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

Who is this study for? Infant patients with tuberous sclerosis complex
What treatments are being studied? GWP42003-P+Standard of care
Status: Terminated
Location: See all (11) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 1 month
Maximum Age: 1
Healthy Volunteers: f
View:

• Participants with TSC (1 month to \< 2 years of age), or DS (1 year to \< 2 years of age), or LGS (1 year to \< 2 years of age) within the specified age range at the time of initial informed consent.

• Parent(s)/legal representative is/are willing and able to give informed consent for participation in the study.

• Parent(s)/legal representative is/are willing and able (in the investigator's opinion) to comply with all study requirements (including accurate electronic participant-reported outcome \[ePRO\] diary completion).

• Participants with TSC must have a diagnosis per the 2012 International Tuberous Sclerosis Complex Consensus Conference. Participants with LGS or DS must have a diagnosis that is consistent with International League Against Epilepsy (ILAE) guidelines and confirmed by the Epilepsy Study Consortium (ESCI).

• Participants who have uncontrolled seizures, and who are currently receiving 1 or more antiseizure medication (ASMs).

• A suitable VEEG, as available in the medical record, within 1 year of Visit 1. When a historical VEEG is not available, and if clinically indicated and appropriate (due to uncertainties or new seizures), a VEEG will be completed and read to confirm diagnosis prior to Visit 3. All VEEGs are to be read at baseline by the investigator and by an independent reviewer.

• Has seizures which are not adequately controlled through their current ASMs, defined as ≥ 1 seizure reported on the seizure diary during the screening/baseline period

Locations
United States
Arkansas
Clinical Trial Site
Little Rock
California
Clinical Trial Site
Los Angeles
Illinois
Clinical Trial Site
Chicago
Massachusetts
Clinical Trial Site
Boston
Ohio
Clinical Trial Site
Cincinnati
Texas
Clinical Trial Site
Houston
Other Locations
Italy
Clinical Trial Site
Florence
Clinical Trial Site
Genova
Clinical Trial Site
Rome
Spain
Clinical Trial Site
Barcelona
Clinical Trial Site
Madrid
Time Frame
Start Date: 2021-05-19
Completion Date: 2025-01-28
Participants
Target number of participants: 3
Treatments
Experimental: GWP42003-P
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.).
Sponsors
Leads: Jazz Pharmaceuticals
Collaborators: Jazz Pharmaceuticals Research UK Limited

This content was sourced from clinicaltrials.gov

Similar Clinical Trials