Cannabidiol Oral Solution in Pediatric Subjects With Treatment- Resistant Seizure Disorders

Seizures Clinical Trial

Official title:

A Phase 1/2 Study to Assess the Pharmacokinetics and Safety of Multiple Doses of Pharmaceutical Cannabidiol Oral Solution in Pediatric Subjects With Treatment- Resistant Seizure Disorders

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02324673
• Other study ID #: INS011-14-029
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: December 12, 2014
• Last updated: May 16, 2016
• Start date: April 2015
• Est. completion date: May 2016

Study information

• Verified date: May 2016
• Source: INSYS Therapeutics Inc
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

This is a Phase 1/2, open-label, trial designed to assess the pharmacokinetics, safety, tolerability, and preliminary efficacy of 3 multiple ascending doses of Cannabidiol Oral Solution in a sequential fashion.

Participants will be pediatric (aged 1-17, inclusive), experiencing treatment-resistant seizures, and satisfy all inclusion/exclusion criteria.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 61
• Est. completion date: May 2016
• Est. primary completion date: May 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year to 17 Years

Eligibility

Inclusion Criteria:

• Meets protocol-specified criteria for qualification and contraception, including treatment-resistant seizure disorder

• Is able to speak and understand the language in which the study is being conducted, is able to understand the procedures and study requirements and has voluntarily signed and dated an informed consent form approved by the Institutional Review Board before the conduct of any study procedure

• In the opinion of the Investigator, the participants and parent(s)/caregiver(s) are willing and able to comply with the study procedures and visit schedules, including venipuncture, inpatient stay at the study center, dosing at the study center twice a day as needed while an outpatient), and the Follow-up Visits (if applicable)

Exclusion Criteria:

• Subject or parent(s)/caregiver(s) have daily commitments during the study duration that would interfere with attending all study visits

• History or current use of dietary supplements, drugs or over-the counter medications outside protocol-specified parameters

• Signs, symptoms or history of any condition that, per protocol or in the opinion of the investigator, might compromise:

1. the safety or well-being of the participant or study staff

2. the safety or well-being of the participant's offspring (such as through pregnancy or breast-feeding)

3. the analysis of results

Study Design

Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Epilepsy
• Seizures

Intervention

Drug:
• Cannabidiol Oral Solution
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).

Locations

Country	Name	City	State
United States	University of Chicago Medical Center	Chicago	Illinois
United States	Texas Scottish Rite Hospital for Children	Dallas	Texas
United States	Clinical Research Center of Nevada LLC	Las Vegas	Nevada
United States	Le Bonheur Children's Hospital	Memphis	Tennessee
United States	Miami Children's Hospital	Miami	Florida
United States	Child Neurology Center - NW F	Pensacola	Florida
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Oregon Health Services University	Portland	Oregon
United States	University of California San Francisco Medical Center	San Francisco	California
United States	Mary Bridge Children's Hospital	Tacoma	Washington

Sponsors (1)

Lead Sponsor	Collaborator
INSYS Therapeutics Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Maximum plasma concentration (Cmax)	Categories: Cmax and dose normalized Cmax (Cmax/D)	within 11 days	No
Primary	Time to Cmax (tmax)		within 11 days	No
Primary	Half life (t1/2)		within 11 days	No
Primary	Elimination rate		within 11 days	No
Primary	Oral clearance of cannabidiol		within 11 days	No
Primary	Volume of distribution of cannabidiol		within 11 days	No
Primary	Area under the plasma-concentration time curve (AUC) on Day 1	Categories: AUC(0-12), AUC(0-12)/D, AUC(0-last), AUC(0-inf), AUC(0-inf)/D for participants at least 2 years old	Day 1	No
Primary	Metabolite to parent ratio for Cmax on Day 1		Day 1	No
Primary	Metabolite to parent ratios for AUC on Day 1	Categories: AUC(0-inf), AUC(0-12)	Day 1	No
Primary	AUC on Day 10	Categories: AUC(0-12), AUC(0-12)/D	Day 10	No
Primary	Minimum plasma concentration (Cmin) on Day 10		Day 10	No
Primary	Average plasma concentration (Cavg) on Day 10		Day 10	No
Primary	Accumulation ratio for Cmax on Day 10		Day 10	No
Primary	Accumulation ratio for AUC(0-12) on Day 10		Day 10	No
Primary	Number of participants with treatment emergent adverse events		within 17 days	No
Primary	Number of participants with clinically significant changes in physical examination and/or laboratory measurements		within 11 days	No
Primary	Metabolite to parent ratio for Cmax on Day 10		Day 10	No
Primary	Metabolite to parent ratios for AUC on Day 10	Categories: AUC(0-inf), AUC(0-12)	Day 10	No
Primary	Clinical Global Impression of lmprovement (CGI-I) assessment	Categories: Assessed by parent(s)/caregiver(s), Assessed by investigator - The participant's overall clinical condition is compared to the one week period just before the start of medication (the baseline visit). The participant's condition is compared to the patient's condition at admission to the project [prior to starting treatment] on a 7-point scale, where 1=very much improved since the initiation of treatment; and 7=very much worse since the initiation of treatment.	on Day 11	No
Primary	Change from baseline in Clinical Global Impression of Severity (CGI-S) assessment	Categories: Assessed by parent(s)/caregiver(s), Assessed by investigator - The severity of the participant's illness is rated on a seven-point scale, where 1=normal, not at all ill, and 7=among the most extremely ill patients. This rating is based upon observed and reported symptoms, behavior, and function in the past seven days. The score reflects the average severity level across the seven days.	Baseline, Day 11	No
Primary	Time linearity	A linear regression analysis will assess time linearity of the individual ratios AUC(0-12) on Day 10/AUC[0-inf] on Day 1.	Day 1	No
Primary	Change from baseline in daily seizure activity	Number of seizures per day is recorded in a diary, and the change in that number between baseline and Day 11 is calculated.	Baseline, Day 11	No