Study of Intranasal Ketamine for Social Impairment in Autism Spectrum Disorder

NCT02611921 | Completed Phase 1 DMC

• 1 other identifier: 2015-2494
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

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.

Conditions

Autism Spectrum Disorder

Keywords

Ketamine; Social Impairment; Autism; Autistic Disorder

Outcome Measures

Primary Outcomes (1)

• 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.

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

Secondary Outcomes (1)

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

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

Study Arms (2)

Crossover Group: Ketamine verses Placebo

EXPERIMENTAL

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

Drug: Ketamine; Drug: Placebo

Crossover Group: Placebo verses Ketamine

EXPERIMENTAL

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

Drug: Ketamine; Drug: Placebo

Interventions

Ketamine DRUG

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

Also known as: Ketalar

Crossover Group: Ketamine verses Placebo; Crossover Group: Placebo verses Ketamine

Placebo DRUG

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

Also known as: Saline

Crossover Group: Ketamine verses Placebo; Crossover Group: Placebo verses Ketamine

Eligibility Criteria

• Age: 12 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• Roivant Sciences, Inc.: collaborator
• Cures Within Reach: collaborator

Study Sites (1)

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder

Interventions

Ketamine; Sodium Chloride

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Cyclohexanes; Cycloparaffins; Hydrocarbons, Alicyclic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Officials

• Logan K Wink, MD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 17, 2015
• First Posted: November 23, 2015
• Study Start: December 22, 2015
• Primary Completion: May 7, 2018
• Study Completion: May 7, 2018
• Last Updated: October 12, 2018

Record last verified: 2018-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)