SRX246 for Fragile X Syndrome

Not yet recruiting at 2 trial locations

Overseen By Neal G Simon, PhD

Age: 18 - 65

Sex: Male

Trial Phase: Phase 2

Sponsor: Azevan Pharmaceuticals

Disqualifiers: Psychosis, Suicidal behavior, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 8 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a drug called SRX246 to determine if it can reduce irritability, agitation, aggression, and self-injury behaviors in adult males with Fragile X Syndrome. Researchers aim to find out if SRX246 can decrease the frequency of these behaviors and assess its safety. Participants will receive either SRX246 or a placebo and will take capsules twice daily for up to eight months. The trial seeks adult males aged 18 to 45 with Fragile X Syndrome who exhibit these behaviors and have a caregiver to help report symptoms. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but it requires that your medication doses have been stable for the last month. It's best to discuss your specific situation with the trial team.

Is there any evidence suggesting that SRX246 is likely to be safe for humans?

Research has shown that SRX246 is generally safe and well tolerated in previous studies. In studies involving individuals with Huntington's disease, SRX246 was tested at doses similar to those planned for this trial (120 mg twice daily). These studies found the treatment to be safe and well tolerated, with participants experiencing no major issues with the medication. This suggests it could be safe for humans. However, as with any treatment, some side effects may occur, so participants should maintain contact with the trial team and report any concerns. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Fragile X Syndrome?

SRX246 is unique because it targets the vasopressin 1a receptor, offering a novel approach to treating Fragile X Syndrome. Most treatments for this condition focus on symptoms rather than underlying mechanisms, but SRX246 works differently by potentially modulating social and emotional responses through this specific receptor. Researchers are excited about this treatment as it could lead to improvements in social behavior and anxiety, which are core challenges in Fragile X Syndrome. This targeted mechanism could provide a new avenue for relief that current options, like behavioral therapies and medications for symptoms, do not directly address.

What evidence suggests that SRX246 might be an effective treatment for Fragile X Syndrome?

Research shows that SRX246, which participants in this trial may receive, may help reduce aggressive behaviors. In studies on other conditions, SRX246 decreased aggression and irritability. The drug blocks a brain receptor called vasopressin 1a, linked to aggression and mood control. Previous research has shown that SRX246 is safe and well-tolerated at the doses being tested. These early findings offer hope that SRX246 could help manage irritability and aggression in people with Fragile X Syndrome. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

This trial is for adult males with Fragile X Syndrome who struggle with irritability, agitation, aggression, and self-injury. Participants will be involved in the study for up to 8 months, requiring weekly check-ins and periodic home visits by nurses.

Inclusion Criteria

Stable medication doses for the last month

I am a man aged between 18 and 45.

My behavior issues are severe, as rated by a professional.

Exclusion Criteria

Suicidal behavior

SGPT and SGOT values 2 or more times the upper limit of normal

History of psychosis

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants take SRX246 or placebo daily for up to 8 months to assess its effect on IAAS behaviors

8 months

Weekly checkups (phone/video), periodic home visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• SRX246

Trial Overview

The trial is testing SRX246 against a placebo to see if it can reduce aggressive behaviors in participants. The effectiveness of SRX246 will be measured by tracking the frequency of these behaviors over the course of treatment.

How Is the Trial Designed?

Group I: SRX246 Experimental Treatment 1 Intervention

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

Group II: Placebo Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

Azevan Pharmaceuticals

Lead Sponsor

Trials

8

Recruited

470+

Congressionally Directed Medical Research Programs

Collaborator

Trials

59

Recruited

10,600+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07328529

Inhibition of Aggressive Behavior in Participants With ...

The goal of this clinical trial is to learn if drug SRX246 works to treat irritability, agitation, aggression and self-injury (IAAS) behaviors ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC9605366/

The Vasopressin 1a Receptor Antagonist SRX246 Reduces ...

Our results showed that SRX246 reduced aggressive behavior in selected members of the per-protocol patient population. More specifically, those ...

3.

withpower.com

withpower.com/clinical-trials/for-fragile-x-syndrome

19 Fragile X Syndrome Trials Near You

It has been previously studied for its potential to improve cognitive function and behavioral outcomes in patients with fragile X syndrome. No Placebo Group.

4.

researchgate.net

researchgate.net/publication/363772611_The_Vasopressin_1a_Receptor_Antagonist_SRX246_Reduces_Aggressive_Behavior_in_Huntington's_Disease

(PDF) The Vasopressin 1a Receptor Antagonist SRX246 ...

This was a dose-escalation study; subjects received final doses of 120 mg BID, 160 mg BID, or placebo. The compound was safe and well tolerated.

5.

fraxa.org

fraxa.org/fragile-x-research-update-a-turning-point-for-treatments-and-curative-approaches/

Fragile X Research Update: A Turning Point for Treatments ...

These studies build on earlier FRAXA-funded research showing improvements in cognition, language and quality of life. If results hold up, ...

6.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/33207828/

Safety and Tolerability of SRX246, a Vasopressin 1a ...

The compound was safe and well tolerated in HD patients and can be moved forward as a candidate to treat irritability and aggression.

7.

clinicaltrials.gov

clinicaltrials.gov/study/NCT02507284

Tolerability, Safety, and Activity of SRX246 in Irritable ...

This study evaluates the tolerability, safety and activity of SRX246 in the treatment of irritability in patients with Huntington's disease.