## Protocol Section
### Identification Module
**NCT ID:** NCT06413537

**Brief Title:** Study of SPG601 in Adult Men With Fragile X Syndrome

**Official Title:** A Phase 2a, Randomized, Double-Blinded, Study Evaluating the Neurophysiologic vs Clinical Effects of Single-Dose SPG601 and Placebo in Adult Men With Fragile X Syndrome

#### Organization Study ID Info
**ID:** SPG601-01
#### Organization
**Class:** INDUSTRY
**Full Name:** Spinogenix
### Status Module
#### Completion Date
**Date:** 2025-02

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-21

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-20

**Overall Status:** NOT_YET_RECRUITING

#### Primary Completion Date
**Date:** 2024-12

**Type:** ESTIMATED

#### Start Date
**Date:** 2024-06

**Type:** ESTIMATED

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-14
**Type:** ACTUAL
**Study First Submit Date:** 2024-05-09
**Study First Submit QC Date:** 2024-05-09
### Sponsor Collaborators Module

#### Collaborators

**Class:** INDUSTRY

**Name:** Avance Clinical Pty Ltd.

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** Spinogenix

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** True

**Oversight Has DMC:** True

### Description Module

**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 Module

**Conditions:**

- Fragile X Syndrome

**Keywords:**

- Fragile X Syndrome

- Fragile X Chromosome

- cognitive outcomes

### Design Module

#### Design Info

**Allocation:** RANDOMIZED

**Intervention Model:** CROSSOVER

##### Masking Info

**Masking:** QUADRUPLE

**Masking Description:** Double-blinded

**Who Masked:**

- PARTICIPANT

- CARE_PROVIDER

- INVESTIGATOR

- OUTCOMES_ASSESSOR

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 10

**Type:** ESTIMATED

**Phases:**

- PHASE2

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** 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.

**Intervention Names:**

- Drug: SPG601

**Label:** Experimental: Active SPG601 to be administered to participants with Fragile X Syndrome

**Type:** ACTIVE_COMPARATOR

#### Arm Group 2

**Description:** 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.

**Intervention Names:**

- Drug: Placebo

**Label:** Placebo Comparator: Placebo comparator to be administered to participants with Fragile X Syndrome

**Type:** PLACEBO_COMPARATOR

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Experimental: Active SPG601 to be administered to participants with Fragile X Syndrome

**Description:** synthetic small molecule

**Name:** SPG601

**Type:** DRUG

#### Intervention 2

**Arm Group Labels:**

- Placebo Comparator: Placebo comparator to be administered to participants with Fragile X Syndrome

**Description:** Placebo

**Name:** Placebo

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** Change in clinician rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation

**Measure:** Clinical Global Impressions Improvement scale as determined by the treating clinician

**Time Frame:** 15 days

**Description:** Change in caregiver rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation

**Measure:** Clinical Global Impressions Improvement scale as determined by the caregiver

**Time Frame:** 15 days

**Description:** Change 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

**Measure:** Visual Analog scale as determined by the patient caregiver

**Time Frame:** 15 days

**Description:** Auditory test will be evaluated for difference in responses to stimuli.

**Measure:** Change in auditory response to chirp stimulus

**Time Frame:** 15 days

#### Secondary Outcomes

**Description:** KiTAP Tests of Attentional Performance for Children, to measure alertness and assessments of visual reactions

**Measure:** Change in attention and inhibition symptoms

**Time Frame:** 15 days

**Description:** 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.

**Measure:** Change in cognitive outcomes measured by NIH Cognitive Toolbox

**Time Frame:** 15 days

**Description:** 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.

**Measure:** Change in eye tracking for social gaze

**Time Frame:** 15 days

**Description:** RBANS (Repeatable Battery for the Assessment of Neuropsychological Status) is scored from -4 to 4, with higher values indicate higher memory and cognitive assessment

