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Cannabidiol for Drug Resistant Pediatric Epilepsy (Expanded Access Use)

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ClinicalTrials.gov Identifier: NCT03676049
Expanded Access Status : Available
First Posted : September 18, 2018
Last Update Posted : November 16, 2020
Sponsor:
Information provided by (Responsible Party):
John, University of Mississippi Medical Center

Tracking Information
First Submitted Date September 11, 2018
First Posted Date September 18, 2018
Last Update Posted Date November 16, 2020
 
Descriptive Information
Brief Title Cannabidiol for Drug Resistant Pediatric Epilepsy (Expanded Access Use)
Brief Summary This is a treatment study under an approved Expanded Use IND protocol for using Cannabidiol (CBD) Extract. CBD will be used for the treatment of 5-10 children with drug resistant epilepsy. The CBD used in this study is prepared at the University of Mississippi under approval of the National Institute on Drug Abuse (NIDA) for its preparation and FDA approval under an expanded access mechanism on a compassionate use basis. The target patient population is who would otherwise have no appropriate remaining treatment modality left. These are patients for whom the risks of a relatively untested product are outweighed by the potential benefit. Using seizure-diaries to register seizure frequency, drug log and questionnaire to measure parent/patient quality of life and side effects will be assessed in each visit. Visits are: baseline, 4, 8, and 12 weeks visit. A 24 weeks visit (6 months) will be performed if the patient is stable on therapy during the 3 initial months and want to continue on the study for 3 more months. CBD will be administered as an adjunct to all current anti-epileptic therapies.
Detailed Description

Expanded Access Use: Restrictions to those with most significant epilepsy severity and especially neurocognitive delays (to minimize side effect profiles) remain in place. Study design, hover, is targeted at an Expanded Access use of product for children who would otherwise have no appropriate remaining treatment modality left.

Timeline: 3 months with the option to continue for up to 3 months of follow up therapy.

Procedures:

Participants: Five to ten (5-10) refractory epilepsy patients, aged 5-19 years old, all sexes, who must have failed at least 3 appropriate drugs, +/- vagal nerve stimulator, +/- dietary therapy, +/- resective surgery will be provided CBD as part of the Principal Investigator's Physician Expanded Access IND.

Written parental permission obtained from the patient's parent or legal representative must be obtained prior to beginning treatment.

Drug administration: CBD will be administered as an adjunct to all current antiepileptic drugs. A dosing titration period will start with 100 mg/day, with 50mg BID dosing, and will be titrated monthly as tolerated based on clinical response, up to 300mg/day. This is based on a targeted dosing of 2.5---5---7.5 mg/kg/d. Any patients under 40kg will have adjusted dosing based on weight as follows:

For 40 kg and up:

1 ml BID, escalating to 2 ml BID, and then 3 ml BID

For 30 - 39 kg:

0.75 ml BID, escalating to 1.5 and 2.25 ml BID

For 20 - 29 kg 0.5 ml BID, escalating to 1 and 1.5 ml BID

Clinical evaluations: All subjects will be clinically evaluated at baseline, once a month for the three months of titration period and during the three months of follow up period. During the first week of the study drug initiation, patients will be called at day 3 and then at day 7 to review any changes in condition. Subsequently, patients will receive a phone call weekly and will be evaluated in their physician study visits during the titration period to review any changes in condition and determine whether an increase in dosing is required. The investigator will be available by telephone throughout the 3 months of the titration period of the study, in between the visits and throughout the 3 months in the follow up period. Patients will receive the CBD during their monthly study visits during the titration and follow up period. The first dose of CBD will be administered by the physician in the Pediatric Clinical Research Clinic.

Pregnant patients will be excluded from the study. Female patients of childbearing potential will be screened for pregnancy prior to enrollment, and during the study if a potential for pregnancy arise. A -blood pregnancy test will be used. Therefore, parent/guardian of female subjects must tell the investigator and consult an obstetrician or maternal-fetal specialist if the child become pregnant during the study.

During the study, the patients will be constantly monitored as the investigators describe below:

Onset:

  • Pediatric Neurology Physician evaluation.
  • Initial lab work to include complete blood counts, comprehensive metabolic panel and baseline co-existent antiepileptic drug levels. Blood pregnancy testing will be performed on those patients with childbearing potential.
  • Initial body weight and routine vital signs
  • Initial baseline Electroencephalogram (EEG). Video EEG prior to enrollment if epileptic syndrome or severity is unclear. Decision and enrollment classification to be made by Dr. Ingram prior to enrollment.
  • Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview prior to enrollment.

    4 weeks/1 month:

  • Pediatric Neurology Physician evaluation.
  • Lab work to include complete blood counts, comprehensive metabolic panel and concomitant epilepsy medication levels. Blood testing for CBD/THC will also be performed. Additional lab draws can be performed with any symptoms to suggest anticonvulsant toxicity or concerning clinical signs/symptoms.
  • Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview at the end of 1-month study window.

    8 weeks/2 months:

  • Pediatric Neurology Physician evaluation.
  • Lab work as indicated for 4 weeks/1 month.
  • Body weight and routine vital signs
  • Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview at the end of 2-months study window.

    12 weeks/3 months:

  • Pediatric Neurology Physician evaluation.
  • EEG at the completion of the 3-month study window.
  • Lab work as indicated for 4 weeks/1 month.
  • Body weight and routine vital signs
  • Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview at the end of 3-months study window.

Follow up:

Patients who are stable on therapy and happy with the results may remain on study for up to 3 more months after titration period. These patients will have 6 months total on medication and one visit/month during the follow up time for clinical evaluation:

  • Complete blood count
  • Comprehensive metabolic profile (including liver function tests)
  • Measurement of levels of concomitant anti-epileptic drugs
  • Patient quality of life, side effect profile, and seizure count will be completed as typical clinical interview.
  • EEG (at 6 months visit)
Study Type Expanded Access
Expanded Access Type Intermediate-size Population
Intervention Drug: Cannabidiol
CBD will be administered as an adjunct to all current antiepileptic drugs. A dosing titration period will start with 100 mg/day, with 50mg BID dosing, and will be titrated monthly as tolerated based on clinical response, up to 300mg/day. This is based on a targeted dosing of 2.5 ---5---7.5 mg/kg/d.
Other Name: CBD
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Expanded Access Status Available
Contacts
Contact: John B Ingram, M.D. (601) 984-5210 ext 4-5210 jingram@umc.edu
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03676049
Responsible Party John, University of Mississippi Medical Center
Study Sponsor John
Collaborators Not Provided
Investigators Not Provided
PRS Account University of Mississippi Medical Center
Verification Date November 2020