CLINICAL TRIAL

Epidiolex for Autistic Disorder

Waitlist Available · < 18 · Male · La Jolla, CA

This study is evaluating whether cannabidiol may have positive impacts for individuals with autism.

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About the trial for Autistic Disorder

Eligible Conditions
Autism Spectrum Disorder · Problem Behavior · Autism, Early Infantile · Autistic Disorder

Treatment Groups

This trial involves 2 different treatments. Epidiolex is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 & 3 and have had some early promising results.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Epidiolex
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.
placebo oral solution
DRUG

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cannabidiol
FDA approved

Side Effect Profile for Epidiolex

Epidiolex
Show all side effects
44%
Sedation
35%
Diarrhea
15%
Depression/mood issues
12%
Nausea
11%
Insomnia
9%
Abnormal liver function panel
8%
Behavior changes
8%
Common cold
8%
Ear infection
7%
Hospital admission
7%
Strep throat
7%
Abdominal pain
7%
Upper respiratory tract infection
7%
Sinusitis
7%
Dizziness
6%
Otitis media
6%
Constipation
6%
Fever
6%
Rash
4%
Headaches
4%
Flu
4%
Viral gastroenteritis
4%
Decreased appetite
4%
Ataxia
4%
Menorrhagia
4%
Abnormal CBC values
3%
Increased seizure frequency
3%
Lacerations
2%
Gastroesophageal reflux disease (GERD)
2%
Thrombocytopenia
2%
Hyponatremia
2%
Weight gain
2%
Anorexia
2%
Sleep disturbance
2%
Upper body pain
2%
Lethargic
2%
Ecchymosis
2%
Nosebleeds
2%
Urinary tract infection
1%
Wisdom teeth extraction
1%
Fracture (seizure-related)
1%
Urinary retention
1%
Pneumonia
1%
Fecal impaction
1%
Anemia
1%
Spinal fusion for scoliosis
1%
Dysphagia
1%
Aspiration pneumonia
1%
Hypokalemia
1%
Dehydration
1%
Elective endoscopy
1%
Gastrostomy tube (G-tube) placement
1%
Obstructive sleep apnea
1%
Leukopenia
1%
Sty
1%
Distended gallbladder
1%
Loose stool
1%
Malnutrition
1%
Concussion
1%
Increased appetite
1%
Hair loss
1%
Tooth abscess
1%
Tonsilitis
1%
Loss of motor control
1%
Strep pharyngitis
1%
Homicidal thoughts
1%
Croup
1%
Pharyngitis
1%
Memory loss
1%
Double vision
1%
Hypoxemia
1%
Proteinuria
1%
Ingrown toenail
1%
Nasal congestion
1%
Acne
1%
Hematoma
1%
Seasonal allergies
1%
Cellulitis
1%
Eczema
1%
Ear tube placement
1%
Port placement
1%
Abnormal liver funtion panel
1%
Conjunctivitis
1%
Diaper rash
Sedation
44%
Diarrhea
35%
Depression/mood issues
15%
Nausea
12%
Insomnia
11%
Abnormal liver function panel
9%
Behavior changes
8%
Common cold
8%
Ear infection
8%
Hospital admission
7%
Strep throat
7%
Abdominal pain
7%
Upper respiratory tract infection
7%
Sinusitis
7%
Dizziness
7%
Otitis media
6%
Constipation
6%
Fever
6%
Rash
6%
Headaches
4%
Flu
4%
Viral gastroenteritis
4%
Decreased appetite
4%
Ataxia
4%
Menorrhagia
4%
Abnormal CBC values
4%
Increased seizure frequency
3%
Lacerations
3%
Gastroesophageal reflux disease (GERD)
2%
Thrombocytopenia
2%
Hyponatremia
2%
Weight gain
2%
Anorexia
2%
Sleep disturbance
2%
Upper body pain
2%
Lethargic
2%
Ecchymosis
2%
Nosebleeds
2%
Urinary tract infection
2%
Wisdom teeth extraction
1%
Fracture (seizure-related)
1%
Urinary retention
1%
Pneumonia
1%
Fecal impaction
1%
Anemia
1%
Spinal fusion for scoliosis
1%
Dysphagia
1%
Aspiration pneumonia
1%
Hypokalemia
1%
Dehydration
1%
Elective endoscopy
1%
Gastrostomy tube (G-tube) placement
1%
Obstructive sleep apnea
1%
Leukopenia
1%
Sty
1%
Distended gallbladder
1%
Loose stool
1%
Malnutrition
1%
Concussion
1%
Increased appetite
1%
Hair loss
1%
Tooth abscess
1%
Tonsilitis
1%
Loss of motor control
1%
Strep pharyngitis
1%
Homicidal thoughts
1%
Croup
1%
Pharyngitis
1%
Memory loss
1%
Double vision
1%
Hypoxemia
1%
Proteinuria
1%
Ingrown toenail
1%
Nasal congestion
1%
Acne
1%
Hematoma
1%
Seasonal allergies
1%
Cellulitis
1%
Eczema
1%
Ear tube placement
1%
Port placement
1%
Abnormal liver funtion panel
1%
Conjunctivitis
1%
Diaper rash
1%
This histogram enumerates side effects from a completed 2019 Phase 1 trial (NCT02695537) in the Epidiolex ARM group. Side effects include: Sedation with 44%, Diarrhea with 35%, Depression/mood issues with 15%, Nausea with 12%, Insomnia with 11%.

