 msec inter-train interval (duration), at standard tone and at 40Hz tone. The magnitude of ITC represents the phase consistency of oscillatory activities. ITC is a measure of phase consistency across trials, expressed as a dimensionless value ranging from 0 to 1. A value of 0 indicates completely random phase across trials, while 1 denotes a perfectly phase-locked response across trials. Higher ITC values reflect greater phase consistency of neural oscillations. ITC as a unit of measure is expressed as frequency (40Hz) vs time (msec) | Posted | Mean | Standard Deviation | ITC | 15 days |
|
|
|
| Secondary | 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) | Posted | Number | participants | 15 days |
|
|
|
| Secondary | Change From Baseline in Attention and Inhibition Symptoms-Flexibility Subtest | The KiTAP is a computerized executive function battery that consists of eight nonverbal subtests measuring different basal as well as higher-order components of attention and executive functioning. The alertness subtest requires subjects to tap a button every time a stimulus appears on the screen. Mean reaction time was measured over 90 seconds and change from baseline was calculated. A lower reaction time (net decrease in reaction time) indicates better performance. | Posted | Mean | 95% Confidence Interval | milliseconds | 15 days |
|
|
|
| Secondary | Change From Baseline in Attention and Inhibition Symptoms-Alertness Subtest | KiTAP Tests of Attentional Performance for Children, to measure alertness and assessments of visual reactions. The alertness subtest records the number of times a subject taps a button when a stimulus appears on screen. Change in Baseline summary statistics for reaction time presented below. A lower number indicates lower reaction time and better performance. | Posted | Mean | 95% Confidence Interval | milliseconds | 15 days |
|
|
|
| Secondary | Change From Baseline in Attention and Inhibition Symptoms-Distractability Subtest | The KiTAP is a computerized executive function battery that consists of eight nonverbal subtests measuring components of attention and executive functioning. The distractibility subtest measures how easily the subject is distracted by extraneous stimuli. This subtest requires subjects to tap a button when a target stimulus appears on the screen while ignoring distractions. Mean number of correct responses were recorded, and change from baseline was calculated, and a higher mean number of correct responses indicates better performance. | Posted | Mean | 95% Confidence Interval | correct responses | 15 days |
|
|
|
| Secondary | 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. | unable to collect and record data due to technical difficulties with equipment. Pre-specified measurement values were not obtained and will not be collected or analyzed in the future. | Posted | 15 days |
|
|
| Secondary | Change in Eye Tracking Measured by Electroretinography | This test will measure the electrical activity in the retina in response to stimuli, evaluating change in stimuli response post dose minus stimuli pre dose-RIGHT EYE | Posted | Mean | Standard Deviation | milli volts | 15 days |
|
|
|
| Secondary | Change in Eye Tracking Measured by Electroretinography | This test will measure the electrical activity in the retina in response to stimuli, evaluating change in stimuli response post dose minus stimuli pre dose-LEFT EYE | Posted | Mean | Standard Deviation | milli volts | 15 days |
|
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|
| 0 |
| 10 |
| 0 |
| 10 |
| 1 |
| 10 |
| EG001 | 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. Placebo: Placebo | 0 | 10 | 0 | 10 | 0 | 10 |
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| D009422 | Nervous System Diseases |
| D025064 | Sex Chromosome Disorders |
| D025063 | Chromosome Disorders |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D040181 | Genetic Diseases, X-Linked |
| D020271 | Heredodegenerative Disorders, Nervous System |