BridgeMD
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
EU CTIS ID: 2023-505851-33-00
What this study is testing
To evaluate the safety and tolerability of adjunctive GWP42003-P assessed during the 52-week treatment period To investigate the exposure of GWP42003-P and its major metabolites following multiple doses of GWP42003-P To evaluate the efficacy of GWP42003-P in reducing the frequency of indication-specific countable seizures.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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)/LAR is/are willing to allow the responsible authorities to be notified of participation in the study, if mandated by local law
- Parent(s)/LAR is/are willing to allow the participant’s primary care practitioner (if they have one) and consultant (if they have one) to be notified of participation in the study if the primary care practitioner/consultant is different from the investigator
- 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 ASMs.
- Parent(s)/LAR is/are willing and able to give informed consent for participation in the study
You likely can't join if
- Has clinically significant unstable medical condition other than epilepsy
- Has any concurrent cardiovascular conditions that will, in the investigator’s opinion, interfere with the ability to assess their ECGs
- Has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the study intervention such as sesame seed oil
- Has significantly impaired hepatic function prior to Visit 3, defined as: • Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 × upper limit of normal (ULN) and (total bilirubin [TBL] > 2 × ULN or international normalized ratio [INR] > 1.5). • Serum ALT or AST > 5 × ULN. • Serum ALT or AST > 3 × ULN with the presence of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia (> 5%). • Elevated ALT or AST should be discussed with the medical monitor prior to Visit 3; the medical monitor may allow for a confirmatory re-draw prior to Visit 3.
- Has received another study intervention within 4 weeks prior to Visit 1 or plans to take another study intervention during the study
- Caregiver is currently giving or has given recreational or medicinal cannabis, cannabinoid-based medications (including Sativex) or CBD (including Epidiolex/ Epidyolex [GWP42003-P]) to the participant within the 4 weeks prior to Visit 1 or is unwilling to abstain from doing so for the duration of the study
See the full eligibility criteria
Who can join
- 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)/LAR is/are willing to allow the responsible authorities to be notified of participation in the study, if mandated by local law
- Parent(s)/LAR is/are willing to allow the participant’s primary care practitioner (if they have one) and consultant (if they have one) to be notified of participation in the study if the primary care practitioner/consultant is different from the investigator
- 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 ASMs.
- Parent(s)/LAR is/are willing and able to give informed consent for participation in the study
- Parent(s)/LAR is/are willing and able (in the investigator’s opinion) to comply with all study requirements (including accurate electronic patient-reported outcome [ePRO] diary completion)
- Caregiver completes at least 75% of ePRO and paper seizure diary entries during the 28 days of the baseline period (≥ 21 days of entries)
- 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 an independent reviewer. • A suitable VEEG meets all the following criteria: i. Multichannel (minimum 8-channel) ii. Prolonged continuous recording up to 24 hours iii. Completed within 1 year of Visit 1 iv. Consistent with the participant’s current seizures (in the investigator’s opinion) v. Can be reviewed by the investigator and an independent reviewer prior to Visit 3 vi. Consistent with a diagnosis of inadequately-controlled seizures
- Currently taking ≥ 1 ASMs at a dose that remains stable 2 weeks prior to Visit 3 and during the treatment period. Where required for participant safety, adjustments of concomitant ASMs or addition of new ASM may be permitted following discussion with the medical monitor. • Adrenocorticotropic hormone (ACTH) or high dose corticosteroids for the treatment of IS/ES are counted as ASMs.
- Has seizures that are not adequately controlled through their current ASMs, defined as ≥ 1 seizure reported on the seizure diary during the screening/baseline period.
What rules you out
- Has clinically significant unstable medical condition other than epilepsy
- Has any concurrent cardiovascular conditions that will, in the investigator’s opinion, interfere with the ability to assess their ECGs
- Has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the study intervention such as sesame seed oil
- Has significantly impaired hepatic function prior to Visit 3, defined as: • Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 × upper limit of normal (ULN) and (total bilirubin [TBL] > 2 × ULN or international normalized ratio [INR] > 1.5). • Serum ALT or AST > 5 × ULN. • Serum ALT or AST > 3 × ULN with the presence of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia (> 5%). • Elevated ALT or AST should be discussed with the medical monitor prior to Visit 3; the medical monitor may allow for a confirmatory re-draw prior to Visit 3.
- Has received another study intervention within 4 weeks prior to Visit 1 or plans to take another study intervention during the study
- Caregiver is currently giving or has given recreational or medicinal cannabis, cannabinoid-based medications (including Sativex) or CBD (including Epidiolex/ Epidyolex [GWP42003-P]) to the participant within the 4 weeks prior to Visit 1 or is unwilling to abstain from doing so for the duration of the study
- Mother (if breastfeeding) is currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex) or CBD (including Epidiolex/Epidyolex [GWP42003-P]) within the 4 weeks prior to Visit 1 or is unwilling to abstain from doing so for the duration of the study
- Has any other clinically significant disease or disorder which, in the opinion of the investigator, may either put the participant, other participants, or site staff at risk because of participation in the study, may influence the result of the study, or may affect the participant’s ability to take part in the study
- Any clinically significant abnormalities identified following a physical examination of the participant that, in the opinion of the investigator, would jeopardize the safety of the participant if they took part in the study
- Has previously been enrolled into this study
- Has plans to travel outside their country of residence during the study, unless the participant has confirmation that the study intervention is permitted in the destination country and all stops along the way
- Has had clinically significant symptoms or a clinically significant illness within the 4 weeks prior to Visit 1, other than epilepsy, which in the opinion of the investigator could affect seizure frequency
- Has undergone general anesthesia within 4 weeks prior to Visit 1
- Has undergone surgery for epilepsy within 6 months prior to Visit 1 or has plans to undergo surgery for epilepsy during the study
- Has taken felbamate < 1 year prior to Visit 1
- Is < 1 year of age and taking valproic acid
- Has tumour growth which, in the opinion of the investigator, could affect participant safety
- Has clinically significant abnormal laboratory values, in the investigator’s opinion, at screening/baseline
- Has clinically significant abnormalities in the ECG measured at screening/baseline
The study team makes the final eligibility decision.
Where it's taking place
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.