Biomarker-Driven Pharmacological Treatment of Adolescents With Autism Spectrum Disorder With Gabapentin

Completed · Phase 4

Conditions studied: Autism Spectrum Disorder

In brief

The social cognitive deficits associated with autism spectrum disorder (ASD) are related to an imbalance in excitatory and inhibitory neurotransmission, specifically a deficit in the inhibitory neurotransmitter GABA. The investigators have used magnetic resonance spectroscopy (MRS) techniques to measure GABA in specific brain regions and have demonstrated that a single dose of gabapentin increases GABA in brain regions associated with social cognition. This study will use a biomarker-driven approach to investigate gabapentin to correct the underlying imbalance of neurotransmitters and improve the core social cognitive deficits in ASD. By using a brain-based biomarker (GABA) that is quantifiable and measurable, the investigators can target this biomarker directly and measure the impact of the treatment. This will help with the future development of targeted therapies for ASD and provide an early marker of response to aid in the selection of individuals more likely to respond to various treatments. The specific aims of this study are to: 1) determine if treatment with gabapentin sustainably increases GABA in the right anterior insula (RAI; an area of the brain involved in social cognition), 2) determine if response of RAI GABA levels to a single dose challenge of gabapentin predicts a sustained response after treatment, and 3) determine if the increase in GABA levels with gabapentin treatment translates into clinically measurable improvement in social cognition. The investigators will conduct an 8-week open-label clinical trial of gabapentin in 40 adolescents (age 13-17 years) with ASD, using MRS before and after treatment to measure GABA in the RAI (the primary outcome for the study). Before the trial, a single dose challenge of gabapentin will be used to evaluate the immediate response of GABA levels in the RAI, to determine if this predicts later response. A secondary outcome will be the clinical effects of gabapentin on social cognition. This study can demonstrate for the first time that neuroimaging biomarkers can be used to guide treatment of social cognition deficits seen in ASD and that the excitatory-inhibitory imbalance in neurotransmitters in ASD can be pharmacologically targeted. This can provide a rational basis for pharmacological treatment of the core social deficits of ASD, providing direct benefit to participants in the study as well as indirect benefit to countless patients in the future.

Key facts

Study ID: NCT05063656
Run by: David Cochran
People needed: 16
Starts: 2022-02-04
Expected to finish: 2025-12-31
Last updated by the study team: 2026-02-20

Who can join

Age: 13 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Age 13-17 years
English as primary language (both child and legal guardian)
DSM-5 criteria for Autism Spectrum Disorder
IQ >70 per Weschler Abbreviated Scale of Intelligence (WASI)
Informed assent for the study (The guardian must also give written informed consent)
For sexually active females, agreement to use two forms of contraception during trial to avoid pregnancy

You may not qualify if…

Any neurological disorder (e.g., cerebral palsy, fetal alcohol syndrome, cerebral neoplasm, bacterial meningitis, epilepsy, etc.)
Genetic disorders (e.g., Fragile X, Rett Syndrome, etc.)
Contraindications for MRI, such as metallic or electronic implants in the body, or severe claustrophobia
Unstable psychiatric disorder expected to require psychotropic medication changes over the course of the study
History of psychotic disorder
Any condition that would prevent the subject from being able to complete study protocol
Unstable medical illness such as diabetes, asthma, thyroid disease
Currently on medications that cause respiratory depression, e.g. opioids, benzodiazepines
Clinically significant suicidal ideation as assessed by the Columbia Suicide Severity Rating Scale
History of intolerance to gabapentin or pregabalin
Current substance use (including nicotine)
Current treatment with gabapentin
History of renal dysfunction
Pregnancy in female participants.

Where it is running

University of Massachusetts Medical School — Worcester, Massachusetts, United States

Full record on ClinicalTrials.gov

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