Cannabidiol Oral Solution in Pediatric Participants With Treatment-resistant Seizure Disorders

INSYS Therapeutics

Status and phase: Completed; Phase 2; Phase 1

Conditions: Seizures

Treatments: Drug: Cannabidiol Oral Solution

Study type: Interventional

Funder types: Industry

Identifiers

NCT02324673

INS011-14-029

Details and patient eligibility

About

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.

Enrollment: 61 patients

Sex: All

Ages: 1 to 17 years old

Volunteers: No Healthy Volunteers

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

• Participant 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

---

61 participants in 3 patient groups

Low Dose Cannabidiol Oral Solution [10 mg/kg/day]

Experimental group

Description:

Low Dose \[10 milligrams/kilogram/day (mg/kg/day)\] oral solution containing pharmaceutical grade cannabidiol (nonplant-based). Starting dose of 5 mg/kg in the morning on Day 1 followed by total dose of 10 mg/kg/day (5 mg/kg in the morning and 5 mg/kg in the evening) on Days 4 to 10.

Treatment:

Drug: Cannabidiol Oral Solution

Mid Dose Cannabidiol Oral Solution [20 mg/kg/day]

Experimental group

Description:

Mid Dose \[20 mg/kg/day\] oral solution containing pharmaceutical grade cannabidiol (nonplant-based). Starting dose of 10 mg/kg in the morning on Day 1 followed by total dose of 20 mg/kg/day (10 mg/kg in the morning and 10 mg/kg in the evening) on Days 4 to 10.

Treatment:

Drug: Cannabidiol Oral Solution

High Dose Cannabidiol Oral Solution [40 mg/kg/day]

Experimental group

Description:

High Dose \[40 mg/kg/day\] oral solution containing pharmaceutical grade cannabidiol (nonplant-based). Starting dose of 20 mg/kg in the morning on Day 1 followed by total dose of 40 mg/kg/day (20 mg/kg in the morning and 20 mg/kg in the evening) on Days 4 to 10.

Treatment:

Drug: Cannabidiol Oral Solution