Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome

NCT07328529 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: AVN014CDMRP-PR240390
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 3

Brief Summary

The goal of this clinical trial is to learn if drug SRX246 works to treat irritability, agitation, aggression and self-injury (IAAS) behaviors in adult males with Fragile X Syndrome (FXS). It will also learn about the safety of drug SRX246. The main questions it aims to answer are:

• Does drug SRX246 lower the number of times participants experience IAAS behaviors?
• What medical problems do participants have when taking drug SRX246? Researchers will compare drug SRX246 to a placebo (a look-alike substance that contains no drug) to see if drug SRX246 works to treat IAAS behaviors. Participants will:
• Take drug SRX246 or a placebo every day for up to 8 months
• Have weekly checkups by phone or video to answer study questions
• Have periodically scheduled home visits by nurses to conduct medical check-ups and tests
• Keep a diary of their symptoms

Conditions

Aggressive Behavior in Fragile X Syndome

Keywords

Fragile X Syndrome; SRX246; vasopressin 1a; irritability; agitation; aggression; self-injury

Outcome Measures

Primary Outcomes (1)

• Aberrant Behavior Checklist

  The Aberrant Behavior Checklist-Community Edition (ABC-C) Irritability subscale - Caregiver rated and then scored using the using the FXS-specific factoring system (ABCFX). Responses will be rated using a scale ranging from 0=not a problem to 3=severe problem.

  Over an 8 month period

Study Arms (2)

SRX246

EXPERIMENTAL

SRX246 will be provided as 120mg capsule to be taken orally, twice per day, once in the morning and once in the evening.

Drug: SRX246

Placebo

PLACEBO COMPARATOR

Placebo will be provided as a matching capsule to be taken orally, twice per day, once in the morning and once in the evening.

Drug: Placebo

Interventions

SRX246 DRUG

SRX246 120mg capsules for oral administration

SRX246

Placebo DRUG

Placebo capsules for oral administration

Placebo

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Biological male, age 18 to 45 years inclusive
• Molecular genetic confirmation of the full Fragile X messenger ribonucleoprotein mutation (≥200 CGG repetitions)
• IAAS symptoms leading to a CGI-S score for Disruptive Behavior of 5 or more
• Reported history of aggression based on the CMAI-Agg subscale (average score \>22) and an average associated disruptiveness scale of at least 3.
• IQ \< 70
• Stable medication doses for the last month
• Has a caregiver who lives with or spends extensive time with the participant most days and can report behavioral symptoms
• Must be able to swallow study drug capsules whole

You may not qualify if:

• Medical condition contraindicating study participation
• SGPT and SGOT values 2 or more times the upper limit of normal
• History of psychosis
• Suicidal behavior

Sponsors & Collaborators

• Azevan Pharmaceuticals: lead
• Congressionally Directed Medical Research Programs: collaborator

Study Sites (3)

University of California, Davis, MIND Institute

Sacramento, California, 95817, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Fragile X Syndrome; Psychomotor Agitation; Aggression; Self-Injurious Behavior

Interventions

SRX246

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System; Dyskinesias; Psychomotor Disorders; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Aberrant Motor Behavior in Dementia; Behavioral Symptoms; Behavior; Social Behavior

Central Study Contacts

Neal G Simon, PhD

CONTACT

610-610-0100; ngsimon@azevan.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 7, 2026
• First Posted: January 9, 2026
• Study Start: March 1, 2026
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2028
• Last Updated: January 9, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share

Locations

US (3)