What side effects are associated with this type of intervention?

Epidiolex (cannabidiol) is commonly used as an add-on therapy for reducing seizure frequency and severity in Tuberous Sclerosis Complex. Known side effects of Epidiolex include somnolence, decreased appetite, diarrhea, transaminase elevations, fatigue, malaise, rash, insomnia, sleep disorder, and infections. These side effects are generally consistent with those observed in other treatments that modulate the endocannabinoid system.

What prior FDA approvals exist?

Epidiolex (cannabidiol) is an FDA-approved treatment for seizures associated with Tuberous Sclerosis Complex (TSC). It works by modulating the endocannabinoid system to reduce seizure frequency and severity. Other treatments for TSC-related seizures include traditional antiepileptic drugs such as vigabatrin, which is specifically approved for infantile spasms in TSC, and everolimus, an mTOR inhibitor that has shown efficacy in reducing seizure frequency in TSC patients. These treatments, along with Epidiolex, provide a range of options for managing the complex neuropsychiatric manifestations of TSC.

What other types of research exist?

Promising alternatives to Epidiolex for Tuberous Sclerosis Complex patients include: 1) Everolimus, an mTOR inhibitor that has shown efficacy in reducing seizure frequency in TSC patients. 2) Fenfluramine, which has been effective in reducing seizures in Dravet syndrome and may have potential for TSC. 3) Stiripentol, which has shown benefits in other refractory epilepsy syndromes and could be considered for TSC. These alternatives should be discussed with a healthcare provider to determine the best individualized treatment plan.

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Epidiolex for Tuberous Sclerosis Complex (EpiCom Trial)

Phase 4

Recruiting

Research Sponsored by Jazz Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 4, week 13, week 26, week 52

Awards & highlights

Summary

This trial investigates how adding EPIDIOLEX to existing treatments affects behavior and other outcomes in people aged 1 to 65 with TSC who have seizures. The medication aims to reduce seizure frequency and severity by influencing brain chemicals. Epidiolex, a cannabidiol (CBD) product, has been studied for its safety and effectiveness in treating seizures related to tuberous sclerosis complex (TSC).

Who is the study for?

This trial is for individuals aged 1 to 65 with tuberous sclerosis complex (TSC) who have seizures. Participants must be on stable anti-seizure medication, not have used CBD-OS recently, and agree to consistent contraceptive use if applicable. They cannot join if they've had certain medical conditions or treatments that could affect the study, or a history of substance abuse.

What is being tested?

The study tests Epidiolex (Cannabidiol Oral Solution) as an additional treatment for TSC-related seizures and behavioral issues. It aims to see how adding this solution affects patients' behavior and other related outcomes alongside their current seizure medications.

What are the potential side effects?

Epidiolex may cause side effects such as sleepiness, decreased appetite, diarrhea, fever, vomiting, fatigue. More serious but less common effects include thoughts of suicide or liver problems.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 4, week 13, week 26, week 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 4, week 13, week 26, week 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 4, week 13, week 26, week 52 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of participants considered treatment responders

Number of participants experiencing a worsening, no change, or improvement in seizure frequency

Retention Rate

Side effects data

From 2015 Phase 3 trial • 120 Patients • NCT02091375

31%

Diarrhoea

31%

Somnolence

26%

Decreased appetite

18%

Fatigue

15%

Vomiting

15%

Pyrexia

11%

Upper respiratory tract infection

11%

Lethargy

Convulsion

Weight decreased

Gamma-glutamyltransferase increased

Irritability

Cough

Transaminases increased

Status epilepticus

Nasopharyngitis

Oral herpes

Pneumonia

Platelet count

Abdominal distension

Abdominal pain

Gastrointestinal haemorrhage

Asthenia

Lower respiratory tract infection

Gamma-glutamyltransferase

Hypophagia

Hypotonia

Myoclonus

Respiratory failure

Epistaxis

Hypovolaemic shock

Aspartate aminotransferase increased

Liver function test abnormal

Headache

100%

80%

60%

40%

20%

Study treatment Arm

GWP42003-P 20 mg/kg/Day Dose

Placebo

Trial Design

1Treatment groups

Experimental Treatment

Group I: Cannabidiol Oral SolutionExperimental Treatment1 Intervention

Participants who will receive the Cannabidiol Oral Solution (CBD-OS) titrated up to a dose of 12.5 mg/kg administered twice daily for a total dose of up to 25 mg/kg/day for 26 consecutive weeks. Participants will have the option to continue receiving the CBD-OS for an additional 26 weeks, for a total of 52 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cannabidiol Oral Solution [Epidiolex]

2020

Completed Phase 3

~20

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Tuberous Sclerosis Complex (TSC) Neuropsychiatric Disorder include medications like Epidiolex (Cannabidiol), which modulates the endocannabinoid system to reduce seizure frequency and severity. This is particularly important for TSC patients as seizures are a prevalent and debilitating symptom. Other treatments may include antiepileptic drugs (AEDs) such as topiramate, which stabilizes neuronal membranes and inhibits certain neurotransmitter receptors to prevent seizures. Additionally, behavioral therapies and medications targeting specific neuropsychiatric symptoms, such as ADHD or anxiety, are often used. These treatments are essential for improving the quality of life in TSC patients by managing both seizures and associated neuropsychiatric symptoms.