40 Participants Needed

Gabapentin for Autism

LV
DC
AM
TM
Overseen ByTaylor Merk
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 3 JurisdictionsThis treatment is already approved in other countries

Trial Summary

What is the purpose of this trial?

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.

Do I need to stop my current medications to join the trial?

The trial does not specify if you need to stop all current medications, but you cannot participate if you are on medications that cause respiratory depression, like opioids or benzodiazepines, or if you are currently being treated with gabapentin.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are currently taking medications that cause respiratory depression or if you are already on gabapentin.

What data supports the idea that Gabapentin for Autism is an effective treatment?

The available research does not provide specific data supporting the effectiveness of Gabapentin for treating autism. Instead, it discusses other drugs like risperidone and haloperidol, which have shown some positive effects on symptoms associated with autism. However, these drugs can have significant side effects. Overall, there is no consensus or strong evidence from controlled studies validating the effectiveness of Gabapentin or other drugs specifically for autism.12345

What safety data exists for gabapentin treatment?

Gabapentin has been studied for safety in various contexts, primarily as an add-on therapy for partial seizures. Common adverse effects include somnolence, dizziness, and fatigue. In clinical trials, adverse events were reported in 69% of patients taking 900 mg/day and 64% taking 1,200 mg/day, compared to 52% in the placebo group. No significant hepatic or hematopoietic effects were observed. Additionally, there are reports of hypoglycemia as a potential adverse effect, particularly in patients with diabetes or on dialysis.678910

Is gabapentin generally safe for humans?

Gabapentin has been studied for safety in people with seizures and other conditions. Common side effects include sleepiness, dizziness, and tiredness. Some reports mention low blood sugar in certain patients, but no major liver or blood-related issues were found in clinical studies.678910

Is the drug Gabapentin promising for treating autism?

The research articles mainly discuss Gabapentin as a drug for epilepsy and pain management, not autism. They highlight its effectiveness in treating seizures and certain types of pain, but do not provide evidence or claims about its use for autism. Therefore, based on the available information, Gabapentin is not shown to be a promising treatment for autism.1112131415

How is the drug Gabapentin unique for treating autism?

Gabapentin is unique because it is primarily used as an anti-epileptic drug and for treating nerve pain, but its use in autism is novel as there are no standard treatments specifically for autism. It has a low incidence of side effects and minimal drug interactions, which may make it a promising option for managing symptoms in autism.1112131415

Research Team

DC

David Cochran, MD

Principal Investigator

UMass Medical School

Eligibility Criteria

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.

Inclusion Criteria

For sexually active females, agreement to use two forms of contraception during trial to avoid pregnancy
Meets DSM-5 criteria for Autism Spectrum Disorder
IQ >70 per Weschler Abbreviated Scale of Intelligence (WASI)
See 3 more

Exclusion Criteria

History of psychotic disorder
History of intolerance to gabapentin or pregabalin
I am taking medications that can slow my breathing.
See 11 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Acute Response Assessment

Single dose challenge of gabapentin to evaluate immediate GABA response

1 day
1 visit (in-person)

Treatment

8-week open-label treatment with gabapentin to assess sustained GABA response and clinical improvements

8 weeks
Visits at baseline, Weeks 2, 4, and 8

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Gabapentin
Trial OverviewThe 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).
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Gabapentin Open-label treatmentExperimental Treatment1 Intervention
8-week treatment with gabapentin

Gabapentin is already approved in United States, European Union, Canada for the following indications:

🇺🇸
Approved in United States as Neurontin for:
  • Postherpetic neuralgia
  • Partial-onset seizures
🇪🇺
Approved in European Union as Gabapentin for:
  • Peripheral neuropathic pain
  • Partial-onset seizures
🇨🇦
Approved in Canada as Gabapentin for:
  • Postherpetic neuralgia
  • Partial-onset seizures

Find a Clinic Near You

Who Is Running the Clinical Trial?

David Cochran

Lead Sponsor

Trials
1
Recruited
40+

Eagles Autism Foundation

Collaborator

Trials
1
Recruited
40+

Findings from Research

While there are no medications specifically approved for treating autism, some medications like haloperidol and risperidone can help manage associated symptoms, although they may cause significant side effects.
Current treatments focus on alleviating specific symptoms, such as hyperactivity, rather than addressing the core social and communication challenges of autism, as no medications have been proven effective for those core symptoms.
Pharmacotherapeutic management of autism.Posey, DJ., McDougle, CJ.[2022]
In a double-blind clinical trial involving 40 children with autism, adding amantadine to risperidone significantly reduced hyperactivity and irritability compared to a placebo, indicating its potential effectiveness as an adjunct treatment.
The study found no significant difference in adverse effects between the amantadine and placebo groups, suggesting that amantadine is generally well tolerated in this population.
Double-blind, placebo-controlled trial of risperidone plus amantadine in children with autism: a 10-week randomized study.Mohammadi, MR., Yadegari, N., Hassanzadeh, E., et al.[2013]
In a 12-week randomized, placebo-controlled trial involving 121 children with autism, memantine did not show significant clinical efficacy in improving symptoms, although it was well-tolerated with only mild to moderate adverse effects reported.
The study found that while memantine was safe and tolerable, it did not lead to improvements in the primary outcome measure of social responsiveness, and one communication measure even worsened in the memantine group compared to placebo.
Safety and Efficacy of Memantine in Children with Autism: Randomized, Placebo-Controlled Study and Open-Label Extension.Aman, MG., Findling, RL., Hardan, AY., et al.[2022]

References

Pharmacotherapeutic management of autism. [2022]
Double-blind, placebo-controlled trial of risperidone plus amantadine in children with autism: a 10-week randomized study. [2013]
Safety and Efficacy of Memantine in Children with Autism: Randomized, Placebo-Controlled Study and Open-Label Extension. [2022]
[Review of psychopharmacological treatments in adolescents and adults with autistic disorders]. [2007]
Efficacy and safety of naltrexone use in pediatric patients with autistic disorder. [2013]
Outcome evaluation of gabapentin as add-on therapy for partial seizures. "NEON" Study Investigators Group. Neurontin Evaluation of Outcomes in Neurological Practice. [2018]
Gabapentin as add-on therapy in children with refractory partial seizures: a 12-week, multicentre, double-blind, placebo-controlled study. Gabapentin Paediatric Study Group. [2019]
Restoring invisible and abandoned trials of gabapentin for neuropathic pain: a clinical and methodological investigation. [2022]
Patient Case Report: Gabapentin-Induced Hypoglycemia. [2022]
10.United Statespubmed.ncbi.nlm.nih.gov
Gabapentin (Neurontin) as add-on therapy in patients with partial seizures: a double-blind, placebo-controlled study. The International Gabapentin Study Group. [2019]
Gabapentin--a new antiepileptic drug. [2018]
A rare case of a gabapentin-induced cardiomyopathy. [2019]
Gabapentin: a unique anti-epileptic agent. [2018]
14.United Statespubmed.ncbi.nlm.nih.gov
Pharmacokinetics, efficacy, and tolerability of a once-daily gastroretentive dosage form of gabapentin for the treatment of postherpetic neuralgia. [2022]
[Gabapentin (Neurontin): a new possibility in the add-on therapy of partial epilepsies]. [2018]