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

September 5, 2018 updated by: PhytoTech Therapeutics, Ltd.

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
16

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Israel
      • Tel Aviv, Israel
        • Pediatric Epilepsy department- Souraskey Medical center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

2 years to 15 years (CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
EXPERIMENTAL: PLT101
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
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

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Incidence of study treatment related adverse events (AEs)
Time Frame: 12 weeks of treatment + 2 weeks follow up
12 weeks of treatment + 2 weeks follow up
Percent change in mean countable monthly seizure frequency
Time Frame: 12 week treatment period
12 week treatment period

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Incidence of all adverse events (AEs)
Time Frame: 12 weeks of treatment + 2 weeks follow up
12 weeks of treatment + 2 weeks follow up
assessment of Caregiver Global Impression of Improvement using a 5-point rating scale
Time Frame: after 5 weeks of maintenance dose and at end of treatment
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
Time Frame: after 5 weeks of maintenance dose and at end of treatment
after 5 weeks of maintenance dose and at end of treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
PhytoTech Therapeutics, Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 13, 2017

Primary Completion (ACTUAL)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 28, 2018

Study Completion (ACTUAL)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 28, 2018

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 6, 2016

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 7, 2016

First Posted (ESTIMATE)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 8, 2016

Study Record Updates

Last Update Posted (ACTUAL)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 6, 2018

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 5, 2018

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2017

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CS-100

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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