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
Sponsored by
About this trial
This is an interventional treatment trial for Autism Spectrum Disorder focused on measuring Ketamine, Social Impairment, Autism, Autistic Disorder
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.
Sites / Locations
- Cincinnati Childrens Hospital Medical Center
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
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
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
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
No
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
No
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Study of Intranasal Ketamine for Social Impairment in Autism Spectrum Disorder
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