Epidiolex for Autism, Early Infantile

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Autism, Early Infantile+6 More
Epidiolex - Drug
Eligibility
< 18
Male
What conditions do you have?
Select

Study Summary

This trial is testing whether cannabidiol can help reduce severe behavioral problems in people with autism.

Eligible Conditions
  • Autism, Early Infantile
  • Autism Spectrum Disorders
  • Autism Spectrum Disorder

Treatment Effectiveness

Study Objectives

3 Primary · 6 Secondary · Reporting Duration: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)

Week 8
Autism Diagnostic Observation Scale- 2 (ADOS-2)
Magnetic Resonance Spectroscopy
Total Score on Aberrant Behavior Checklist (ABC)
Total Score on Child Behavior Checklist (CBCL)
Total Score on Children's Sleep Habits Questionnaire (CSHQ)
Total Score on Expressive One Word Picture Vocabulary test, Fourth Edition (EOWPVT-4)
Total Score on Peabody Picture Vocabulary test (PPVT-4)
Total Score on Repetitive Behavior Scale-Revised (RBS-R)
Total Score on Self Harm Inventory
Total Score on Social Responsiveness Scale

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Side Effects for

Epidiolex
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%Dizziness
7%Hospital admission
7%Strep throat
7%Upper respiratory tract infection
7%Abdominal pain
7%Sinusitis
6%Fever
6%Constipation
6%Otitis media
6%Rash
4%Ataxia
4%Decreased appetite
4%Menorrhagia
4%Abnormal CBC values
4%Viral gastroenteritis
4%Flu
4%Headaches
3%Increased seizure frequency
3%Lacerations
2%Lethargic
2%Hyponatremia
2%Urinary tract infection
2%Upper body pain
2%Thrombocytopenia
2%Gastroesophageal reflux disease (GERD)
2%Weight gain
2%Anorexia
2%Nosebleeds
2%Ecchymosis
2%Sleep disturbance
1%Tooth abscess
1%Abnormal liver funtion panel
1%Proteinuria
1%Hematoma
1%Fracture (seizure-related)
1%Ingrown toenail
1%Strep pharyngitis
1%Hypokalemia
1%Homicidal thoughts
1%Acne
1%Dysphagia
1%Loss of motor control
1%Dehydration
1%Spinal fusion for scoliosis
1%Aspiration pneumonia
1%Memory loss
1%Seasonal allergies
1%Ear tube placement
1%Port placement
1%Pneumonia
1%Loose stool
1%Urinary retention
1%Double vision
1%Tonsilitis
1%Malnutrition
1%Gastrostomy tube (G-tube) placement
1%Leukopenia
1%Fecal impaction
1%Elective endoscopy
1%Obstructive sleep apnea
1%Sty
1%Distended gallbladder
1%Anemia
1%Increased appetite
1%Hair loss
1%Concussion
1%Conjunctivitis
1%Croup
1%Eczema
1%Hypoxemia
1%Pharyngitis
1%Wisdom teeth extraction
1%Cellulitis
1%Diaper rash
1%Nasal congestion
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%.

Trial Design

2 Treatment Groups

cannabidiol
1 of 2
placebo
1 of 2

Experimental Treatment

Non-Treatment Group

42 Total Participants · 2 Treatment Groups

Primary Treatment: Epidiolex · Has Placebo Group · Phase 2 & 3

cannabidiol
Drug
Experimental Group · 1 Intervention: Epidiolex · Intervention Types: Drug
placebo
Drug
PlaceboComparator Group · 1 Intervention: placebo oral solution · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cannabidiol
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)

Who is running the clinical trial?

Wholistic Research and Education FoundationUNKNOWN
University of California, San DiegoLead Sponsor
1,012 Previous Clinical Trials
1,793,970 Total Patients Enrolled
Doris Trauner, MD3.511 ReviewsPrincipal Investigator - University of California, San Diego
University of California, San Diego
1Patient Review
They were quite rude to my little ones and myself. I would not recommend this dentist to anyone.

Eligibility Criteria

Age < 18 · Male Participants · 7 Total Inclusion Criteria

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.
This text is about a person who has been diagnosed with autism based on ADOS testing.
The autism severity is assessed as being severe, with substantial behavioral problems.
You have frequent, persistent stereotypies (repetitive behaviors such as hand flapping, running in circles, jumping repeatedly, waving fingers in front of eyes).
Child is so physically active that he cannot sit for meals or school work, is moving all the time, jumping off furniture, climbing onto furniture, etc.
One or more of the above activities are deemed to contribute significantly to child's inability to function by parental report and with clinician agreement based on history and/or direct observation.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 7th, 2021

Last Reviewed: November 1st, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

Who else is applying?

How old are they?
< 18100.0%
What portion of applicants met pre-screening criteria?
Did not meet criteria100.0%