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Study of Intranasal Ketamine for Social Impairment in Autism Spectrum Disorder

Primary Purpose

Autism Spectrum Disorder

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Ketamine

Placebo

About this trial

This is an interventional treatment trial for Autism Spectrum Disorder focused on measuring Ketamine, Social Impairment, Autism, Autistic Disorder

Eligibility Criteria

12 Years - 30 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Cincinnati Childrens Hospital Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Crossover Group: Ketamine verses Placebo

Crossover Group: Placebo verses Ketamine

Arm Description

Phase 1: Two ascending doses of intranasal ketamine Two week washout Phase 2: Two doses of placebo

Phase 1: Two doses of placebo Two week washout Phase 2: Two ascending doses of intranasal ketamine

Outcomes

Primary Outcome Measures

Change in Social Withdrawal subscale score of the Aberrant Behavior Checklist (ABC)

The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.

Secondary Outcome Measures

Change in the Clinician-rated CGI Improvement scale (CGI-I)

The CGI-I is a 7-point scale designed to measure symptomatic change at a specific time as compared to baseline. The CGI-I will be focused on the target symptoms of social impairment. CGI-I is a gold standard measure of potential change with treatment in placebo- controlled pharmacotherapy trials in ASD

Full Information

NCT ID

NCT02611921

First Posted

November 17, 2015

Last Updated

October 10, 2018

Sponsor

Children's Hospital Medical Center, Cincinnati

Collaborators

Roivant Sciences, Inc., Cures Within Reach

1. Study Identification

Unique Protocol Identification Number

NCT02611921

Brief Title

Study of Intranasal Ketamine for Social Impairment in Autism Spectrum Disorder

Official Title

Intranasal Ketamine Use in Autism Spectrum Disorder: A Placebo-Controlled Crossover Pilot Study

Study Type

Interventional

2. Study Status

Record Verification Date

October 2018

Overall Recruitment Status

Completed

Study Start Date

December 22, 2015 (Actual)

Primary Completion Date

May 7, 2018 (Actual)

Study Completion Date

May 7, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Children's Hospital Medical Center, Cincinnati

Collaborators

Roivant Sciences, Inc., Cures Within Reach

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Autism Spectrum Disorder

Keywords

Ketamine, Social Impairment, Autism, Autistic Disorder

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Crossover Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

21 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Crossover Group: Ketamine verses Placebo

Arm Type

Experimental

Arm Description

Phase 1: Two ascending doses of intranasal ketamine Two week washout Phase 2: Two doses of placebo

Arm Title

Crossover Group: Placebo verses Ketamine

Arm Type

Experimental

Arm Description

Phase 1: Two doses of placebo Two week washout Phase 2: Two ascending doses of intranasal ketamine

Intervention Type

Drug

Intervention Name(s)

Ketamine

Other Intervention Name(s)

Ketalar

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

Placebo

Other Intervention Name(s)

Saline

Intervention Description

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

Primary Outcome Measure Information:

Title

Change in Social Withdrawal subscale score of the Aberrant Behavior Checklist (ABC)

Description

The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.

Time Frame

Social withdrawal subscale change from baseline to final visit on day 35 ± 2 days

Secondary Outcome Measure Information:

Title

Change in the Clinician-rated CGI Improvement scale (CGI-I)

Description

Time Frame

CGI-I change from baseline to final visit on day 35 ± 2 days

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Maximum Age & Unit of Time

30 Years

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Logan K Wink, MD

Organizational Affiliation

Children's Hospital Medical Center, Cincinnati

Official's Role

Principal Investigator

Facility Information:

Facility Name

Cincinnati Childrens Hospital Medical Center

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45229

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

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Study of Intranasal Ketamine for Social Impairment in Autism Spectrum Disorder

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