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

NCT05063656 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2026-02-20

No results posted yet for this study

Summary

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.

Conditions

Interventions

DRUG

Gabapentin

8 weeks of gabapentin, titrated up to 20 mg/kg in 3 divided doses daily

Sponsors & Collaborators

  • Eagles Autism Foundation

    collaborator UNKNOWN

  • David Cochran

    lead OTHER

Principal Investigators

  • David Cochran, MD · UMass Medical School

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
13 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-02-04
Primary Completion
2025-12-31
Completion
2025-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05063656 on ClinicalTrials.gov