What side effects are associated with this type of intervention?

Gabapentin, used to increase GABA levels and correct neurotransmitter imbalances in ASD, can cause side effects such as sedation, dizziness, and gait unsteadiness. Other common treatments for ASD include antipsychotic medications like risperidone and aripiprazole, which can lead to weight gain, drowsiness, and metabolic changes. Stimulants such as methylphenidate, used for attention deficits, may cause insomnia, decreased appetite, and increased anxiety. It's important to discuss these potential side effects with a healthcare provider to tailor the treatment plan to the individual's needs.

What prior FDA approvals exist?

The FDA has approved several medications for the treatment of Autism Spectrum Disorder (ASD), primarily focusing on managing symptoms rather than correcting neurotransmitter imbalances. Risperidone and aripiprazole are two antipsychotic medications approved for treating irritability associated with ASD. These drugs do not specifically target GABA levels but work on different neurotransmitter systems, primarily dopamine and serotonin. While Gabapentin, which increases GABA levels, is being studied for its potential to address social cognitive deficits in ASD, it is not yet FDA-approved for this indication.

What other types of research exist?

Promising alternatives to Gabapentin for treating Autism Spectrum Disorder by targeting neurotransmitter imbalances include: 1) Arbaclofen, which has shown potential in improving social behaviors by modulating GABA-B receptors. 2) Bumetanide, a diuretic that reduces intracellular chloride levels, thereby enhancing GABAergic signaling. 3) Oxytocin, which has been studied for its role in social bonding and may improve social cognition in ASD patients. These alternatives are being investigated in clinical trials and may offer new avenues for treatment in 2024.

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Gabapentin for Autism

Phase 4

Recruiting

Led By David Cochran, MD

Research Sponsored by David Cochran

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age 13-17 years

English as primary language for both child and legal guardian

Must not have

Currently on medications that cause respiratory depression, e.g. opioids, benzodiazepines

Unstable medical illness such as diabetes, asthma, thyroid disease

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 8 weeks

Awards & highlights

Pivotal Trial

Drug Has Already Been Approved

No Placebo-Only Group

Summary

This trial tests whether gabapentin can improve social skills in adolescents with autism by increasing levels of a calming brain chemical called GABA. The study involves 40 adolescents and aims to see if this treatment can correct brain chemical imbalances and improve social interactions.

Who is the study for?

This trial is for English-speaking adolescents aged 13-17 with Autism Spectrum Disorder and an IQ over 70. They must be able to give informed assent, with a guardian's consent, and not have genetic disorders like Fragile X or Rett Syndrome. Participants should not have metal implants, severe claustrophobia, unstable psychiatric conditions requiring medication changes during the study, significant medical illnesses, renal dysfunction or be pregnant.

What is being tested?

The trial tests Gabapentin in adolescents with ASD to see if it can increase GABA levels in the brain and improve social cognition. It involves an initial single dose challenge followed by an 8-week treatment period where effects on GABA levels and social cognition are measured using magnetic resonance spectroscopy (MRS).

What are the potential side effects?

Potential side effects of Gabapentin may include dizziness, sleepiness, water retention leading to swelling in limbs, difficulty walking or controlling muscle movements (ataxia), blurred vision, nausea or vomiting.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 13 and 17 years old.

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Both my child and I primarily speak English.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am taking medications that can slow my breathing.

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I do not have uncontrolled diabetes, asthma, or thyroid disease.

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I have had kidney problems in the past.

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I expect to change my psychiatric medication during the study.

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I am currently taking gabapentin.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 8 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~8 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 8 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Right Anterior Insula GABA level

Side effects data

From 2021 Phase 4 trial • 88 Patients • NCT03012815

Allergic reaction

100%

80%

60%

40%

20%

Study treatment Arm

Gabapentin

Benzodiazepine

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Gabapentin Open-label treatmentExperimental Treatment1 Intervention

8-week treatment with gabapentin

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Gabapentin

2013

Completed Phase 4

~1550

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Autism, such as Gabapentin, work by increasing levels of the inhibitory neurotransmitter GABA to correct imbalances in neurotransmission. This mechanism is significant for Autism patients because it directly targets the core social cognitive deficits associated with the disorder. By improving the balance between excitatory and inhibitory neurotransmitters, these treatments can enhance social cognition and potentially lead to better social interactions and quality of life for individuals with Autism.

Prenatal valproate in rodents as a tool to understand the neural underpinnings of social dysfunctions in autism spectrum disorder.Study protocol of the ASD-Net, the German research consortium for the study of Autism Spectrum Disorder across the lifespan: from a better etiological understanding, through valid diagnosis, to more effective health care.New directions in the treatment of autism spectrum disorders from animal model research.