Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Pediatric Participants With Treatment-Resistant Childhood Absence Seizures

A Multicenter, Open-Label, Flexible Dose Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Pediatric Patients With Treatment-Resistant Childhood Absence Seizures

The primary purpose of this study is to assess the long-term safety and tolerability of Cannabidiol Oral Solution (CBD) in pediatric participants with treatment-resistant childhood absence seizures.

Study Overview

• Status: Terminated
• Conditions: Childhood Absence Epilepsy
• Intervention / Treatment: Drug: Cannabidiol Oral Solution

• Study Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33155
      • Nicklaus Children's Hospital
    • Tampa, Florida, United States, 33609
      • Pediatric Epilepsy and Neurology Specialists
  • Georgia
    • Atlanta, Georgia, United States, 30328
      • Clinical Integrative Research Center of Atlanta
  • Ohio
    • Akron, Ohio, United States, 44308
      • Akron Children's Hospital
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health and Science University
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • Primary Children's Hospital
  • Virginia
    • Norfolk, Virginia, United States, 23510
      • Children's Specialty Group, Division of Child & Adolescent Neurology
  • Washington
    • Tacoma, Washington, United States, 98405
      • Institute for Research and Innovation | MultiCare Health System

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Completed all activities through Visit 6 (End of Study) of INS011-17-103.
2. Participant and/or parent(s)/caregiver(s) fully comprehend the informed consent form (ICF) and assent form, understand all study procedures, and can communicate satisfactorily with the investigator and study coordinator, in accordance with applicable laws, regulations, and local requirements.
3. A female participant is eligible to participate in the study if she is premenarchal, or of childbearing potential with a negative urine pregnancy test at the Screening Visit. If sexually active, she must agree to either complete abstinence from intercourse or use acceptable methods of contraception throughout the study and for 4 weeks after completion of study participation or discontinuation from investigational product.
4. A sexually active male participant or partner of enrolled participant must be willing to use acceptable methods of contraception throughout the study and for 4 weeks after completion of study participation or discontinuation from investigational product.
5. In the opinion of the investigator, the parent(s)/caregiver(s) is (are) willing and able to comply with the study procedures and visit schedules, including venipuncture, and the visit schedules.

Exclusion Criteria:

1. Participant or parent(s)/caregiver(s) have daily commitments during the study duration that would interfere with attending all study visits.
2. Experienced an anoxic episode related to study drug requiring resuscitation during their previous study.
3. Developed an adverse event thought to be related to CBD in the previous study and for whom the Investigator determines that continuing treatment with CBD would not be in the best interest of the participant.
4. Evidence of other clinically significant disease such as unstable hepatic, hematological, renal, cardiovascular, gastrointestinal, immunological, or pulmonary diseases or ongoing malignancies.
5. Compromised respiratory function or severe respiratory insufficiency.
6. Clinically significant abnormal laboratory values within the past 14 days.
7. In the opinion of the investigator, the participant is unsuitable in any other way to participate in this study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cannabidiol Oral Solution; Cannabidiol Oral solution, dose as assigned in INS-17-103.	Drug: Cannabidiol Oral Solution; An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	An AE is defined as any untoward medical occurrence in a participant administered a pharmaceutical product during the course of a clinical investigation. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of an investigational product, whether or not thought to be related to the investigational product. An SAE is any untoward medical occurrence that results in death, is life-threatening, requires the participant be at a risk of death at the time of the event, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality/birth defect, or other serious event that requires medical or surgical intervention. A summary of SAEs and all other non-serious AEs, regardless of causality, is located in the Reported AEs module.	Baseline (Visit 6 of INS011-17-103) and Visit 10 (up to approximately 54 weeks)

Collaborators and Investigators

• Sponsor: Radius Pharmaceuticals, Inc.
• Collaborators: Benuvia Therapeutics Inc.
• Investigators: Study Director: Ahmed Elkashef, MD, INSYS Therapeutics Inc

Study record dates

Study Major Dates

• Study Start (Actual): February 20, 2018
• Primary Completion (Actual): June 24, 2019
• Study Completion (Actual): June 28, 2019

Study Registration Dates

• First Submitted: November 9, 2017
• First Submitted That Met QC Criteria: November 21, 2017
• First Posted (Actual): November 28, 2017

Study Record Updates

• Last Update Posted (Estimated): June 16, 2023
• Last Update Submitted That Met QC Criteria: May 24, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epilepsy, Generalized; Epileptic Syndromes; Neurologic Manifestations; Epilepsy; Seizures; Epilepsy, Absence; Anticonvulsants; Cannabidiol
• Other Study ID Numbers: INS011-17-113

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No