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

Autism is a pervasive developmental disorder characterized by core deficits in social behavior and communication and the presence of repetitive/stereotyped behaviors. The objective of the study is to evaluate the efficacy of Esomeprazole as a treatment for social communication deficits in children with Autism Spectrum Disorder (ASD). This prospective 12 week open-label study will invite 35 children with ASD between the ages of 2 and 6 years of age to participate.

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).

The study will require several visits with the study team that can either be conducted remotely over Zoom or done in person at Stanford University.

The child must be willing to take Esomeprazole orally for at least 8 weeks, complete diagnostic and behavioral assessments, and be free of serious medical problems.

The parent/caregiver will be required to complete questionnaires for each visit. There is also an optional research blood draw.

Investigators hope to investigate the efficacy of esomeprazole on
treating ASD symptoms. Investigators hope to demonstrate the the
use of esomeprazole results in an improvement of social communication
deficits in children with autism spectrum disorder. Currently, there
are no effective drugs for individuals with autism spectrum disorder
to treat core features including stereotypies. There are still unmet
medical needs for direct treatment of ASD patients. The proposed
study will provide the evidence for treating ASD, and more
importantly, it will pave the way for a new area of research in the
field of ASD and neurodevelopmental disorders.