Symptoms and General Pathology

All Conditions

Musculoskeletal Diseases

Nervous System Diseases

Diseases and Abnormalities at or Before Birth

Neoplasms

Skin and Connective Tissue Diseases

Rare Diseases

Sclerosis

Syndrome

Muscle Cramp

Spasm

Epilepsy

Seizures

Lennox Gastaut Syndrome

Tuberous Sclerosis

Spasms, Infantile

Epilepsies, Myoclonic

Neurologic Manifestations

Hamartoma

Neoplasms, Multiple Primary

Neoplastic Syndromes, Hereditary

Congenital Abnormalities

Malformations of Cortical Development

Nervous System Malformations

Neurocutaneous Syndromes

Heredodegenerative Disorders, Nervous System

Neurodegenerative Diseases

Genetic Diseases, Inborn

Epilepsy, Generalized

Brain Diseases

Central Nervous System Diseases

Epileptic Syndromes

Dravet Syndrome

CDKL5 Deficiency Disorder

Lennox-Gastaut Syndrome

Tuberous Sclerosis Complex

West Syndrome

Pharmaceutical Solutions

All Drugs and Chemicals

Anticonvulsants

Cannabidiol

The 52-week treatment period includes a fixed 2-week titration schedule followed by flexible dose optimization.

Day 1: 5 mg/kg/day (2.5 mg/kg twice daily (b.i.d.))

Day 8: 10 mg/kg/day (5 mg/kg b.i.d.)

Day 15 to Week 52: Flexible dosing based on the participant's observed efficacy, safety, and tolerability per the investigator's clinical judgement. Up to a maximum of 20 mg/kg/day (10 mg/kg b.i.d.) for LGS and DS or 25 mg/kg/day (12.5 mg/kg b.i.d.) for TSC, in maximum weekly increments of 5 mg/kg/day (≤ 2.5 mg/kg b.i.d.).

GWP42003-P

Clinical Trial Disclosure & Transparency

This study will be conducted to evaluate the safety, pharmacokinetics (PK), and efficacy of adjunctive GWP42003-P in participants \< 2 years of age with tuberous sclerosis complex (TSC), Lennox-Gastaut syndrome (LGS), or Dravet syndrome (DS).

The study duration will be up to approximately 62 weeks, including a 4-week screening/baseline period, a 52-week dose optimization treatment period (which includes a fixed 2-week titration period followed by flexible dose optimization), a 10-day taper period, and a safety follow-up period (4 weeks after the end-of-taper visit).

Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures

An Open-label, Single-arm Study to Assess the Safety, Pharmacokinetics, and Efficacy of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Participants With Tuberous Sclerosis Complex (Age 1 Month to < 2 Years of Age), Dravet Syndrome (1 Year to < 2 Years of Age), or Lennox-Gastaut Syndrome (1 Year to < 2 Years of Age) Who Experience Inadequately-controlled Seizures

The CGIC/S is a comprehensive neurodevelopmental assessment that covers the following domains: sensory, motor, cognition, emotional/behavioral health, communication, social, and adaptive functioning. This assessment is a 2-question survey per domain to be completed by the clinician. Individual domain scores will be summed and the total will be reported. Higher scores indicate poor clinical outcome.

The Infant and Toddler Quality of Life Questionnaire Short Form 47 (ITQOL-47) was developed for use in infants and toddlers from 12-months-to-5 years of age and assesses levels of health and well-being. The caregiver will complete the assessment on an electronic device. For each concept, item responses are scored, summed, and transformed on a scale from 0 (worst health) to 100 (best health). Higher scores indicate better clinical outcome.

Treatment Responders are defined as participants with ≥ 50% reduction from baseline in caregiver-reported total countable seizures

This endpoint includes the following changes in percentage of seizures:

* \> 25% (increase);
* ≥ 0% to ≤ 25% (increase);
* \> -25% to \< 0% (reduction);
* \> -50% to ≤ -25% (reduction);
* \> -75% to ≤ -50% (reduction);
* ≤ -75% (reduction).

Jazz Pharmaceuticals

Spots Global Cancer Trial Database for tuberous sclerosis 2