Cannabidiol Oral Solution as an Adjunctive Treatment for Treatment-resistant Seizure Disorder

NCT02318602 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: INS011-14-030
• Study Type: interventional
• Target: 52
• Locations: 1 country
• Sites: 11

Brief Summary

This is a multicenter, open-label trial to assess the long-term safety and efficacy of Cannabidiol Oral Solution as adjunctive therapy for pediatric participants with treatment-resistant seizure disorders, including Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS). All participants have rolled over from previous trials: INS011-14-029 (NCT02324673) and INS011-15-054 (NCT02551731).

Conditions

Seizures

Keywords

Lennox-Gastaut syndrome; Dravet syndrome; Treatment-resistant seizures

Outcome Measures

Primary Outcomes (6)

• Percentage of Participants With Adverse Events

  An Adverse Event (AE) is any untoward medical occurrence in a subject administered a pharmaceutical product. It does not necessarily have a causal relationship with this treatment.

  Up to Week 50

• Percentage of Participants With Serious Adverse Events

  A serious adverse event is any untoward medical occurrence ( whether considered to be related to investigational product or not) that at any dose results in death, is life threatening, requires inpatient hospitalization, results in disability/incapacity, is a congenital abnormality/ birth defect, or medically significant as determined by an investigator.

  Up to Week 50

• Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Values

  Laboratory values include chemistry and hematology, and urinary analysis.

  Up to Week 50

• Percentage of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Findings

  Up to Week 48

• Percentage of Participants With Clinically Significant Change From Baseline in Vital Signs

  Up to Week 50

• Change From Baseline in Trough Plasma Levels of Cannabidiol and Its 7-OH Metabolite

  Up to Week 50

Secondary Outcomes (2)

• Vineland Adaptive Behavior Scales (VABS)

  Up to Week 48

• Number of Participants With a Positive Response on the Columbia-Suicide Severity Rating Scale (C-SSRS)

  Up to Week 50

Other Outcomes (4)

• Clinical Global Impression of Severity (CGI-S)

  through study completion, up to 48 weeks or marketing approval, whichever is earlier

• Impact of Pediatric Epilepsy Scale (IPES)

  through study completion, up to 48 weeks or marketing approval, whichever is earlier

• Clinical Global Impression of Improvement (CGI-I)

  through study completion, up to 48 weeks or marketing approval, whichever is earlier

Study Arms (3)

Infants

EXPERIMENTAL

Participants 1 to\<2 years of age. Participants who completed INS011-14-029 initiated this study on the dose with which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant. The maximum daily dose was 40 mg/kg/day. Participants who enrolled from INS011-15-054 continued treatment with the dose at which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant. The total daily dose, milligrams per kilograms per day (mg/kg/day), will be evenly split between morning and evening doses (12 hours apart). If tolerability issues arise, the participant's dose may be changed at the investigator's discretion.

Drug: Cannabidiol Oral Solution

Children

EXPERIMENTAL

Participants 2 to \<12 years of age. Participants who completed INS011-14-029 initiated this study on the dose with which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant.The maximum daily dose was 40 mg/kg/day. Participants who enrolled from INS011-15-054 continued treatment with the dose at which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant. The total daily dose (mg/kg/day) will be evenly split between morning and evening doses (12 hours apart). If tolerability issues arise, the participant's dose may be changed at the investigator's discretion.

Drug: Cannabidiol Oral Solution

Adolescents

EXPERIMENTAL

Participants 12 to \<17 years of age. Participants who completed INS011-14-029 initiated this study on the dose with which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant. The maximum daily dose was 40 mg/kg/day. Participants who enrolled from INS011-15-054 continued treatment with the dose at which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant. The total daily dose (mg/kg/day) will be evenly split between morning and evening doses (12 hours apart). If tolerability issues arise, the participant's dose may be changed at the investigator's discretion.

Drug: Cannabidiol Oral Solution

Interventions

Cannabidiol Oral Solution DRUG

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

Adolescents; Children; Infants

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Completed activities through Day 11 in INS011-14-029 or Part A (Visit 6) INS011-15-054
• Informed consent/assent (as applicable) was voluntarily provided by the participant and/or parent(s)/caregiver(s) in accordance with applicable laws, regulations, and local requirements
• Is medically stable with no anticipated changes in chronic medications in the opinion of the Investigator
• Continues to meet protocol-specified criteria for qualification and contraception, including treatment-resistant seizure disorder
• In the opinion of the Investigator, the subject and/or parent(s)/caregiver(s) are able to continue keeping accurate seizure diaries

You may not qualify if:

• Inadequate supervision by parent(s)/caregiver(s)
• 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:
• the safety or well-being of the participant or study staff
• the safety or well-being of the participant's offspring (such as through pregnancy or breast-feeding)
• the analysis of results

Sponsors & Collaborators

• INSYS Therapeutics Inc: lead

Study Sites (11)

University of California San Francisco Medical Center

San Francisco, California, 94143, United States

Location

Miami Children's Hospital

Miami, Florida, 33155, United States

Location

Child Neurology Center - NW F

Pensacola, Florida, 32504, United States

Location

University of Chicago Medical Center

Chicago, Illinois, 60637, United States

Location

Clinical Research Center of Nevada LLC

Las Vegas, Nevada, 89104, United States

Location

Oregon Health Services University

Portland, Oregon, 97239, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Le Bonheur Children's Hospital

Memphis, Tennessee, 38103, United States

Location

Texas Scottish Rite Hospital for Children

Dallas, Texas, 79219, United States

Location

Granger Medical Clinic

Riverton, Utah, 84096, United States

Location

Mary Bridge Children's Hospital

Tacoma, Washington, 98403, United States

Location

MeSH Terms

Conditions

Seizures; Lennox Gastaut Syndrome; Epilepsies, Myoclonic

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Epileptic Syndromes; Epilepsy; Brain Diseases; Central Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Epilepsy, Generalized

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Results Point of Contact

• Title: Director, Clinical Development
• Organization: Insys Therapeutics, Inc.

gdecastro@insysrx.com

480-500-3105

Study Officials

• Neha Parikh

  INSYS Therapeutics Inc

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 12, 2014
• First Posted: December 17, 2014
• Study Start: January 8, 2016
• Primary Completion: June 22, 2017
• Study Completion: June 22, 2017
• Last Updated: July 26, 2018
• Results First Posted: July 26, 2018

Record last verified: 2018-06

Locations

US (11)