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

NCT03355300 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: INS011-17-113
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 9

Brief Summary

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.

Conditions

Childhood Absence Epilepsy

Outcome Measures

Primary Outcomes (1)

• 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)

Study Arms (1)

Cannabidiol Oral Solution

EXPERIMENTAL

Cannabidiol Oral solution, dose as assigned in INS-17-103.

Drug: Cannabidiol Oral Solution

Interventions

Cannabidiol Oral Solution DRUG

An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).

Cannabidiol Oral Solution

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Completed all activities through Visit 6 (End of Study) of INS011-17-103.
• 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.
• 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.
• 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.
• 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.

You may not qualify if:

• Participant or parent(s)/caregiver(s) have daily commitments during the study duration that would interfere with attending all study visits.
• Experienced an anoxic episode related to study drug requiring resuscitation during their previous study.
• 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.
• Evidence of other clinically significant disease such as unstable hepatic, hematological, renal, cardiovascular, gastrointestinal, immunological, or pulmonary diseases or ongoing malignancies.
• Compromised respiratory function or severe respiratory insufficiency.
• Clinically significant abnormal laboratory values within the past 14 days.
• In the opinion of the investigator, the participant is unsuitable in any other way to participate in this study.

Sponsors & Collaborators

• Radius Pharmaceuticals, Inc.: lead
• Benuvia Therapeutics Inc.: collaborator

Study Sites (9)

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Pediatric Epilepsy and Neurology Specialists

Tampa, Florida, 33609, United States

Location

Clinical Integrative Research Center of Atlanta

Atlanta, Georgia, 30328, United States

Location

Akron Children's Hospital

Akron, Ohio, 44308, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

Location

Children's Specialty Group, Division of Child & Adolescent Neurology

Norfolk, Virginia, 23510, United States

Location

Institute for Research and Innovation | MultiCare Health System

Tacoma, Washington, 98405, United States

Location

MeSH Terms

Conditions

Epilepsy, Absence

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Limitations and Caveats

This study was prematurely terminated by the Sponsor. Participants ongoing at the time of discontinuation of the study are neither considered completed or discontinued.

Results Point of Contact

• Title: Tarek El Akkad, Head of Clinical Development
• Organization: Radius Pharmaceuticals, Inc.

telakkad@radiuspharm.com

6175514000

Study Officials

• Ahmed Elkashef, MD

  INSYS Therapeutics Inc

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 9, 2017
• First Posted: November 28, 2017
• Study Start: February 20, 2018
• Primary Completion: June 24, 2019
• Study Completion: June 28, 2019
• Last Updated: June 18, 2023
• Results First Posted: June 18, 2023

Record last verified: 2023-05

Locations

US (9)