Clinical trial NCT02551731: Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms
Official title: A Phase 2 Study to Assess the Efficacy and Safety of Cannabidiol Oral Solution for the Treatment of Refractory Infantile Spasms
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Spasms, Infantile
Lead sponsor: INSYS Therapeutics Inc
Sponsor class: INDUSTRY
First posted: 2015-09-16
Enrollment: 9
Sex: ALL
Minimum age (years): 6
Maximum age (years): 36
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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 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

Primary outcomes:
measure: Part A: Percentage of Participants Who Are Considered Complete Responders at Day 14
timeFrame: Day 14
measure: Part B: Percentage of Participants Experiencing Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), and Serious Adverse Events (SAEs)
timeFrame: Up to Week 64
Secondary outcomes:
measure: Part A: Percentage of Participants With Absence of Infantile Spasms at Day 14
timeFrame: Day 14
measure: Part A: Percentage of Participants With Absence of Hypsarrhythmia at Day 14
timeFrame: Day 14
measure: Part A: Median Reduction in Seizure-burden Comparing Video-EEG at Baseline to Repeat Video-EEG at Day 14
timeFrame: Baseline, Day 14
measure: Part A: Parent Impression of Efficacy and Tolerability of Study Drug
timeFrame: Visit 3 (Day 14), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 10), and end of study.
measure: Part A: Percentage of Participants With a Partial Response to Treatment
timeFrame: Day 14
measure: Part A: Percentage of Complete Responders With Relapse
timeFrame: Day 14
measure: Part A: Time to Complete Responder Relapse
timeFrame: Day 14
measure: 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
timeFrame: Up to Week 64
measure: 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
timeFrame: Up to Week 64
measure: Part B: Median Reduction in Seizure-burden Comparing Seizure Diaries Throughout Part B.
timeFrame: Up to Week 64
measure: Part B: Percentage of Participants Who Have a Relapse of Spasms Based on Video-EEG
timeFrame: Up to Week 64
measure: Part B: Time to Relapse as Confirmed by Video-EEG
timeFrame: Up to Week 64
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Mattel Children's Hospital at UCLA
state: California
city: San Francisco
country: United States
facility: University of California - San Francisco
state: California
city: Miami
country: United States
facility: Miami Children's Hospital
state: Florida
city: Royal Oak
country: United States
facility: Beaumont Health System
state: Michigan
Study officials:
affiliation: INSYS Therapeutics Inc
name: Neha Parikh
role: STUDY_DIRECTOR
References: