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Safety, and Tolerability of Epidiolex In Patients ((Ages 1 - 19 Years) With Intractable Epilepsy

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ClinicalTrials.gov Identifier: NCT02695537
Recruitment Status : Recruiting
First Posted : March 1, 2016
Last Update Posted : October 9, 2017
Sponsor:
Information provided by (Responsible Party):
Martina Bebin, University of Alabama at Birmingham

Brief Summary:
The purpose of this study is to evaluate the safety and tolerability of Epidiolex at various doses between 5 mg/kg/day and 50 mg/kg/day as an additional (add-on) drug for treating debilitating, drug-resistant epilepsy.

Condition or disease Intervention/treatment Phase
Epilepsy Seizures Drug: Epidiolex Phase 1

Detailed Description:

The specific goals of this phase I dose finding study conducted in consecutively enrolled patients 1-18 years of age are to prospectively and longitudinally assess the safety and tolerability, including cognitive effects, of CBD at various doses between 5 mg/kg/day and 25 mg/kg/day with additional titration in some subjects up to 50 mg/kg/day. In order to participate in the study, participants will need to fulfill the inclusion and exclusion criteria.

The goal of the study is to fulfill the mandate of "Carly's Law" and to provide patients with debilitating epileptic conditions with access to CBD as an add-on treatment. Other care including routine neurological care that is not related to participation in the CBD study will need to be provided by patients' primary/current treating neurologist.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Tolerability of Epidiolex In Patients ((Ages 1 - 19 Years) With Intractable Epilepsy
Study Start Date : April 2015
Estimated Primary Completion Date : February 2020
Estimated Study Completion Date : February 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Epilepsy Seizures

Arm Intervention/treatment
Experimental: 5 mg/kg/day
In some patients additional titration by 5 mg/kg/day every 2 weeks up to 50 mg/kg/day may be instituted at the discretion of the PI upon discussion with the Co-PI
Drug: Epidiolex
In some patients additional titration by 5 mg/kg/day every 2 weeks up to 50 mg/kg/day may be instituted at the discretion of the PI upon discussion with the Co-PI




Primary Outcome Measures :
  1. Evaluate severe adverse events (increase in seizure frequency by more than 25% or any hospitalization related to increase in seizure frequency) [ Time Frame: 1 year ]
  2. Evaluate change in resting blood pressure or heart rate by 25% [ Time Frame: 1 year ]
  3. Evaluate any change in, CBC, CMP, Liver function tests (LFTSs ), Urinary Analysis or Antiepileptic drug (AED) levels considered by managing neurologists as clinically significant. [ Time Frame: 1 year ]
    Clinically significant will be determined by using the Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03. Adverse events categorized as a grade 3 or above will be considered clinically significant. Adverse Events graded 4 or above will be considered severe adverse events (SAE)


Secondary Outcome Measures :
  1. Assess the frequency in decrease in seizure frequency as measured in total number of seizures per month. [ Time Frame: 30 days ]
  2. Assess the frequency in decrease in seizure severity as measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP). [ Time Frame: 30 days ]


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Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients between 1 years (12 months)-18 years with drug resistant epilepsy confirmed by video EEG recording report
  • Patient should have history of a trial of at leas four drugs, including one trial of a combination of two concomitant drugs without successful seizure control. Vagal nerve stimulation, RNS deep brain stimulation, or the ketogenic diet can be considered equivalent to a drug trial. Patient suffering from an epileptic syndrome that is known to be refractory to treatment, such as Dravet or Lernnox Gastaut Syndrome, may be included after a trial of only two drugs
  • Between 1-4 baseline anti-epileptic drugs at stable doses for a minimum of 4 weeks prior to submitting records for review by the CBD Treatment Approval Committee
  • VNS or RBS must be on stable settings for a minimum of 3 months
  • If on ketogenic diet, must be on stable ration for a minimum of 3 months
  • Review of the following patient medical information:
  • Most recent ECG report,
  • Video/EEG monitoring report confirming the diagnosis of epilepsy,
  • Evidence that the patient has failed 4 AEDs as indicated above,
  • Patient must have at least 4 clinically countable seizures per month,
  • Seizure history to include a documented history of generalized (drop, atonic, tonic clonic, and/or myoclonic) seizures, focal seizures without loss of consciousness with a motor component, focal seizures with loss of consciousness, or focal seizures with secondary generalization, complex partial seizures with a motor or tonic component, and I or altered awareness seizures,
  • Results of routine testing including blood work (CBC, CMP, LFTs renal panel, Urinary Analysis, and levels of all AEDs) and digital copy of a routine EEG along with the formal written report performed within 3 months prior to submitting records for CBD Treatment Approval review. If applicable, results of any metabolic or genetic testing performed should be included in submitted records for review! If any AED dose was adjusted in the preceding 3 months, level on the new dose will need to be provided.
  • If applicable, documentation (including date of surgery) of prior VNS, RNS, Corpus Callostomy, or other epilepsy surgery the patient has received.
  • Acceptable method of contraception (or abstinence) for women of childbearing potential and for male patients with partners of childbearing potential, and female patients must have_a negative urine pregnancy test on the day ofinitating CBD.
  • For patients who agree to participate in the optional neuroimaging sub study, an MRI screen will be obtained to show that the patient does not have contraindication to receiving MRI/fMRI at 3 Tesla (e.g., metallic artifact).
  • Patients are able to supply investigator with seizure calendars for the past 3 months prior to submitting records for CBD Treatment Approval Committee review. The patient will need to provide an updated calendar at the time of enrollment.
  • Approval for inclusion by the CBD Treatment Approval Committee.
  • Current State of Alabama Resident
  • Acceptable documentation of Alabama residency includes the following:

