Clinical trial NCT02987114: A Study to Evaluate the Safety, Tolerability and Efficacy of Oral Administration of PTL101 (Cannabidiol) as an Adjunctive Treatment for Pediatric Intractable Epilepsy
Official title: A Phase II, Open-label, Single-center Clinical Study to Evaluate the Safety, Tolerability and Efficacy of Oral Administration of PTL101 (Cannabidiol) as an Adjunctive Treatment for Pediatric Intractable Epilepsy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Epilepsy Intractable
Lead sponsor: PhytoTech Therapeutics, Ltd.
Sponsor class: INDUSTRY
First posted: 2016-12-08
Enrollment: 16
Sex: ALL
Minimum age (years): 2
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To evaluate the safety, tolerability and efficacy of oral administration of PTL101 (cannabidiol) for the treatment for pediatric intractable epilepsy.

Study will include a 4-week observation period ,12 weeks of treatment and 2 weeks of follow up.

Detailed description: This is an open-label, single-center study recruiting approximately 15 male or female pediatric (ages 2-15, inclusive) patients with, intractable epilepsy, on stable doses of antiepileptic drugs (AEDs). The study comprised of the following period: 4 weeks observation period, followed by a 2-week dose titration period, 10-week maintenance treatment period, and a 2-week follow-up of which 1 week is a tapering-off period. Seizures will be recorded by the legal guardian/caregiver in seizure diaries throughout the first 16 weeks of the study.

Eligibility criteria: Inclusion Criteria:

1. Pediatric subjects with refractory epilepsy
2. 2-15 years old (inclusive), male or female
3. History of the any of the following seizure types: tonic, clonic, tonic-clonic in the form of partial seizures, partial seizures secondarily generalized or primary generalized, complex partial seizures and drop attacks (tonic/atonic).
4. At least four clinically countable seizures within 4 weeks of study entry \[tonic, clonic, tonic-clonic in the form of partial seizures, partial seizures secondarily generalized or primary generalized and/or complex partial seizures and drop attacks (tonic/atonic)\]
5. Subject on a stable regimen of 1-4 concomitant antiepileptic drugs (AEDs) for a minimum of 4 weeks prior to enrollment
6. History of treatment with at least four AEDs, including one trial of a combination of two concomitant drugs, without successful seizure control
7. Subjects with vagal nerve stimulation system must be on stable settings for a minimum of 6 months prior to enrollment
8. For subjects undergoing dietary treatment (e.g., ketogenic or modified Atkins diet):

   the fat to carbohydrate ratio must be stable for a minimum of eight weeks prior to enrollment
9. The subject's legal guardian voluntarily provides consent for participation in the study and signs an Informed Consent Form
10. Completed seizures diary for four weeks (±3 days) prior to initiation of the dose titration period (visit 2). Subject will be considered a screen failure if seizures diary was not appropriately completed.

Exclusion Criteria:

1. The subject is currently using or has used cannabis-based or synthetic cannabinoid within three months of study entry
2. Subject is unwilling to abstain from use of cannabis-based or synthetic cannabinoid throughout the study period
3. Neurodegenerative or deteriorated neurological disease
4. History of heart failure
5. Known family history (first-degree) of psychiatric disorders
6. Psychosis or past psychotic event and/or anxiety disorder
7. Current or history of drug abuse/addiction
8. Renal, hepatic (ALT/AST \>2x upper limit of normal (ULN), bilirubin \>2x ULN), pancreatic dysfunctions or laboratory test abnormalities, at the investigator's discretion
9. Clinically significant finding in baseline ECG
10. Initiation of felbamate treatment within 9 months of screening
11. Allergy to CBD or any cannabinoid and/or formulation excipients
12. Subject is pregnant, lactating, or planning a pregnancy during the course of the study or within 3 months of study completion
13. Subject and legal guardian/caregiver unable to comply with study visits/requirements
14. Subject is currently enrolled in, or has not yet completed a period of at least 60 days since ending another investigational device or drug trial(s)

Primary outcomes:
measure: Incidence of study treatment related adverse events (AEs)
timeFrame: 12 weeks of treatment + 2 weeks follow up
measure: Percent change in mean countable monthly seizure frequency
timeFrame: 12 week treatment period
Secondary outcomes:
measure: Incidence of all adverse events (AEs)
timeFrame: 12 weeks of treatment + 2 weeks follow up
measure: assessment of Caregiver Global Impression of Improvement using a 5-point rating scale
timeFrame: after 5 weeks of maintenance dose and at end of treatment
measure: assessment of Caregiver Global Impression of Seizure Severity using a 5-point rating scale
timeFrame: after 5 weeks of maintenance dose and at end of treatment
Other outcomes:
Locations:
city: Tel Aviv
country: Israel
facility: Pediatric Epilepsy department- Souraskey Medical center
Study officials:
References: