Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms

A Phase 2 Study to Assess the Efficacy and Safety of Cannabidiol Oral Solution for the 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 (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.

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

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