Adults 18 to 30 Years Old with Autism Spectrum Disorders Needed for a Research Study
Why are we doing this research?
Cincinnati Children’s is conducting a research study, sometimes known as a clinical trial or clinical study, to learn more about an investigational drug, ketamine, and see if it may be helpful for the treatment of autism spectrum disorders (ASD). We also hope to learn more about the safety of the drug and if it works on symptoms of ASD, such as social and communication impairment.
The United States Food and Drug Administration (FDA) has approved ketamine for use as a pain relieving agent (analgesic) and pain and sensation blocker (anesthetic). Ketamine is considered investigational in this research study because the FDA has not yet approved it for the treatment of autism.
Who can participate?
Adults, 18 to 30 years old, who have an ASD may be eligible for participation.
Those who have the following will NOT be able to participate in this study:
- Schizophrenia, schizoaffective disorder, bipolar disorder or psychosis
- History of drug or alcohol abuse
- Cardiac (heart) disease including coronary artery disease, congestive heart failure or uncontrolled high blood pressure
- History of airway instability, tracheal surgery or tracheal stenosis
- Central nervous system masses or hydrocephalus
- Thyroid disorder or thyroid medication use
- Allergy to ketamine
- Any major chronic medical or chronic respiratory illness that is not well controlled
What will happen in the study?
If you qualify and you decide you want to be in this research study, you will come to Cincinnati Children’s 7 times over the next 6 to 8 weeks.
In order to make sure that you qualify, you will need to come in for a screening visit. If you are eligible to take part in this study, based on the results of the screening tests, you will be asked to return to Cincinnati Children’s to begin the study.
The following is a list of some of the tests and procedures that may happen during the study:
- Review of your medical and medication history
- Physical exam: including your height, weight blood pressure, pulse and breathing rate
- Assessments to measure your autism symptoms (completed by a psychologist)
- Electrocardiogram (ECG or EKG): measures the electrical activity (heartbeat or heart rhythm) of your heart
- Eye tracking: you will watch videos on a computer screen during which the screen tracks where your eyes go and how big your pupils are
- Transcranial magnetic stimulation (TMS): a strong hand-sized magnet, like the strong magnet used for MRI scans, is placed over your scalp and triggered to give an impulse which causes the brain cells underneath to activate, sending a message through the brain - just as brain cells activate when a person decides to move part of his or her body (optional and not required for participation)
- Electroencephalography (EEG): you will wear a headset with sensors that monitor how TMS affects the brain and “listens” to how brain communication is affected by the TMS pulse (this is optional and not required for participation)
- Questionnaires: you will answer questions about you, your autism symptoms, how you have been doing at all visits, your medications, how you are feeling, and if there are any changes in your health
- Urine samples: this will look at the health of your body, such as your kidneys
- Blood samples: the samples are taken for various reasons including to look at the health of your body, such as your kidneys, immune system and hormones; to look for any effects of the study drug on the body; to test for pregnancy (if you are female); to test for things that are specific to people with ASD, such as biomarkers (molecules in the blood called proteins and ribonucleic acid or RNA); and to measure how much study drug is in the body (this test is optional and not required for participation)
If more tests are needed, the study doctor may ask to schedule an additional study visit for you.
During the study, you will have 4 dosing visits at Cincinnati Children’s where you will receive 2 doses of intra-nasal ketamine and 2 doses of intra-nasal placebo. The study drug and placebo will both be administered just like a nasal spray.
You will be randomly assigned to 1 of 2 study groups to receive either the intra-nasal ketamine or placebo for the first 2 dosing visits. Being randomized means you will be put into a study group by chance, like flipping a coin. You will have a 50% chance of being in either group.
If you receive intra-nasal ketamine during the first 2 dosing visits, then you will receive placebo at the next 2 dosing visits.
If you receive placebo during the first 2 dosing visits, then you will receive intra-nasal ketamine at the next 2 dosing visits.
Neither you nor the researchers will know what group you are in. But, if there is an emergency, they are able to get this information.
Your vital signs (blood pressure, breathing rate, heart rate and oxygen levels) will be monitored for a minimum of 3 hours after the study drug is administered. A nurse and the study doctor will ask you questions and check to see if you have any side effects from the study drug.
A member of the study team will call you the day after to ask how you are doing. Study staff will also call 4 days after each treatment day to ask how you are doing and to ask you to complete a questionnaire.
The investigator of this research study may terminate your participation without your consent if it is determined from a safety standpoint that it is not in your best interest to continue receiving treatment. Your study doctor will discuss follow up care with you in the event this occurs.
Participants and/or parents or guardians interested in participation will be given a consent form that thoroughly explains all of the details of the study. A member of the study staff will review the consent form with you and will be sure that all of your questions are answered.
What are the good things that can happen from this research?
Your may not receive a direct medical benefit from being in this research study. We hope that the study drug will help you feel better or help to reduce your symptoms, but we don’t know if it will. We still have a lot to learn about this study drug but when we finish this research, we expect that we will know more about ketamine and autism.
This may also help other with autism later on.
What are the bad things that can happen from this research?
You may experience some risks during this study. Common risks include pain, bruising and, rarely, infection at the location where blood is taken. A trained professional will take your blood to minimize these risks and discomforts.
Other common, but mild, risks include increased blood pressure, heart rate and breathing rate. There are also limited risks associated with the TMS, such as tiredness, neck pain, arm pain, hand pain or weakness, and arm tingling. These are mild and temporary.
A detailed list of possible side effects will be provided to those participants, parents or guardians interested in knowing more about this study.
Will you/your child be paid to be in this research study?
Participants will receive $260 for their time, effort and travel for this 7-visit study.