Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome

Azevan Pharmaceuticals

Status and phase: Begins enrollment this month; Phase 2

Conditions: Aggressive Behavior in Fragile X Syndome

Treatments: Drug: SRX246; Drug: Placebo

Study type: Interventional

Funder types: Industry; Other U.S. Federal agency

Identifiers

NCT07328529

AVN014

CDMRP-PR240390 (Other Grant/Funding Number)

Details and patient eligibility

About

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

Enrollment: 80 estimated patients

Sex: Male

Ages: 18 to 45 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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

Exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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80 participants in 2 patient groups, including a placebo group

SRX246

Experimental group

Description:

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

Treatment:

Drug: SRX246

Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo