NCT04485104

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

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Jazz Pharmaceuticals · Industry
• Sex: All
• Age: 1 Month – 23 Months
• Healthy volunteers: Not accepted

Summary

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).

Detailed description

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).

Conditions

• Seizure in Participants With Tuberous Sclerosis Complex
• Seizure in Participants With Dravet Syndrome
• Seizure in Participants With Lennox-Gastaut Syndrome

Interventions

Type	Name	Description
DRUG	GWP42003-P	Oral Solution

Timeline

• Start date: 2021-05-19
• Primary completion: 2025-01-28
• Completion: 2025-01-28
• First posted: 2020-07-24
• Last updated: 2025-10-31
• Results posted: 2025-10-31

Locations

11 sites across 3 countries: United States, Italy, Spain

Regulatory

• FDA-regulated drug study