Phase 3
N=224
A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
Tuberous Sclerosis Complex · Seizures
Bottom Line
View on ClinicalTrials.gov: NCT02544763 ↗Enrolled (actual)
224
Serious AEs
12.5%
Results posted
Sep 2020
Primary outcome: Primary: Percent Change From Baseline in the Number of Tuberous Sclerosis Complex (TSC)-Associated Seizures During the Treatment Period (Maintenance and Titration) — -43.36; -36.55; -20.08 percent change — p==0.0009
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- GWP42003-P (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 1+ yrs
- Sex
- All
- Sponsor
- Jazz Pharmaceuticals
- Primary completion
- Jan 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in the Number of Tuberous Sclerosis Complex (TSC)-Associated Seizures During the Treatment Period (Maintenance and Titration) |
-43.36; -36.55; -20.08 | =0.0009 sig |
| SECONDARY Number of Participants Considered Treatment Responders During the Treatment Period (Maintenance and Titration) |
27; 29; 17; 48; 44; 59 | =0.0692 |
| SECONDARY Change From Baseline in the Caregiver Global Impression of Change (CGIC) or Participant Global Impression of Change (PGIC) Score at the Participant's Last Visit |
3.0; 3.1; 3.5; 3.0; 3.2; 3.5 | =0.0074 sig |
| SECONDARY Percent Change From Baseline in Total Seizures During the Treatment Period (Maintenance and Titration) |
-34.71; -35.14; -19.63 | =0.0013 sig |
| SECONDARY Number of Participants With Any Severe Treatment-emergent Adverse Event (TEAE) |
7; 9; 1 | — |
Summary
This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The blinded phase only will be described in this record. Participants will receive 1 of 2 doses of GWP42003-P or matching placebo. The primary clinical hypothesis is that there will be a difference between GWP42003-P and placebo in their effect on seizure frequency.
Eligibility Criteria
Key Inclusion Criteria
- Participant has a well-documented clinical history of epilepsy.
- Participant has a clinical diagnosis of Tuberous Sclerosis Complex (TSC) according to the criteria agreed by the 2012 International TSC Consensus Conference.
- All medications or interventions for epilepsy (including ketogenic diet and any neurostimulation devices for epilepsy) must have been stable for 1 month prior to screening and the participant is willing to maintain a stable regimen throughout the trial.
Key Exclusion Criteria
- Participant has a history of pseudo-seizures.
- Participant has clinically significant unstable medical conditions other than epilepsy.
- Participant has an illness in the 4 weeks prior to screening or randomization, other than epilepsy, which in the opinion of the investigator could affect seizure frequency.
- Participant has undergone general anesthetic in the 4 weeks prior to screening or randomization.
- Participant has undergone surgery for epilepsy in the 6 months prior to screening.
- Participant is being considered for epilepsy surgery or any procedure involving general anesthesia.
- Participant has been taking felbamate for less than 1 year prior to screening.
- Participant is taking an oral mTOR inhibitor.
- Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the Investigational Medicinal Product (IMP), such as sesame oil.
- Participant has any history of suicidal behavior or any suicidal ideation of type 4 or 5 on the C-SSRS in the last month or at screening.
- Participant is currently using or has in the past used recreational or medicinal cannabis, or cannabinoid-based medications, within the 3 months prior to screening and is unwilling to abstain for the duration for the study.
- Participant has tumor growth which, in the opinion of the Investigator, could affect the primary endpoint.
- Participant has significantly impaired hepatic function at the screening or randomization visit
- Participant has received an IMP within the 12 weeks prior to the screening visit.
Data sourced from ClinicalTrials.gov (NCT02544763). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.