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

Epilepsy Intractable Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02987114
• Other study ID #: CS-100
• Status: Completed
• Phase: Phase 2
• Start date: February 13, 2017
• Est. completion date: June 28, 2018

Study information

• Verified date: September 2017
• Source: PhytoTech Therapeutics, Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. completion date: June 28, 2018
• Est. primary completion date: June 28, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 15 Years

Eligibility

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)

Related Conditions & MeSH terms

• Drug Resistant Epilepsy
• Epilepsy
• Epilepsy Intractable

Intervention

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

Locations

Country	Name	City	State
Israel	Pediatric Epilepsy department- Souraskey Medical center	Tel Aviv

Sponsors (1)

Lead Sponsor	Collaborator
PhytoTech Therapeutics, Ltd.

Country where clinical trial is conducted

Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of study treatment related adverse events (AEs)		12 weeks of treatment + 2 weeks follow up
Primary	Percent change in mean countable monthly seizure frequency		12 week treatment period
Secondary	Incidence of all adverse events (AEs)		12 weeks of treatment + 2 weeks follow up
Secondary	assessment of Caregiver Global Impression of Improvement using a 5-point rating scale		after 5 weeks of maintenance dose and at end of treatment
Secondary	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