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Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02551731
First Posted: September 16, 2015
Last Update Posted: October 5, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
INSYS Therapeutics Inc
  Purpose

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 (COS) 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 COS 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.


Condition Intervention Phase
Spasms, Infantile Drug: Cannabidiol Oral Solution Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Study to Assess the Efficacy and Safety of Cannabidiol Oral Solution for the Treatment of Refractory Infantile Spasms

Resource links provided by NLM:


Further study details as provided by INSYS Therapeutics Inc:

Primary Outcome Measures:
  • Part A: Percentage of participants who are considered complete responders at Day 14 [ Time Frame: at Day 14 ]
    Complete response is defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-EEG at Day 14.

  • Part B: Percentage of participants experiencing adverse events (AEs), treatment-emergent AEs (TEAEs), and Serious Adverse Events (SAEs) with severity ratings [ Time Frame: through week 64 ]

Secondary Outcome Measures:
  • Part A: Percentage of participants with absence of infantile spasms at Day 14 [ Time Frame: at Day 14 ]
  • Part A: Percentage of participants with absence of hypsarrhythmia at Day 14 [ Time Frame: at Day 14 ]
  • Part A: Median reduction in seizure-burden comparing video-EEG at Baseline to repeat video-EEG at Day 14 [ Time Frame: Baseline, Day 14 ]
  • Part A: Parent impression of efficacy and tolerability of study drug [ Time Frame: while on study (maximum 20 weeks/140 Days) ]
  • Part A: Percentage of participants with a partial response to treatment [ Time Frame: while on study (maximum 20 weeks/140 Days) ]
  • Part A: Percentage of complete responders with relapse, based on clinical judgement by Investigator [ Time Frame: while on study (maximum 20 weeks/140 Days) ]
  • Part A: Time to complete responder relapse [ Time Frame: while on study (maximum 20 weeks/140 Days) ]
  • Part B: Parent impression of efficacy and tolerability of study drug as measured by the change in Clinical Global Impression of Improvement Assessment (CGI-I), responses at every visit throughout Part B [ Time Frame: throughout Part B (up to Week 64) ]
  • Part B: Investigator impression of efficacy and tolerability of study drug as measured by the change in CGI-I responses at every visit throughout Part B [ Time Frame: throughout Part B (up to Week 64) ]
  • Part B: Median reduction in seizure-burden comparing Seizure Diaries throughout Part B. [ Time Frame: throughout Part B (up to Week 64) ]
  • Part B: Percentage of subjects who have a relapse of spasms based on video-EEG [ Time Frame: throughout Part B (up to Week 64) ]
  • Part B: Time to relapse as confirmed by video-EEG [ Time Frame: throughout Part B (up to Week 64) ]

Enrollment: 9
Actual Study Start Date: January 2016
Estimated Study Completion Date: October 2017
Estimated Primary Completion Date: October 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Full Analysis Set - All Participants
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
20-40 mg/kg/day BID

Detailed Description:

A protocol amendment in May 2016 created two parts to this trial: Part A (the extended treatment period) and Part B (the safety treatment period), whose objectives are as follows:

Primary Part A: To evaluate the efficacy of Cannabidiol Oral Solution in treating refractory infantile spasms (IS).

Secondary:

Part A:

  • To evaluate the safety of Cannabidiol Oral Solution in treating refractory infantile spasms.

Part B:

  • To assess the long-term safety of Cannabidiol Oral Solution as an adjunctive treatment for subjects with Infantile Spasms (IS)
  • To establish the continued efficacy of Cannabidiol Oral Solution in maintaining seizure control in subjects with IS
  • To assess the global status of subjects taking Cannabidiol Oral Solution for an extended period of time determined by various qualitative assessments
  • To monitor for changes in plasma levels of Cannabidiol Oral Solution during long-term treatment of subjects with IS
  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   6 Months to 36 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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:

    1. the safety or well-being of the participant or study staff
    2. the analysis of results
  • During the Safety Treatment and Follow-up Periods, subjects are not to receive the following:

    1. any cannabinoids (CBD, Δ9-tetrahydrocannabinol (THC), hemp oil, Realm Oil or marijuana)
    2. any other investigational drug or investigational device
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02551731


Locations
United States, California
Mattel Children's Hospital at UCLA
Los Angeles, California, United States, 90095
University of California - San Francisco
San Francisco, California, United States, 94143
United States, Florida
Miami Children's Hospital
Miami, Florida, United States, 33155
United States, Michigan
Beaumont Health System
Royal Oak, Michigan, United States, 48073
Sponsors and Collaborators
INSYS Therapeutics Inc
Investigators
Study Director: Neha Parikh INSYS Therapeutics Inc
  More Information

Responsible Party: INSYS Therapeutics Inc
ClinicalTrials.gov Identifier: NCT02551731     History of Changes
Other Study ID Numbers: INS011-15-054
First Submitted: September 14, 2015
First Posted: September 16, 2015
Last Update Posted: October 5, 2017
Last Verified: October 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Spasm
Spasms, Infantile
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Epilepsy, Generalized
Epilepsy
Brain Diseases
Central Nervous System Diseases
Pharmaceutical Solutions