Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms

NCT02551731 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: INS011-15-054
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 4

Brief Summary

Infantile Spasms (IS) is a diagnosis described as a fairly rare and terrible form of epilepsy that usually strikes children in the first year of life. There is a great need for safe and effective therapies in the treatment of IS. This need is even more important for infants and toddlers still sick after being treated with medicine that is already available. This is a multi-center study to evaluate the efficacy and safety of Cannabidiol Oral Solution (CBD) in the treatment of children aged 6 months through 36 months with a diagnosis of infantile spasms who have not responded to first line therapies. The overall study duration is expected to be 64 weeks for those subjects who respond to CBD treatment. The maximum possible study duration for each patient is approximately 64 weeks, however a subject will be deemed to have completed the study after 58 weeks.

Conditions

Spasms, Infantile

Outcome Measures

Primary Outcomes (2)

• Part A: Percentage of Participants Who Are Considered Complete Responders at Day 14

  Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-electroencephalogram (EEG) at Day 14.

  Day 14

• Part B: Percentage of Participants Experiencing Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), and Serious Adverse Events (SAEs)

  Up to Week 64

Secondary Outcomes (12)

• Part A: Percentage of Participants With Absence of Infantile Spasms at Day 14

  Day 14

• Part A: Percentage of Participants With Absence of Hypsarrhythmia at Day 14

  Day 14

• Part A: Median Reduction in Seizure-burden Comparing Video-EEG at Baseline to Repeat Video-EEG at Day 14

  Baseline, Day 14

• Part A: Parent Impression of Efficacy and Tolerability of Study Drug

  Visit 3 (Day 14), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 10), and end of study.

• Part A: Percentage of Participants With a Partial Response to Treatment

  Day 14

Study Arms (1)

Cannabidiol Oral Solution: 20 or 40 mg/kg/day BID

EXPERIMENTAL

The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day \[10 mg/kg twice per day (BID)\], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.

Drug: Cannabidiol Oral Solution

Interventions

Cannabidiol Oral Solution DRUG

20 or 40 mg/kg/day BID

Cannabidiol Oral Solution: 20 or 40 mg/kg/day BID

Eligibility Criteria

• Age: 6 Months - 36 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Meets protocol-specified criteria for qualification, including infantile spasms
• Parent(s)/caregiver(s) fully comprehend and sign the informed consent form, understand all study procedures, and can communicate satisfactorily with the Investigator and study coordinator.

You may not qualify if:

• History or current use of over-the-counter medications, dietary supplements, or drugs 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 analysis of results
• During the Safety Treatment and Follow-up Periods, subjects are not to receive the following:
• any cannabinoids (CBD, Δ9-tetrahydrocannabinol (THC), hemp oil, Realm Oil or marijuana)
• any other investigational drug or investigational device

Sponsors & Collaborators

• INSYS Therapeutics Inc: lead

Study Sites (4)

Mattel Children's Hospital at UCLA

Los Angeles, California, 90095, United States

Location

University of California - San Francisco

San Francisco, California, 94143, United States

Location

Miami Children's Hospital

Miami, Florida, 33155, United States

Location

Beaumont Health System

Royal Oak, Michigan, 48073, United States

Location

MeSH Terms

Conditions

Spasms, Infantile

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

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

Study Record Dates

• First Submitted: September 14, 2015
• First Posted: September 16, 2015
• Study Start: January 27, 2016
• Primary Completion: September 6, 2016
• Study Completion: September 6, 2016
• Last Updated: September 19, 2018
• Results First Posted: September 19, 2018

Record last verified: 2018-08

Locations

US (4)