**Measure:** Change in memory and cognitive assessment with RBANS list learning.

**Time Frame:** 15 days

**Description:** Auditory test will be evaluated for difference in responses to stimuli.

**Measure:** Change in auditory response to steady state auditory stimuli

**Time Frame:** 15 days

**Description:** This test will measure the electrical activity in the retina in response to stimuli

**Measure:** Change in eye tracking measured by electroretinography

**Time Frame:** 15 days

**Description:** Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)

**Measure:** Safety and tolerability of SPG601 in patients with Fragile X Syndrome

**Time Frame:** 15 days

### Eligibility Module

**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

**Maximum Age:** 45 Years

**Minimum Age:** 18 Years

**Sex:** MALE

**Standard Ages:**

- ADULT

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** sarah.richter@cchmc.org

**Name:** Sarah Richter

**Phone:** 513-803-3324

**Role:** CONTACT

**Contact 2:**

**Email:** Craig.Erickson@cchmc.org

**Name:** Craig Erickson, MD

**Role:** CONTACT

#### Locations

**Location 1:**

**City:** Cincinnati

**Contacts:**

***Contact 1:***

- **Email:** sarah.richter@cchmc.org,

- **Name:** Sarah Richter

- **Phone:** 513-803-3324

- **Role:** CONTACT

***Contact 2:***

- **Email:** ashley.dapore@cchmc.org

- **Name:** Ashley Dapore

- **Role:** CONTACT

***Contact 3:***

- **Name:** Craig A Erikson, MD

- **Role:** PRINCIPAL_INVESTIGATOR

***Contact 4:***

- **Name:** Ernest Pedapati, MD

- **Role:** SUB_INVESTIGATOR

**Country:** United States

**Facility:** Cincinnati Children's Hospital Medical Center

**State:** Ohio

**Zip:** 45229

#### Overall Officials

**Official 1:**

**Affiliation:** Children's Hospital Medical Center, Cincinnati

**Name:** Craig Erickson, MD

**Role:** PRINCIPAL_INVESTIGATOR

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000004194

- Term: Disease

- ID: D000010335

- Term: Pathologic Processes

- ID: D000038901

- Term: Mental Retardation, X-Linked

- ID: D000008607

- Term: Intellectual Disability

- ID: D000019954

- Term: Neurobehavioral Manifestations

- ID: D000009461

- Term: Neurologic Manifestations

- ID: D000009422

- Term: Nervous System Diseases

- ID: D000025064

- Term: Sex Chromosome Disorders

- ID: D000025063

- Term: Chromosome Disorders

- ID: D000000013

- Term: Congenital Abnormalities

- ID: D000030342

- Term: Genetic Diseases, Inborn

- ID: D000040181

- Term: Genetic Diseases, X-Linked

- ID: D000020271

- Term: Heredodegenerative Disorders, Nervous System

### Condition Browse Module - Browse Branches

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: All

- Name: All Conditions

- Abbrev: BC10

- Name: Nervous System Diseases

- Abbrev: BC16

- Name: Diseases and Abnormalities at or Before Birth

- Abbrev: BXM

- Name: Behaviors and Mental Disorders

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M16355

- Name: Syndrome

- Relevance: HIGH

- As Found: Syndrome

- ID: M8721

- Name: Fragile X Syndrome

- Relevance: HIGH

- As Found: Fragile X Syndrome

- ID: M11589

- Name: Intellectual Disability

- Relevance: LOW

- As Found: Unknown

- ID: M24774

- Name: Mental Retardation, X-Linked

- Relevance: LOW

- As Found: Unknown

- ID: M21826

- Name: Neurobehavioral Manifestations

- Relevance: LOW

- As Found: Unknown

- ID: M12404

- Name: Neurologic Manifestations

- Relevance: LOW

- As Found: Unknown

- ID: M23023

- Name: Chromosome Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M23024

- Name: Sex Chromosome Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M12

- Name: Congenital Abnormalities

- Relevance: LOW

- As Found: Unknown

- ID: M23686

- Name: Genetic Diseases, Inborn

- Relevance: LOW

- As Found: Unknown

- ID: M24877

- Name: Genetic Diseases, X-Linked

- Relevance: LOW

- As Found: Unknown

- ID: M22092

- Name: Heredodegenerative Disorders, Nervous System

- Relevance: LOW

- As Found: Unknown

- ID: T2370

- Name: Fragile X Syndrome

- Relevance: HIGH

- As Found: Fragile X Syndrome

### Condition Browse Module - Meshes

- ID: D000005600

- Term: Fragile X Syndrome

- ID: D000013577

- Term: Syndrome

### Misc Info Module

- Version Holder: 2024-05-31