    • a state issued ID, such as a driver's license, from patient or patient's parent/ legally authorized representative (LAR).
    • documents showing the patient or patient's parent/LAR rents/owns property in the state,
    • state voter registration from patient or patient's parent/LAR, or
    • a recent state tax return from patient or patient's parent/LAR.

Exclusion Criteria:

  • Active Psychogenic non-epileptic seizures (PNES); Patients with more than 1 year freedom from PNES will not be excluded,
  • Patients who are pregnant, breastfeeding, or not using acceptable methods of contraception during the course of the study and for three months thereafter,
  • Male patient's partner is of child bearing potential; unless willing to ensure that they (male patients) or their partner(s) are using acceptable methods of contraception during the course of the study and for three months thereafter
  • History of substance abuse/addiction,
  • Use of medical marijuana or CBD based product in the past 30 days,
  • Initiation offelbamate within last 12 months,
  • Allergy to CBD or any marijuana-type products,
  • ALT >5 x ULN or AST >5 x ULN, as seen in participant's laboratory results submitted to the CBD Treatment Approval Committee for review.
  • Hemoglobin <1 0 or Hematocrit <30 or WBC < 2000, as seen in participant's laboratory results submitted to the CBD Treatment Approval Committee for review.
  • In Investigator's judgement, active medical condition/treatment that impacts study activities.
  • Unable to provide consent (and no LAR),
  • Unable/Failure to comply with study visits/requirements and/or instructions.
  • Confirmed diagnosis for Dravet Syndrome or Lennox Gastaut Syndrome that qualifies the patient for a GW Dravet Syndrome or LelUlox Gastaut Syndrome randomized controlled clinical trial for which the patient is eligible pursuant to the GW clinical trial enrollment criteria unless
  • (a) there is no study that is either actively open for enrollment of patients at UAB or that is expected to actively begin enrolling patients at The University of Alabama at Birmingham within two (2) months of the date on which the patient is screened for the UAB Pediatric CBD Program or UAB Adult CBD Program.Primary residence in a State different than Alabama.
  • Subjects with contraindications to MRI!fMRI at 3 Tesla (e.g., metallic artifact) will not be offered participation in the optional substudy.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02695537


Contacts
Contact: Martina Bebin, MD 205-934-3866 ebebin@uab.edu

Locations
United States, Alabama
University of Alabama at Birmingham Recruiting
Birmingham, Alabama, United States, 35233
Contact: Martina Bebin, MD         
Sponsors and Collaborators
University of Alabama at Birmingham
Investigators
Principal Investigator: Martina Bebin, MD Neurology Chair Office - University of Alabama at Birmingham

Responsible Party: Martina Bebin, Professor, University of Alabama at Birmingham
ClinicalTrials.gov Identifier: NCT02695537     History of Changes
Other Study ID Numbers: IND# 124160
First Posted: March 1, 2016    Key Record Dates
Last Update Posted: October 9, 2017
Last Verified: October 2017

Additional relevant MeSH terms:
Epilepsy
Seizures
Drug Resistant Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurologic Manifestations
Signs and Symptoms