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Epidiolex

Phase 2

Sturge - Weber Syndrome (SWS) | Small molecule | Other |Jazz Pharmaceuticals plc|Last Updated: Aug 21, 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment10

FDA Designations

No designations recorded

Clinical trial landscape

Epidiolex · 2 trials · 2 indications

Phase 2 1Phase 1 1
NCT07778810Epidiolex Trial for Presymptomatic Treatment of Sturge-Weber SyndromeSturge - Weber Syndrome (SWS)
NOT YET_RECRUITING10 Analytics
PHASE2NOT YET_RECRUITING
Epidiolex Trial for Presymptomatic Treatment of Sturge-Weber Syndrome
Sturge - Weber Syndrome (SWS)Unlock trial analytics

Study Endpoints

Primary Endpoints

Safe completion as assessed by the number of participants with no significant side effects
6 months
Safe completion as assessed by the number of participants with no seizure onset
6 months
Frequency of Spike Wave Index
Up to 20 weeks

Assess the reduction in spike wave index by evaluating the differences in activity from the four 24-hour ambulatory EEGs each participant obtains while enrolled in this study

Secondary Endpoints

Sturge-Weber Syndrome Neuroscore (SWS-NRS)
baseline, week 5, week 12, and week 24
Milestone checklist
baseline, week 5, week 12, and week 24
Modified Early Motor Questionnaire (EMQ)
Baseline, week 24
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Presymptomatic Epidiolex TreatmentEXPERIMENTALEpidiolex will be started at 2.5 mg/kg/day divided twice daily. The dose will increase weekly as tolerated over the next 5 weeks to a target dose of 20 mg/kg/day.
IPACTIVE_COMPARATOREpidiolex (Cannabidiol) is a colorless to yellow solution in a 100mL vial that is to be administered with a 5mL syringe.The starting dosage is 2.5 mg/kg twice daily, or 5 mg/kg/day. After one week, the dosage can be increased to a maintenance dosage of 5mg/kg twice daily, or 10 mg/kg/day. Patients who are tolerating Epidiolex at 10mg/kg per day and require further reduction of seizures may benefit from a dosage increase up to a maximum recommended maintenance. Dosage of 10mg/kg twice daily (20mg/kg/day), in weekly increments of 2.5 mg/kg twice daily(5mg/kg/day), as tolerated. For patients in whom a more rapid titration from 10 mg/kg/day to 20 mg/kg/day is warranted, the dosage may be increased to no more frequently than every other day administration of the 20 mg/kg/day. Dosage resulted in somewhat greater reductions in seizure rates than the recommended maintenance dosage of 10 mg/kg/day, but with an increase in adverse reactions.
PlaceboPLACEBO_COMPARATORPlacebo is a colorless to yellow solution in a 100mL vial that is to be administered with a 5mL syringe. The starting dosage is 2.5 mg/kg twice daily, or 5 mg/kg/day. After one week, the dosage can be increased to a maintenance dosage of 5mg/kg twice daily, or 10 mg/kg/day. Patients who are tolerating Placebo at 10mg/kg per day and require further reduction of seizures may benefit from a dosage increase up to a maximum recommended maintenance. Dosage of 10mg/kg twice daily (20mg/kg/day), in weekly increments of 2.5 mg/kg twice daily(5mg/kg/day), as tolerated. For patients in whom a more rapid titration from 10 mg/kg/day to 20 mg/kg/day is warranted, the dosage may be increased to no more frequently than every other day administration of the 20 mg/kg/day. Dosage resulted in somewhat greater reductions in seizure rates than the recommended maintenance dosage of 10 mg/kg/day, but with an increase in adverse reactions.

Interventions

NameTypeDescription
Epidiolex 100 mg/mL Oral SolutionDRUGEpidiolex is a prescription form of cannabidiol used to treat seizures. Epidiolex will be started at 2.5 mg/kg/day divided twice daily. The dose will increase weekly as tolerated over the next 5 weeks to a target dose of 20 mg/kg/day.
PlaceboOTHERPlacebo is composed of dehydrated alcohol, sesame seed oil, strawberry flavor, and sucralose.
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Eligibility Criteria

Age Range1 Month to 18 Months
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Clinical diagnosis of Sturge-Weber Syndrome. * Age 1 to 18 months of age, inclusive. * Neuroimaging demonstrating involvement of 3 or more lobes, or bilateral involvement. * No history of seizures. * Patient's parent or legal guardian provides written informed consent prior to...

Countries:United States
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Frequently asked questions about Epidiolex

What is Epidiolex/mL used for?

Epidiolex/mL is an investigational oral solution being studied for Sturge-Weber Syndrome (SWS) and Electrical Status Epilepticus of Slow-Wave Sleep (ESES). It is a small molecule developed by Jazz Pharmaceuticals plc (JAZZ). The drug is currently in Phase 1 and Phase 2 clinical trials for these conditions.

What does Epidiolex/mL target?

Epidiolex/mL is a small molecule drug. Its specific molecular target has not been disclosed in available clinical trial information. The drug is being evaluated for its efficacy in treating Electrical Status Epilepticus of Slow-Wave Sleep and Sturge-Weber Syndrome, both of which involve neurological abnormalities.

Who makes Epidiolex/mL?

Epidiolex/mL is developed by Jazz Pharmaceuticals plc, which trades under the ticker JAZZ. The company is conducting clinical trials for the drug in the United States, including a completed Phase 1 study and a planned Phase 2 study for Sturge-Weber Syndrome.

What phase is Epidiolex/mL in?

Epidiolex/mL is in early clinical development. A Phase 1 trial for Electrical Status Epilepticus of Slow-Wave Sleep has been completed, and a Phase 2 trial for Sturge-Weber Syndrome is not yet recruiting. The drug is investigational and not approved for any use.

What clinical trials is Epidiolex/mL in?

Epidiolex/mL has two registered trials. NCT04721691 is a completed Phase 1 study in Electrical Status Epilepticus of Slow-Wave Sleep with 16 participants. NCT07778810 is a Phase 2 trial for presymptomatic Sturge-Weber Syndrome, not yet recruiting, with a planned enrollment of 10.

Is Epidiolex/mL the same as Epidiolex 100 mg/mL Oral Solution?

Yes, Epidiolex/mL is also known as Epidiolex 100 mg/mL Oral Solution. This alternative name reflects the drug's formulation as an oral solution at a concentration of 100 mg/mL. Both names refer to the same investigational drug product developed by Jazz Pharmaceuticals.