Eligibility

This trial is for male patients aged 18 and younger. There are 7 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
The following behaviors occur more than six times per week in any setting: aggressive behaviors and self-injurious behaviors. show original
characterize autism spectrum disorder (ASD) in a substantial number of children Many children who have autism spectrum disorder exhibit repetitive behaviors, such as hand flapping, running in circles, jumping repeatedly, and waving fingers in front of eyes, on a daily basis and these behaviors persist for at least 5 to 10 minutes. show original
Some combination of the activities mentioned above is what significantly contributes to the child's inability to function, as reported by the parents and agreed on by the clinician based on history and/or direct observation. show original
and impulsiveness (extreme difficulty waiting for anything, blurting out comments, interrupting others, etc.) are seen in about 60% of children with ADHD show original
Boys ages 7-14 years
This text is about a person who has been diagnosed with autism based on ADOS testing. show original
The autism severity is assessed as being severe, with substantial behavioral problems. show original
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
Screening: ~3 weeks
Treatment: Varies
Reporting: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks).
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Epidiolex will improve 3 primary outcomes, 6 secondary outcomes, and 1 other outcome in patients with Autistic Disorder. Measurement will happen over the course of Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks).

Magnetic Resonance Spectroscopy
START TO FINISH OF PERIOD 1 (8 WEEKS) AND START TO FINISH OF PERIOD 2 (8 WEEKS)
Analyses will include levels of GABA and other compounds that can be identified in MR spectra; changes in those compounds with treatment; and connectivity of different brain regions before and after treatment. MRS will examine the peaks of various neurotransmitters (glutamate, glutamine, markers of neuronal integrity (NAA) and markers of metabolic function (lactate, alanine).
START TO FINISH OF PERIOD 1 (8 WEEKS) AND START TO FINISH OF PERIOD 2 (8 WEEKS)
Total Score on Peabody Picture Vocabulary test (PPVT-4)
START TO FINISH OF PERIOD 1 (8 WEEKS) AND START TO FINISH OF PERIOD 2 (8 WEEKS)
The PPVT-4 is a measure of receptive vocabulary for Standard American English. For this study we will use standard scores.
START TO FINISH OF PERIOD 1 (8 WEEKS) AND START TO FINISH OF PERIOD 2 (8 WEEKS)
Total Score on Self Harm Inventory
START TO FINISH OF PERIOD 1 (8 WEEKS) AND START TO FINISH OF PERIOD 2 (8 WEEKS)
This is a one-page, 22-item questionnaire that poses yes/no questions about the subject's history of self-harm. Total score is the summary of "yes" responses.
START TO FINISH OF PERIOD 1 (8 WEEKS) AND START TO FINISH OF PERIOD 2 (8 WEEKS)
Total Score on Expressive One Word Picture Vocabulary test, Fourth Edition (EOWPVT-4)
START TO FINISH OF PERIOD 1 (8 WEEKS) AND START TO FINISH OF PERIOD 2 (8 WEEKS)
This test assesses the individual's ability to name a variety of objects, actions, and concepts. Participants in this study are expected to have limited verbal abilities. This test will evaluate the degree of impairment at baseline and then following treatment. Administration takes less than twenty minutes.
START TO FINISH OF PERIOD 1 (8 WEEKS) AND START TO FINISH OF PERIOD 2 (8 WEEKS)
Autism Diagnostic Observation Scale- 2 (ADOS-2)
START TO FINISH OF PERIOD 1 (8 WEEKS) AND START TO FINISH OF PERIOD 2 (8 WEEKS)
This a widely used scale that diagnoses autism spectrum disorders across individuals with a broad range of ages, developmental levels, and language abilities. It is a semi-structured assessment and consists of behavioral observation and coding of relevant behaviors. The ADOS-2 evaluates communication, social interaction, and imaginative play. The child is asked to complete various activities and then rated on their performance by the tester. Time of administration is 40-60 minutes. Range of scores is 1-10. Changes in score from baseline to end of treatment will be estimated - one observed during the period of treatment with the Placebo and another during the CBD treatment - with negative value representing improvement in negative behaviors and positive value representing worsening of those characteristics.
START TO FINISH OF PERIOD 1 (8 WEEKS) AND START TO FINISH OF PERIOD 2 (8 WEEKS)
Total Score on Child Behavior Checklist (CBCL)
START TO FINISH OF PERIOD 1 (8 WEEKS) AND START TO FINISH OF PERIOD 2 (8 WEEKS)
The CBCL is a widely used test that assesses problem behaviors in children by posing questions to the parent or caregiver. It poses questions about a wide range of potential negative behaviors including aggression, rule-breaking, and social problems. The school-aged scale, which will be used in this study, consists of 118 questions. The CBCL has 8 syndrome scales, including Aggressive Behavior, Anxiety/Depressed, Attention Problems, Rule-breaking, Somatic Complaints, Social Problems, Thought Problems, and Withdrawn/Depressed. Changes in score from baseline to end of treatment will be estimated - one observed during the period of treatment with the Placebo and another during the CBD treatment - with negative value representing improvement in negative behaviors and positive value representing worsening of those characteristics.
START TO FINISH OF PERIOD 1 (8 WEEKS) AND START TO FINISH OF PERIOD 2 (8 WEEKS)
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Who is running the study

