NCT02461706

Brief Summary

The main goal of this study is to provide treatment with cannabidiol (CBD) to children with drug resistant epilepsy through a Physician Expanded Access Investigation New Drug protocol. As the controlled therapeutic use of CBD in children is novel, the primary objective of this study is to evaluate the safety and efficacy of CBD when administered as adjunctive therapy in children that have exhausted the majority of FDA approved antiepileptic drug treatment options.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
1 country

2 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 1, 2015

Completed
2 days until next milestone

First Posted

Study publicly available on registry

June 3, 2015

Completed
Last Updated

January 22, 2019

Status Verified

January 1, 2019

First QC Date

June 1, 2015

Last Update Submit

January 18, 2019

Conditions

Epilepsy

Keywords

Drug resistant epilepsy

Interventions

cannabidiolDRUG

All subjects will have a dosing titration starting with 25 mg/kg/day and will be titrated weekly as tolerated based on clinical response. All subjects will be clinically evaluated at baseline, once a month for three months and every three months thereafter. In order to ensure safe use at higher doses, patients receiving more than 600 mg of daily CBD will be evaluated at least monthly until they achieve steady state at the final fixed maintenance dose.

Also known as: CBD
Blood TestOTHER

All subjects will have the following clinical laboratory tests including changes in bone marrow (CBC), liver (AST, ALT), and kidney function (creatinine, electrolytes, BUN) will be assessed at baseline, each month during months 1 to 3, and every 3 months thereafter.

Eligibility Criteria

Age2 Years - 16 Years
Sexall
Age GroupsChild (0-17)

You may qualify if:

  • history of a trial of at least four drugs, including one trial of a combination of two concomitant drugs, without successful seizure control.
  • Vagal nerve stimulation: VNS must be on stable settings for a minimum of 1 month,
  • RNS deep brain stimulation, or
  • the ketogenic diet can be considered equivalent to a drug trial and must be on a stable ratio for a minimum of 3 months.
  • anti-epileptic drugs at stable doses for a minimum of 4 weeks prior to enrollment.
  • clinically countable seizures per month: Seizure history to include a documented history of generalized seizures (drop attacks, atonic, tonic-clonic and/or myoclonic), focal seizures without loss of consciousness with a motor component, focal seizures with loss of consciousness, or focal seizures with secondary generalization,

You may not qualify if:

  • Use of CBD based product within the previous 30 days.
  • Initiation of felbamate within 6 months.
  • Allergy to CBD or any cannabinoid.
  • Unable to comply with study visits/requirements.
  • Diagnosis of Dravet Syndrome or Lennox-Gastaut syndrome will be excluded completely from this trial.
  • Cannabidiol is contraindicated in pregnancy. Female subjects who are pregnant will be excluded from the study. If a female subject is able to become pregnant, she will be given a serum pregnancy test before entry into the study. Female subjects will be informed not become pregnant while taking cannabidiol. Female subjects must tell the investigator and consult an obstetrician or maternal-fetal specialist if they become pregnant during the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

University of Flordia CRC

Gainesville, Florida, 32610, United States

Location

Nicklaus Children's Hospital, Miami Children's Health System

Miami, Florida, 33155, United States

Location

MeSH Terms

Conditions

EpilepsyDrug Resistant Epilepsy

Interventions

CannabidiolHematologic Tests

Condition Hierarchy (Ancestors)

Brain DiseasesCentral Nervous System DiseasesNervous System Diseases

Intervention Hierarchy (Ancestors)

CannabinoidsTerpenesHydrocarbonsOrganic ChemicalsClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisInvestigative Techniques

Study Officials

  • Steven Winesett, MD

    University of Florida

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
expanded access
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 1, 2015

First Posted

June 3, 2015

Last Updated

January 22, 2019

Record last verified: 2019-01

Locations

US(2)