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Summary

for people ages 3–17 (full criteria)
at San Francisco, California and other locations
study started
estimated completion:

Description

Summary

The primary purpose of this study is to assess the efficacy of Cannabidiol Oral Solution in the treatment of pediatric participants with treatment-resistant childhood absence seizures. This study will also assess safety, tolerability and pharmacokinetics of Cannabidiol Oral Solution, and any improvement in qualitative assessments of participant status over the duration of the study in pediatric participants with treatment-resistant childhood absence seizures. The study will include a 4-week Screening Period, a 5 or 10 day Titration Period (depending study Cohort), a 4-week Treatment Period followed by 5-day Tapering for doses >20 mg/kg/day and a 4-week Follow-up Period.

Official Title

A Phase 2, Open-label, Dose-finding Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Pediatric Patients With Treatment-Resistant Childhood Absence Seizures

Keywords

Childhood Absence Epilepsy Seizures Epilepsy, Absence Pharmaceutical Solutions

Eligibility

You can join if…

Open to people ages 3–17

  1. Patient and/or parent(s)/caregiver(s) fully comprehend the informed consent form (ICF)and assent form, understand all study procedures, and can communicate satisfactorily with the investigator and study coordinator, in accordance with applicable laws,regulations, and local requirements.
  2. Male or female between 3 and 12 years (inclusive) at the time of onset and between 3 and 17 years of age (inclusive) at the time of consent.
  3. Body weight ≥ 10 kg.
  4. Diagnosed with childhood absence epilepsy (CAE), confirmed by electroencephalogram(EEG), with ≥ 5 absence seizures during the 4-hour EEG with treatment-resistant absence seizures, and has had an adequate trial of at least 2 anti-epileptic drugs(AED)s and are treatment-resistant to at least one AED.
  5. Willingness to not start a ketogenic diet during the Baseline or Treatment Period.
  6. Have on average ≥ 5 absence seizures per day.
  7. A female patient is eligible to participate in the study if she is premenarchal, or of childbearing potential with a negative urine pregnancy test at the Screening Visit. If sexually active, she must agree to either complete abstinence from intercourse or use acceptable methods of contraception throughout the study and for 4 weeks after completion of study participation or discontinuation from investigational product.
  8. A sexually active male patient must be willing to use acceptable methods of contraception throughout the study and for 4 weeks after completion of study participation or discontinuation from investigational product.
  9. In the opinion of the investigator, the parent(s)/caregiver(s) is willing and able to comply with the study procedures and visit schedules and the Follow-up Visits.
  10. . General good health based on physical and neurological examinations, medical history,and clinical laboratory values completed during the Screening Visit that would prohibit the patient from safely participating in the trial as judged by the investigator.

You CAN'T join if...

  1. Patient or parent(s)/caregiver(s) have daily commitments during the study duration that would interfere with attending all study visits.
  2. Has a history of nonfebrile seizures other than absence seizures.
  3. Has a history of febrile seizures after 3 years of age.
  4. Has a history consistent with juvenile absence epilepsy or juvenile myoclonic epilepsy.
  5. Currently taking felbamate.
  6. Currently taking phenytoin, fluvoxamine, carbamazepine, or St. John's Wort.
  7. Currently taking concomitant medications that are strong inhibitors/inducers/sensitive substrates with a narrow therapeutic index for cytochrome P450 3A4 (CYP3A4), CYP2C9,or CYP2C19.
  8. Currently on a ketogenic diet.
  9. In the opinion of the investigator, any clinically significant, unstable medical abnormality, chronic disease, or a history of a clinically significant abnormality of the cardiovascular, gastrointestinal, respiratory, hepatic, or renal systems.
  10. . History or presence of abnormal electrocardiograms (ECGs) that are clinically significant in the opinion of the investigator.
  11. . Has a current or history of clinically significant intellectual disability or major psychiatric disease, including autism spectrum disorder, which would interfere with compliance.
  12. . For patients aged 7 to 17 years of age and for whom the Columbia Suicide Severity Rating Scale (C-SSRS) is developmentally appropriate, an affirmative answer to queries regarding active suicidal ideation with some intent to act but without a specific plan or active suicidal ideation with a specific plan and intent on the C-SSRS assessment at the Screening Visit.
  13. . Previously received any investigational drug or device or investigational therapy within 30 days before Screening.
  14. . Taken any cannabinoids in the 30 days prior to the Screening Visit. 16. History of an allergic reaction or a known or suspected sensitivity to any substance that is contained in the investigational product formulation.
  15. . Known infection with hepatitis B, hepatitis C, or human immunodeficiency virus (HIV).
  16. . In the opinion of the investigator, the patient is unsuitable in any other way to participate in this study.
  17. . Body weight < 10 kg or > 80 kg.
  18. . Patients who do not have at least 5 absence seizures on 4-hour video-EEG will be considered screen failures.

Locations

  • University of California San Francisco/ Benioff Children's Hospital accepting new patients
    San Francisco, California, 94143, United States
  • Oregon Health and Science University accepting new patients
    Portland, Oregon, 97239, United States
  • Research and Innovation/MultiCare Health System accepting new patients
    Tacoma, Washington, 98405, United States
  • Children's Hospital of Philadelphia accepting new patients
    Philadelphia, Pennsylvania, 19104, United States
  • Nicklaus Children's Hospital accepting new patients
    Miami, Florida, 33155, United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
INSYS Therapeutics Inc
ID
NCT03336242
Phase
Phase 2
Study Type
Interventional
Last Updated
April 2, 2018
I’m interested in this study!