Esomeprazole for social communication problems in young children with autism

An Open-Label Pilot Study of Esomeprazole in Children With Autism

This 12-week open-label study will try esomeprazole in children ages 2–6 with autism to see if it improves social communication.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 25 (estimated)
• Ages: 2 Years to 6 Years
• Sex: All
• Sponsor: Stanford University (Academic / other)
• Locations: 1 site (Stanford, California)
• Trial ID: NCT03866668 on ClinicalTrials.gov

What this trial studies

This Phase 2, prospective, open-label trial will enroll about 25 children with autism spectrum disorder aged 2 to 6 years and treat them with esomeprazole for 12 weeks. Diagnosis must be confirmed by DSM-5 criteria and standardized tools such as the Autism Diagnostic Interview-Revised and the Autism Diagnostic Observation Schedule or CARS-2, and caregivers provide regular outcome ratings. Participants must be physically healthy, able to swallow the medication, on stable concomitant medications, and have no planned changes in psychosocial interventions during the trial. Clinical and caregiver-rated measures of social communication will be collected throughout the treatment period to look for changes from baseline.

Who should consider this trial

Good fit: Ideal candidates are outpatient children aged 2–6 with a confirmed ASD diagnosis, who are physically healthy, can swallow the medication, have stable concomitant medications, and have a caregiver able to attend clinic visits and provide reliable ratings.

Not a fit: Children with comorbid psychotic disorders, unstable seizures or other significant active medical illnesses, those who previously had an adequate trial of esomeprazole, or those unable to swallow the drug or who will change psychosocial treatments during the study are unlikely to benefit or be eligible.

Why it matters

Potential benefit: If successful, the intervention could improve social communication skills in some young children with ASD and offer a new medication option to complement behavioral therapies.

How similar studies have performed: Using a proton pump inhibitor like esomeprazole for core ASD social communication symptoms is a novel approach without robust prior clinical trial evidence of benefit.

Eligibility criteria

Inclusion Criteria:

* outpatients 2 to 6 years of age;
* males and females who are physically healthy;
* diagnosis of autism spectrum disorder based on clinical evaluation and DSM-5 criteria, and confirmed using the Autism Diagnostic Interview-Revised, and the Autism Diagnostic Observation Schedule or Childhood Autism Rating Scale second edition (CARS-2)
* care provider who could reliably bring subject to clinic visits, could provide trustworthy ratings, and interacted with subject on a regular basis;
* ability of subject to swallow the compound;
* stable concomitant medications for at least 2 weeks (4 weeks if patient took fluoxetine);
* no planned changes in psychosocial interventions during the open-label trial.

Exclusion Criteria:

* DSM-5 diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder not otherwise specified;
* prior adequate trial of Esomeprazole;
* active medical problems such as unstable seizures, or significant physical illness (e.g., serious liver or renal pathology).

Where this trial is running

Stanford, California

• Stanford University School of Medicine — Stanford, California, United States (Recruiting)

Study contacts

• Principal investigator: Antonio Hardan, MD — Stanford University
• Study coordinator: Nina Raman

Conditions Autism; Autism Spectrum Disorder; Esomeprazole