Study of Intranasal Ketamine for Social Impairment in Autism Spectrum Disorder

NCT ID: NCT02611921

Last Updated: 2018-10-12

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-12-22
• Study Completion Date: 2018-05-07

Brief Summary

The purpose of the study is to determine if intranasal ketamine shows initial evidence of safety, tolerability and efficacy for the treatment of social impairment in individuals with Autism Spectrum Disorder.

Detailed Description

To address the significant need for effective treatment of core symptoms of Autism Spectrum Disorder (ASD), this trial is designed as a double-blind, placebo-controlled crossover pilot study of intranasal ketamine in 24 individuals with ASD ages 12- 30 years using a novel quantitative eye-tracking outcome measure to assess impact of the drug on social impairment. Additionally, to develop a ketamine-focused personalized medicine approach in ASD, the investigators will include pharmacokinetic, molecular pharmacodynamic, and electrophysiological assessments into initial systematic study.

Conditions

Autism Spectrum Disorder

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Crossover Group: Ketamine verses Placebo

Phase 1: Two ascending doses of intranasal ketamine

Two week washout

Phase 2: Two doses of placebo

Group Type: EXPERIMENTAL

Ketamine

Intervention Type: DRUG

Ketamine will be compounded into a mucosal atomization device and self-administered or administered with assistance of a caregiver/study coordinator

Placebo

Intervention Type: DRUG

Placebo will delivered as an atomized saline spray which will be self-administered or administered with assistance of a caregiver/study coordinator

Crossover Group: Placebo verses Ketamine

Phase 1: Two doses of placebo

Two week washout

Phase 2: Two ascending doses of intranasal ketamine

Group Type: EXPERIMENTAL

Ketamine

Intervention Type: DRUG

Ketamine will be compounded into a mucosal atomization device and self-administered or administered with assistance of a caregiver/study coordinator

Placebo

Intervention Type: DRUG

Placebo will delivered as an atomized saline spray which will be self-administered or administered with assistance of a caregiver/study coordinator

Interventions

Ketamine

Ketamine will be compounded into a mucosal atomization device and self-administered or administered with assistance of a caregiver/study coordinator

Intervention Type: DRUG

Placebo

Placebo will delivered as an atomized saline spray which will be self-administered or administered with assistance of a caregiver/study coordinator

Intervention Type: DRUG

Other Intervention Names

Ketalar; Saline

Eligibility Criteria

Inclusion Criteria

• Ages 12 to 30 years old.
• Weight equal to or greater than 50 kg.
• General good health as determined by physical exam, medical history, laboratory work up, and EKG.
• Diagnostic and Statistical Manual of Mental Disorders 5th Edition diagnosis of autism spectrum disorder (not associated with Fragile X Syndrome or other known genetic syndrome) as confirmed by the Autism Diagnostic Observation Schedule at screen or previous (within last 5 years) if available.
• Valid Intelligence Quotient (IQ) score greater than or equal to 50 as confirmed via testing (Leiter-3) at screen or previous (within last 5 years, any valid testing acceptable).
• Clinical Global Impressions-Severity score of 4 (Moderately Ill).
• Score of 10 on the Social Withdrawal subscale of the Aberrant Behavior Checklist.
• Stable dosing of all concomitant psychotropic medications for five half-lives prior to screening visit and during the study.
• Presence of parent/guardian or significant other or caregiver willing to serve as informant for behavioral outcome measures.

Exclusion Criteria

• Presence of co-morbid schizophrenia, schizoaffective disorder, bipolar disorder with psychosis, bipolar disorder or psychosis not otherwise specified. Comorbid diagnoses determined by psychiatrist clinical interview and use of Diagnostic and Statistical Manual of Mental Disorders 5th Edition diagnostic criteria.
• History of drug or alcohol abuse.
• Presence of cardiac disease including coronary artery disease, congestive heart failure, or uncontrolled hypertension per medical history (individuals with ≥ 2 blood pressure readings of ≥140/90 during screen/baseline will be excluded).
• Airway instability, tracheal surgery, or tracheal stenosis per medical history.
• Central nervous system masses or hydrocephalus per medical history.
• Porphyria, thyroid disorder, or thyroid medication use per medical history.
• Glaucoma or other cause of increased intraocular pressure per medical history.
• Allergy to ketamine.
• Current use of drugs with concomitant modification of non-competitive N-methyl-D-aspartate glutamate activity (acamprosate, amantadine, memantine, d-cycloserine etc.)
• For female subjects of child bearing potential, a positive pregnancy test.
• Any major chronic medical or chronic respiratory illness considered to be uncontrolled by the Principal Investigator.
• Inability to tolerate study procedures or study drug per the discretion of the Principal Investigator.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 30 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Roivant Sciences, Inc.

INDUSTRY

Sponsor Role: collaborator

Cures Within Reach

OTHER

Sponsor Role: collaborator

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Logan K Wink, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Locations

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, United States

Countries

United States

Other Identifiers

2015-2494

Identifier Type: -

Identifier Source: org_study_id