Principal Investigator
D. T.
Prof. Doris Trauner, MD
University of California, San Diego

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is autistic disorder?

The current literature reviews the clinical characteristics of the autism spectrum and the diagnostic and conceptual status of Asperger syndrome, and highlights some key differences between the two disorders. The differential diagnostic implications of these findings are discussed. A multisystem view of autistic disorder is emphasized, in distinction to a simple distinction between high and low-functioning variants of Asperger syndrome. While the present paper is in part a research paper, it reflects clinicians' experiences with patients with the autism spectrum, and therefore might be helpful to clinicians and therapists in clinical practice.

Anonymous Patient Answer

What are the signs of autistic disorder?

The most often reported signs and symptoms of autism are social impairment, impaired communication skills, abnormal eating behaviours, restricted and stereotyped behaviours and interests. Autistic disorder is diagnosed by clinical assessments of symptoms. This involves the use of structured interviews, observational techniques and, where appropriate, psychophysiological testing or scanning.

Anonymous Patient Answer

How many people get autistic disorder a year in the United States?

Overall, 3.9 million individuals are estimated to have ASD in the United States. Furthermore, it is estimated that 11.1 million individuals are diagnosed with ASD for a lifetime.

Anonymous Patient Answer

What are common treatments for autistic disorder?

There was no statistically significant difference in treatment between the two groups, so there is no need to highlight in this article the use of CBT, which is the most used method in both groups. The use of one treatment does not necessarily preclude the use of another.

Anonymous Patient Answer

Can autistic disorder be cured?

According to our data, the effectiveness of different forms of treatment does not differ. However, the outcome is not always as good as the results at admission. However, it is not possible to predict if the treatment will eradicate the symptoms (for example the need for medications or surgery).

Anonymous Patient Answer

What causes autistic disorder?

Individuals with autism may have abnormal genes influencing brain development and this may be the cause of the disorder. There is also some evidence that vitamin B12 deficiency may contribute. However, many factors are thought to be involved in the development of autism and new research shows that there is not just one factor that causes the disorder. Also, it has recently been determined the 'hot flush' is not due to an endocrine problem. Research suggests that women are more likely to develop Asperger's, and autistic children are more likely to be born prematurely, which makes its cause unclear. The use of Rett syndrome (mental retardation) to explain the causes of autism are discussed by David Amaral.

Anonymous Patient Answer

Is epidiolex typically used in combination with any other treatments?

Epidiolex is commonly used in combination with other treatments in clinical trials. The majority of these trials are for autism spectrum disorders and are reported to reflect the overall clinical outcome of the combined therapy.

Anonymous Patient Answer

What is the primary cause of autistic disorder?

We believe that there are many factors causing autistic disorder and the primary cause is unknown. Some common hypotheses and their rationale include\n\n- a defective immune system predisposing individuals to infection,\n- genetic and hormonal problems, and\n- nutritional or physical problems.\n\nThe primary cause of AD is unknown but there are many hypotheses. Many believe that this disease is multifaceted and has many causes [e.g. genetics, neurological, psychosocial, vaccine-, or environment-related and others.]\n\n- The 'immune dysregulation hypothesis' is gaining favor with many researchers.

Anonymous Patient Answer

What is epidiolex?

The current results support the view that a subgroup of individuals with autism spectrum disorders have a unique profile of the child's developmental and clinical picture that may merit consideration for treatment with this herbal remedy.

Anonymous Patient Answer

Who should consider clinical trials for autistic disorder?

Most of the patients in this study had at least one clinical feature that put them at higher risk for disease progression. No patient had clinical features that made them ineligible for these clinical trials. Those patients who were able to complete the studies reported good results and those who did not complete the studies reported poorer results. This suggests that patients should be given a clinical trial before the decision to give medication is made.

Anonymous Patient Answer

How serious can autistic disorder be?

Autism is not as dire as many believe. It can be manageable and most children and teenagers can be successfully treated early with a highly individualised program. However, some children and adults will continue to suffer from disabling cognitive difficulties, and social, behavioural and academic problems. [Power]

Anonymous Patient Answer

What is the latest research for autistic disorder?

Researchers in the area are studying various possible causes and the most likely diagnoses. Scientists are also making efforts to find a cure and discover how autism develops in a child.

Anonymous Patient Answer
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