Autism Spectrum DisorderPhase 1NCT02611921
Study of Intranasal Ketamine for Social Impairment in Autism Spectrum Disorder
A Phase 1 study testing Ketamine in people with Autism Spectrum Disorder, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 30 years
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- Ketamine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Dec 2015to 7 May 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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).
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: Intranasal Ketamine Use in Autism Spectrum Disorder: A Placebo-Controlled Crossover Pilot Study
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.
Read the 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
- Being tested
- Ketamine, Placebo
- Keywords
- Ketamine, Social Impairment, Autism, Autistic Disorder
- Medical subject headings
- Autism Spectrum Disorder, Autistic Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KetamineDrug
Also called Ketalar
Ketamine will be compounded into a mucosal atomization device and self-administered or administered with assistance of a caregiver/study coordinator
PlaceboDrug
Also called Saline
Placebo will delivered as an atomized saline spray which will be self-administered or administered with assistance of a caregiver/study coordinator
| Study group | Role | What they receive |
|---|---|---|
| Crossover Group: Ketamine verses Placebo | Experimental | Phase 1: Two ascending doses of intranasal ketamine
Two week washout
Phase 2: Two doses of placebo Receives: 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 Receives: Drug: Ketamine; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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)
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
Measured: CGI-I change from baseline to final visit on day 35 ± 2 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Nov 2015 (estimated)
- Study start22 Dec 2015
- Primary completion7 May 2018
- Study completion7 May 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Roivant Sciences, Inc., Cures Within Reach
- Responsible party
- Sponsor
- Investigators
- Logan K Wink, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02611921
- Sponsor's protocol ID
- 2015-2494
- Status last verified
- Oct 2018
- First submitted
- 17 Nov 2015
- Last updated
- 12 Oct 2018
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intranasal Ketamine Use in Autism Spectrum Disorder: A Placebo-Controlled Crossover Pilot Study
- ClinicalTrials.gov ID
- NCT02611921
- Acronym / sponsor ID
- 2015-2494
- Conditions
- Autism Spectrum Disorder
- Keywords
- Ketamine, Social Impairment, Autism, Autistic Disorder
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- Roivant Sciences, Inc., Cures Within Reach
- Study start
- 22 Dec 2015 (actual)
- Primary completion
- 7 May 2018 (actual)
- Study completion
- 7 May 2018 (actual)
- First posted
- 23 Nov 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Crossover Group: Ketamine verses Placebo | Experimental | Drug: Ketamine, Drug: PlaceboPhase 1: Two ascending doses of intranasal ketamine Two week washout Phase 2: Two doses of placebo |
| Crossover Group: Placebo verses Ketamine | Experimental | Drug: Ketamine, Drug: PlaceboPhase 1: Two doses of placebo Two week washout Phase 2: Two ascending doses of intranasal ketamine |
Who may qualify
- Ages
- 12 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- 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).
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KetamineAlso: KetalarKetamine will be compounded into a mucosal atomization device and self-administered or administered with assistance of a caregiver/study coordinator | Drug | Crossover Group: Ketamine verses Placebo, Crossover Group: Placebo verses Ketamine |
| PlaceboAlso: SalinePlacebo will delivered as an atomized saline spray which will be self-administered or administered with assistance of a caregiver/study coordinator | Drug | Crossover Group: Ketamine verses Placebo, Crossover Group: Placebo verses Ketamine |
Primary outcome
Change in Social Withdrawal subscale score of the Aberrant Behavior Checklist (ABC)
Time frame: Social withdrawal subscale change from baseline to final visit on day 35 ± 2 days
The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
1 secondary outcome
Change in the Clinician-rated CGI Improvement scale (CGI-I)
Time frame: CGI-I change from baseline to final visit on day 35 ± 2 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Oct 2018.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Logan K Wink, MD principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Ketamine in people with Autism Spectrum Disorder, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Social Withdrawal subscale score of the Aberrant Behavior Checklist (ABC) (time frame: Social withdrawal subscale change from baseline to final visit on day 35 ± 2 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Oct 2018.