Buspirone for Anxiety in Autism

Overseen By Meredith O'Connor, BS

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Massachusetts General Hospital

Must not be taking: MAOIs, CYP3A4 inducers

Disqualifiers: Seizure disorder, Schizophrenia, Bipolar, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of buspirone, a medication for treating anxiety in children and teens with autism spectrum disorders. The goal is to determine if buspirone can reduce anxiety symptoms in this group. Children and teens diagnosed with autism who experience significant anxiety or depression might be suitable for this study. Participants may continue their current medications if they have been stable for at least four weeks. As a Phase 2 trial, this research measures how well buspirone works in an initial, smaller group of patients.

Will I have to stop taking my current medications?

You can continue taking your current medications if you've been on a stable dose for at least 4 weeks, unless they are listed in the protocol's Concomitant Medications section. You cannot take certain medications like MAOIs or CYP3A4 inducers or inhibitors.

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

Research has shown that buspirone is generally well tolerated by children with autism spectrum disorder (ASD). Most studies suggest it is safe for children. For example, several studies found that children taking buspirone did not experience serious side effects. This medication already treats anxiety, supporting its safety. However, more research is needed to fully understand its effects on anxiety in children with ASD. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for anxiety in autism?

Unlike the standard treatments for anxiety in autism, which often include SSRIs or cognitive behavioral therapy, buspirone offers a unique approach by directly targeting serotonin receptors without causing significant sedation or dependency. Researchers are excited about buspirone because it has a different mechanism of action, potentially reducing anxiety symptoms with fewer side effects compared to traditional medications. Additionally, its twice-daily tablet form offers a straightforward dosing schedule, which can be easier for patients and caregivers to manage.

What evidence suggests that buspirone might be an effective treatment for anxiety in autism?

Research has shown that buspirone, the treatment being studied in this trial, can help reduce anxiety in people with autism. One study found that anxiety levels dropped from 55 to 18 on a specific anxiety scale after just two weeks of using buspirone. This suggests that the drug might help manage anxiety in those with autism. Additionally, buspirone may also help with the repetitive behaviors often seen in autism. Most studies find that it is safe and well-tolerated in children with autism, but more research is needed to confirm this. ^{1 2 4 5 6}

Who Is on the Research Team?

Atilla Ceranoglu, MD

Principal Investigator

Massachusetts General Hospital

Are You a Good Fit for This Trial?

This trial is for children and teens aged 6-17 with autism spectrum disorders who also experience anxiety, as indicated by a specific score on the CBCL Anxiety/Depression subscale. They must meet DSM-5 criteria for ASD and can be on stable psychotropic medications not listed in the protocol's Concomitant Medications section. Those with buspirone intolerance, seizure disorders, certain ECG abnormalities, or unstable psychiatric conditions cannot participate.

Inclusion Criteria

I've been on a stable dose of my mental health medication for at least 4 weeks.

I am between 6 and 17 years old.

Participants must meet DSM-5 ASD diagnostic criteria as established by clinical diagnostic interview

Exclusion Criteria

Subjects with a medical condition or treatment that will either jeopardize subject safety or affect the scientific merit of the study, including: pregnant or nursing females, organic brain disorders, uncorrected hypothyroidism or hyperthyroidism, clinically significant abnormalities on ECG (e.g., QT prolongation, arrhythmia), history of renal or hepatic impairment, clinically unstable psychiatric conditions or judged to be at serious suicidal risk, current diagnosis of schizophrenia or bipolar disorder, history of substance use (except nicotine or caffeine) within past 3 months or urine drug screen positive for substances of abuse, current treatment with medication with primary central nervous system activity (as specified in the Concomitant Medication section of the protocol), a non-responder or history of intolerance to buspirone, after treatment at an adequate dose and duration as determined by the clinician, subjects currently taking monoamine oxidase inhibitors (MAOI) and/or CYP3A4 inducers or inhibitors including nefazodone, diltiazem, verapamil, erythromaycin, itraconazole, or rifampin.

I have had a seizure or signs of seizure activity in the last month.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive buspirone tablets twice daily, titrated to a maximum daily dose of 60mg for 8 weeks

8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Buspirone

Trial Overview The study tests the safety and effectiveness of Buspirone in treating anxiety among youth with autism over an 8-week period. It aims to inform a larger future trial. Participants will receive Buspirone while their health outcomes are monitored to assess any changes in their anxiety levels.

How Is the Trial Designed?

Group I: Buspirone Experimental Treatment 1 Intervention

Buspirone tablets will be administered twice daily, and will be titrated to a maximum daily dose of 60mg for 8 weeks.

Buspirone is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Buspar for:

• Generalized anxiety disorder

Approved in European Union as Buspirone for:

• Generalized anxiety disorder

Approved in Canada as Buspirone for:

• Generalized anxiety disorder

Who Is Running the Clinical Trial?

Massachusetts General Hospital

Lead Sponsor

Trials

3,066

Recruited

13,430,000+

Published Research Related to This Trial

Buspirone is an anxiolytic medication that works differently from traditional benzodiazepines, showing comparable effectiveness to diazepam in reducing anxiety but with significantly less sedation and no abuse potential.

It acts as both a dopamine agonist and antagonist, interacting with various neurochemical systems in the brain, and has a rapid onset of action, achieving peak serum concentrations within one hour.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Buspirone hydrochloride: a unique new anxiolytic agent. Pharmacokinetics, clinical pharmacology, abuse potential and clinical efficacy. Goldberg, HL.[2019]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10291962/

Buspirone in Autism Spectrum Disorder: A Systematic ... Limited data indicate that low-dose buspirone can be effective in treating restricted and repetitive behaviors of ASD given its action on the presynaptic 5HT1A ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37378184/

Buspirone in Autism Spectrum Disorder: A Systematic ... Most studies suggested that buspirone was well tolerated and safe in pediatric patients with ASD. Based on the data, there is insufficient evidence to make ...

3. psychiatrist.com psychiatrist.com/pcc/buspirone-for-comorbid-anxiety-in-autism/

Buspirone for Comorbid Anxiety in Autism There was a significant response with buspirone, with improvement of anxiety symptoms and a reduction in SCARED scores from 55 to 18 in 2 weeks.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT00873509

Buspirone in the Treatment of 2-6 Year Old Children With ... The purpose of this study is to evaluate the effects of twice-daily oral buspirone on core features of autism in autistic children aged 2-6 years.

5. sciencedirect.com sciencedirect.com/science/article/pii/S0022347615014444

Efficacy of Low-Dose Buspirone for Restricted and ... Treatment with 2.5 mg of buspirone in young children with ASD might be a useful adjunct therapy to target restrictive and repetitive behaviors.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT01850355

NCT01850355 | An Open-Label Trial of Buspirone for the ... The main objective of this exploratory 8-week pilot study is to evaluate the safety and efficacy of buspirone for the treatment of anxiety in youth (ages 6-17 ...