A Study to Evaluate the Safety, Tolerability and Efficacy of Oral Administration of PTL101 (Cannabidiol) as an Adjunctive Treatment for Pediatric Intractable Epilepsy

NCT02987114 | Completed Phase 2

• 1 other identifier: CS-100
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

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.

Conditions

Epilepsy Intractable

Outcome Measures

Primary Outcomes (2)

• Incidence of study treatment related adverse events (AEs)

  12 weeks of treatment + 2 weeks follow up

• Percent change in mean countable monthly seizure frequency

  12 week treatment period

Secondary Outcomes (3)

• Incidence of all adverse events (AEs)

  12 weeks of treatment + 2 weeks follow up

• assessment of Caregiver Global Impression of Improvement using a 5-point rating scale

  after 5 weeks of maintenance dose and at end of treatment

• assessment of Caregiver Global Impression of Seizure Severity using a 5-point rating scale

  after 5 weeks of maintenance dose and at end of treatment

Study Arms (1)

PLT101

EXPERIMENTAL

PTL101 capsules (50 or 100 mg CBD per capsule) up to 25 mg/kg/day or up to 450 mg/day, the lower of the two. Twice daily (morning and evening).

Drug: PLT101

Interventions

PLT101 DRUG

PTL 101 (Cannabidiol, CBD) Two piece hard capsules filled with seamless gelatin matrix green beads containing CBD (50 or 100 mg CBD per capsule) The beads should be administered following a meal, mixed with food

PLT101

Eligibility Criteria

• Age: 2 Years - 15 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Pediatric subjects with refractory epilepsy
• years old (inclusive), male or female
• 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).
• At least four clinically countable seizures within 4 weeks of study entry \[tonic, clonic, tonic-clonic in the form of partial seizures, partial seizures secondarily generalized or primary generalized and/or complex partial seizures and drop attacks (tonic/atonic)\]
• Subject on a stable regimen of 1-4 concomitant antiepileptic drugs (AEDs) for a minimum of 4 weeks prior to enrollment
• History of treatment with at least four AEDs, including one trial of a combination of two concomitant drugs, without successful seizure control
• Subjects with vagal nerve stimulation system must be on stable settings for a minimum of 6 months prior to enrollment
• 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
• The subject's legal guardian voluntarily provides consent for participation in the study and signs an Informed Consent Form
• 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.

You may not qualify if:

• The subject is currently using or has used cannabis-based or synthetic cannabinoid within three months of study entry
• Subject is unwilling to abstain from use of cannabis-based or synthetic cannabinoid throughout the study period
• Neurodegenerative or deteriorated neurological disease
• History of heart failure
• Known family history (first-degree) of psychiatric disorders
• Psychosis or past psychotic event and/or anxiety disorder
• Current or history of drug abuse/addiction
• Renal, hepatic (ALT/AST \>2x upper limit of normal (ULN), bilirubin \>2x ULN), pancreatic dysfunctions or laboratory test abnormalities, at the investigator's discretion
• Clinically significant finding in baseline ECG
• Initiation of felbamate treatment within 9 months of screening
• Allergy to CBD or any cannabinoid and/or formulation excipients
• Subject is pregnant, lactating, or planning a pregnancy during the course of the study or within 3 months of study completion
• Subject and legal guardian/caregiver unable to comply with study visits/requirements
• 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)

Sponsors & Collaborators

• PhytoTech Therapeutics, Ltd.: lead

Study Sites (1)

Pediatric Epilepsy department- Souraskey Medical center

Tel Aviv, Israel

Location

MeSH Terms

Conditions

Drug Resistant Epilepsy

Condition Hierarchy (Ancestors)

Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 6, 2016
• First Posted: December 8, 2016
• Study Start: February 13, 2017
• Primary Completion: June 28, 2018
• Study Completion: June 28, 2018
• Last Updated: September 6, 2018

Record last verified: 2017-09

Data Sharing

• IPD Sharing: Will not share

Locations

IL (1)