A Study to Evaluate the Safety and Efficacy of CBD-OS in Participants With DEE

Официальное название

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Cannabidiol Oral Solution (CBD-OS, JZP926-OS) in Participants Aged 1 Year and Older With Developmental and Epileptic Encephalopathy (DEE)

Обзор

The efficacy, safety, and tolerability of CBD-OS have been evaluated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and Tuberous sclerosis complex (TSC). The current JZP926-303 study is being conducted to evaluate the safety and efficacy of CBD-OS in participants with Developmental and Epileptic Encephalopathy (DEE).

Подробное описание

This Phase 3, multicenter, randomized, placebo-controlled, double-blind study will evaluate the efficacy and safety of CBD-OS in participants aged ≥ 1 year with DEE. The primary objective of the 6-week Double-blind Treatment Period of the study is to assess the efficacy of CBD-OS in reducing the frequency of countable motor seizures compared with placebo in participants with DEE. In addition, the Double-Blind Treatment Period will also assess the safety and tolerability of CBD-OS. The optional 6-month open-label extension (OLE) will provide additional data on the long-term efficacy, safety, and tolerability of CBD-OS.

Вмешательства

• Препарат CBD-OS
  Oral solution, twice daily
• Препарат Placebo
  Oral solution, twice daily

Первичные конечные точки

• Change in Countable Motor Seizure Frequency Per 28 Days [Срок оценки: Baseline up to 6 weeks of double-blind treatment period]

Вторичные конечные точки (11)

• Change in Total Seizure Frequency Per 28 Days [Срок оценки: Baseline up to 6 weeks of double-blind treatment period]
• Proportion of Participants Who Achieve ≥ 50% Reduction From Baseline in Countable Motor Seizure Frequency [Срок оценки: Baseline up to 6 weeks of double-blind treatment period]
• Caregiver Global Impression of Change (CaGI-C) Score [Срок оценки: Week 6 of double-blind treatment period]
• Change from Baseline in Caregiver Global Impression of Severity (CaGI-S) Score [Срок оценки: Week 6 of double-blind treatment period]
• Change From Baseline in Number of Countable Motor Seizure-free Days per 28 Days [Срок оценки: Baseline up to 6 weeks of double-blind treatment period]
• Clinical Global Impression of Change (CGI-C) Score [Срок оценки: Week 6 of double-blind treatment period]
• Change from Baseline in Clinical Global Impression of Severity (CGI-S) Score [Срок оценки: Week 6 of double-blind treatment period]
• Number of Participants Reporting Treatment-emergent Adverse Events [Срок оценки: Baseline up to 6 weeks of double-blind treatment period]
• Mean Plasma Concentration of CBD [Срок оценки: Baseline up to 6 weeks of double-blind treatment period]
• Mean Plasma Concentration of Metabolite 7-OH-CBD [Срок оценки: Baseline up to 6 weeks of double-blind treatment period]
• Mean Plasma Concentration of Metabolite 7-COOH-CBD [Срок оценки: Baseline up to 6 weeks of double-blind treatment period]

Критерии участия

Participants are eligible to be included in the study only if all the following criteria apply:

• Is at least 1 year of age at the time of signing the informed consent/assent.
• Meets the clinical phenotype for DEE as specified in the protocol.
• Per the investigator, the underlying etiology contributes to developmental impairment and seizures.
• Has had, or is willing to complete, confirmatory imaging and/or genetic testing to determine etiology of DEE.
• Is currently receiving antiseizure intervention, such as treatment with a stable regimen of at least 1 ASM or an established intervention for epilepsy (eg, ketogenic diet or neurostimulation).
• All medications or interventions for epilepsy have been stable for ≥ 28 days prior to starting the baseline period (Visit 2) with no planned changes to the regimen for the duration of the Double-blind Treatment Period.

Participants are excluded from the study if any of the following criteria apply:

• Has a concurrent, confirmed diagnosis of non-epileptic seizures or events that can confound the assessment of the efficacy measures, in the opinion of the investigator.
• The etiology of the participant's seizures is a progressive neurologic disease.
• Has known or suspected hypersensitivity to cannabinoids or any of the excipients of the study intervention, such as sesame oil.
• Has an active central nervous system (CNS) infection, demyelinating disease, degenerative neurologic disease, or any CNS disease deemed to be progressive during the study that may confound the interpretation of the study results (including autoimmune encephalitis).
• Is currently being treated with Epidiolex or recently received treatment with Epidiolex within 28 days prior to screening.
• Has experienced a lack of efficacy and/or poor tolerability to an adequate treatment regimen of Epidiolex based on medical history and the clinical judgement of the investigator. Participants who discontinued treatment for reasons other than safety, tolerability, or lack of efficacy and previously received Epidiolex ≥ 28 days prior to starting the Baseline Period (Visit 2) may be eligible for the study after consultation with the medical monitor and/or sponsor representative.
• Has been taking felbamate for less than 12 months prior to screening. Participants who are stable on felbamate for ≥ 12 months are eligible for inclusion.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Двойное слепое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07723976 · JZP926-303

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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