Phase 1
Completed N=80
University of Alabama at Birmingham (UAB) Adult CBD Program
Epilepsy · Seizures
Source: ClinicalTrials.gov NCT02700412 ↗
Enrolled (actual)
80
Serious AEs
17.5%
Results posted
May 2020
Primary outcomePrimary: Number of Participants With Severe Adverse Events (SAEs) (Increase in Seizure Frequency by More Than 100% Leading to Emergency Room Visit or Hospitalization). — 0 Participants
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Severe Adverse Events (SAEs) (Increase in Seizure Frequency by More Than 100% Leading to Emergency Room Visit or Hospitalization). |
— | — |
| PRIMARY Number of Participants With Change in Resting Blood Pressure or Heart Rate by 25% if Considered Significant by Managing Neurologist. |
0; 0 | — |
| PRIMARY Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant. |
0; 0; 0; 0; 2; 0 | — |
| SECONDARY Change in Seizure Frequency as Measured in Total Number of Seizures Per Month. |
0.73; 0.56; 0.47; 0.44; 0.44; 0.47 | <0.0001 sig |
| SECONDARY Change in Seizure Severity Measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP). |
0.53; 0.32; 0.23; 0.22; 0.24; 0.28 | <0.0001 sig |
Eligibility Criteria
Inclusion Criteria
- Any patient with disabling epilepsy with diagnosis confirmed by video/EEG monitoring, and
- Patient should have history of a trial of at least four anti-epileptic drugs (AEDs) including one trial of a combination of two concomitant AEDs, without successful seizure control. Vagus nerve stimulation (VNS), Responsive Neurostimulation (RNS) deep brain stimulation, or the ketogenic diet can be considered an equivalent to a drug trial,
- 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 RNS must be on stable settings for a minimum of 3 months,
- If on ketogenic diet, must be on stable ratio for a minimum of 3 months.
- The referring provider needs to make available for review all of the following:
- Most recent Brain MRI report,
- Most recent ECG report,
- Video/EEG monitoring report confirming the diagnosis of epilepsy,
- Evidence that the patient has failed 4 AEDs as indicated above,
- Current Medication List
- Patient must have at least 4 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,
- Results of routine testing including blood work (Complete Blood Count (CBC), Comprehensive Metabolic Panel (CMP), Liver Function Tests (LFTs), renal panel, Urinary Analysis (UA), 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 any AED dose was adjusted within 3 months prior to submitting records for CBD Treatment Approval Committee review, level on the new dose will need to be provided. If applicable, results of any metabolic or genetic testing performed should be included in submitted records for review:
- If applicable, documentation (including date of surgery) of prior VNS, RNS, Corpus Callosotomy, or other epilepsy surgery the patient has received.
- Age 15 years and older,
- Patients are able to keep and provide seizure calendar for at least 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,
- Acceptable method of contraception (or abstinence) for women of childbearing potential and for male patients with partners of childbearing potential; female patients must have a negative urine pregnancy test on the day of initiating 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/function MRI (fMRI) at 3 Tesla (e.g., metallic artifact).
- 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 identification (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
Data sourced from ClinicalTrials.gov (NCT02700412). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.