Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms
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.
|Study Design:||Intervention Model: Single Group Assignment
Masking: No masking
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|
- 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 ]
- 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) ]
|Actual Study Start Date:||January 2016|
|Estimated Study Completion Date:||July 2017|
|Estimated Primary Completion Date:||July 2017 (Final data collection date for primary outcome measure)|
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
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).
- To evaluate the safety of Cannabidiol Oral Solution in treating refractory infantile spasms.
- 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT02551731
|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|
|Study Director:||Neha Parikh||INSYS Therapeutics Inc|