Clinical trial NCT03699527: Medical Cannabis Registry and Pharmacology
Official title: Medical Cannabis Registry and Pharmacology
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Autism
ASD
Keywords: medical cannabis
Lead sponsor: Children's Hospital of Philadelphia
Sponsor class: OTHER
Collaborators: name: Zelda Therapeutics
First posted: 2018-10-09
Enrollment: 119
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Those diagnosed with autism who are under age 21.

Brief summary: The overall goals of this research is to describe the 1) natural history of current use and disposition of medical cannabis products including Cannabidiol (CBD) products, being administered to children as standard of care for the treatment of Autism Spectrum Disorder (ASD), 2) understand the pharmacokinetic and pharmacodynamics of medical cannabis products and 3) provide educational feedback on what is learned to families and care providers to provide evidenced based dosing guidance for these products to the pediatric community.

Detailed description: Part 1 of the study will consist of building a patient registry. Eligible patients will be identified and consented for study participation.Parents/legal guardians will be asked the survey related questions from Social Communication Questionnaire (SCQ), personality structure questionnaire (PSQ), Assessment Battery for Children (ABC2), Behavior Rating Inventory of Executive Function (BRIEF2), and the Screen for Child Anxiety Related Disorders (SCARED) (parent version) if appropriate and ASHD5 Rating for either 5-10 year old or 11-17 year old. The description of each survey is below with a link to more information to the ASHD5. All additional questionnaires are already validated by the Institutional Review Board (IRB).

Registry information (as detailed below) will be gathered and entered into the registry.Each subject will be re-contacted approximately every 12 weeks to ask whether medications (cannabis related or other) have changed, efficacy/side effects have changed, or there have been changes in care or medical history. These new data will be recorded in the registry. Subjects may continue in the study for the duration of medical cannabis use, until they remove themselves from the study, or the study closes. All data will remain coded with a Master log of Subject identification numbers (IDs).

Part 2 of the study consists of pharmacokinetic (PK) evaluation of select subjects.

PK sampling of a select formulations will begin subjects are enrolled into the Registry and have consented to PK sampling. . Subjects will have the opportunity to opt in or out of Part 2 upon the initial informed consent process for Part 1.

Part 3 of study related activities will include analysis and summary of the data on approximately a six month basis.

The investigative team will provide study participants with coded summary statistics of medical cannabis products use in the region (either through email, postal mail). This will also be presented at a community-based meeting that all study participants are invited to attend. The study team recognized that participation in a community event will result in loss of de-identification, but also feel that this will occur on a voluntary basis for all study participants.

The study team hypothesizes that the community educational programs will provide study participants an opportunity to connect with others in the community, share concerns, learn from others and allow the development of community-based support systems.

Eligibility criteria: Inclusion Criteria

1. Individuals who consume cannabis products in a state which has legalized medical cannabis for the treatment of ASD related symptoms
2. Individuals who have a diagnosis of autism spectrum disorder

Exclusion Criteria

1. Consumption of cannabis products that are not obtained legally
2. Non English speaking individuals
3. Greater than 21 years of age for entry into Part 2 of the study

Primary outcomes:
measure: Number of children with ASD who use medical cannabis
timeFrame: 5 years
measure: Maximum Cannabinoid concentrations in pediatric patients with ASD
timeFrame: 5 years
Secondary outcomes:
measure: Number of families who complete the educational program to assess epidemiologic outcomes
timeFrame: 5 years
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
Study officials:
affiliation: Children's Hospital of Philadelphia
name: Athena Zuppa, MD, MSCE
role: PRINCIPAL_INVESTIGATOR
References: