Epidiolex for Anxiety in Pediatric Epilepsy

Overseen By Catherine Eliades

Age: < 18

Sex: Any

Trial Phase: Phase 4

Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Must be taking: Anticonvulsants

Must not be taking: Benzodiazepines

Disqualifiers: Psychotic illness, Substance abuse, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 16 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests Epidiolex, a pharmaceutical-grade cannabidiol, to determine if it reduces anxiety in children with difficult-to-treat epilepsy. Researchers aim to assess whether this treatment is effective and well-tolerated alongside regular seizure medications. The trial seeks children aged 6 to 18 with ongoing epilepsy and anxiety affecting daily life, who have not recently used CBD products. Participants must take the medicine orally and have a parent or caregiver to help monitor progress. As a Phase 4 trial, Epidiolex is already FDA-approved and proven effective, and this research seeks to understand how it benefits more patients.

Will I have to stop taking my current medications?

The trial does not require you to stop taking your current anticonvulsant medications, as ongoing use is necessary. However, you must not use any CBD products for 14 days before the study and during the study. If you are taking benzodiazepines regularly, you may need to stop, except for emergency use.

What is the safety track record for Epidiolex?

Research has shown that Epidiolex, a type of cannabidiol (CBD) medicine, is generally safe for children with epilepsy. Studies have found that it can help reduce seizures in many children. While using Epidiolex, some children might experience side effects. About 9.7% of children taking CBD reported side effects, compared to 4% of those not taking it. These side effects are usually mild, such as drowsiness or changes in appetite.

Previous research also found CBD to be safe and well-tolerated for children with epilepsy. Since the FDA approved Epidiolex for treating seizures in epilepsy, confidence in its safety for this condition is high.

In summary, Epidiolex is considered safe and effective, though some children may experience minor side effects. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Unlike the standard treatments for pediatric epilepsy-related anxiety, which often include medications like benzodiazepines or antidepressants, Epidiolex is unique because it's derived from cannabidiol (CBD), a compound found in cannabis. This offers a novel approach as it targets the body's endocannabinoid system, potentially providing relief without the psychoactive effects associated with THC, another cannabis component. Researchers are excited about Epidiolex because it might offer a safer, non-addictive alternative with fewer side effects compared to traditional anxiety medications, which can sometimes lead to dependency or require long-term use.

What evidence suggests that Epidiolex might be an effective treatment for anxiety in pediatric epilepsy?

Research has shown that Epidiolex, a high-quality form of cannabidiol (CBD), effectively treats epilepsy and may also help with anxiety. Studies have found it can significantly reduce seizures, with some individuals experiencing a 51–75% decrease. Early results suggest it also helps with anxiety in children with epilepsy. In this trial, participants will receive Epidiolex to evaluate its effects on anxiety in pediatric epilepsy. Epidiolex is already approved for controlling seizures, and its use for anxiety appears promising. Most people tolerate it well, as it usually doesn't cause serious side effects. ^{1 6 7 8 9}

Who Is on the Research Team?

Jay Salpekar

Principal Investigator

Principal Investigator

Are You a Good Fit for This Trial?

This trial is for children and teens aged 6-18 with epilepsy and anxiety who are on anticonvulsant medication, haven't had severe seizures recently, can take oral medicine, and aren't using other CBD products. Participants need reliable caregivers for consent and must use birth control if sexually active.

Inclusion Criteria

Participants who are sexually active must agree to sexual abstinence or to use an approved birth control method for the full duration of study participation

I experience anxiety that affects my daily activities.

I have epilepsy and need medication to control my seizures.

Exclusion Criteria

Pregnant, planning to become pregnant, breastfeeding, or failing to use an appropriate method of contraception

My caregiver or legal guardian cannot fully understand or consent to the study procedures for me.

I am taking epilepsy drugs, but I'm open to extra monitoring if needed.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive active treatment with flexible dose titration of Epidiolex®

16 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Epidiolex

Trial Overview The study tests Epidiolex (cannabidiol) to see if it helps with anxiety in young patients with hard-to-treat epilepsy. It's an open label study where everyone knows they're getting the actual drug, not a placebo.

How Is the Trial Designed?

Group I: treatment arm Experimental Treatment 1 Intervention

open label treatment intervention

Epidiolex is already approved in United States, European Union for the following indications:

Approved in United States as Epidiolex for:

• Seizures associated with Lennox-Gastaut syndrome
• Seizures associated with Dravet syndrome
• Seizures associated with tuberous sclerosis complex

Approved in European Union as Epidyolex for:

• Seizures associated with Lennox-Gastaut syndrome
• Seizures associated with Dravet syndrome

Who Is Running the Clinical Trial?

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Lead Sponsor

Trials

93

Recruited

25,200+

Published Research Related to This Trial

Epidiolex® (cannabidiol) is an FDA-approved medication for treating epilepsy associated with Lennox-Gastaut and Dravet syndromes, showing promise for treatment-resistant epilepsy.

A case study of a 23-year-old female revealed a delayed skin rash related to Epidiolex®, highlighting the need to monitor for potential side effects even when they are rare in clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Epidiolex-induced skin rash. Singh, J., Antimisiaris, MF.[2021]

Pure, plant-derived cannabidiol (CBD; Epidiolex) has been shown to significantly reduce seizure frequencies in patients with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) by 17% to 23% compared to placebo, making it an effective adjunctive therapy for treatment-resistant epilepsy.

CBD is generally well tolerated, with common side effects including somnolence, diarrhea, and elevated liver enzymes, and it is the first cannabis-derived medication approved by the FDA for these conditions, providing a new treatment option for patients aged 2 years and older.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cannabidiol: A New Hope for Patients With Dravet or Lennox-Gastaut Syndromes. Chen, JW., Borgelt, LM., Blackmer, AB.[2020]

In a study of 108 patients with refractory epilepsy taking Epidiolex®, 75% remained on the treatment at the final evaluation, indicating that it is generally well-tolerated and effective for long-term use.

However, 46.3% of patients experienced treatment-emergent adverse effects, with 14.5% discontinuing due to these effects, highlighting the need for further research on managing side effects and drug interactions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real-world, long-term evaluation of the tolerability and therapy retention of Epidiolex® (cannabidiol) in patients with refractory epilepsy. Georgieva, D., Langley, J., Hartkopf, K., et al.[2023]

Epidiolex, a 99% pure oral CBD extract, has been shown to significantly reduce seizure frequency in patients with medically refractory epilepsy, leading to its FDA approval.

The review highlights the safety profile of Epidiolex, noting that while it is effective, understanding its adverse effects is crucial for patient management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Epidiolex as adjunct therapy for treatment of refractory epilepsy: a comprehensive review with a focus on adverse effects. Sekar, K., Pack, A.[2020]

In a study of 47 patients with refractory epilepsy, highly purified cannabidiol (CBD) was found to be effective in reducing seizure frequency, regardless of whether it was used with clobazam (CLB) or not.

Patients taking CBD alongside CLB had a significantly higher responder rate (more than 50% reduction in seizures) at the point of best seizure control within the first year, indicating that combining these treatments may enhance efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of cannabidiol in subjects with refractory epilepsy relative to concomitant use of clobazam. Savage, TE., Sourbron, J., Bruno, PL., et al.[2021]

Epidiolex® (CBD) is effective for treating epilepsy in patients over 2 years old, but it can interact with other medications, as seen in a case where it contributed to lithium toxicity in a patient with Lennox-Gastaut syndrome.

After discontinuing lithium due to toxicity, the patient improved and remained seizure-free on CBD and two other antiseizure medications, highlighting the importance of monitoring drug interactions when adding CBD to treatment regimens.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Drug-Drug Interactions Between Cannabidiol and Lithium. Singh, RK., Dillon, B., Tatum, DA., et al.[2020]

In a study involving 125 pediatric patients with rare epilepsies, a slower titration protocol for cannabidiol (CBD) resulted in better tolerance and a significant reduction in seizure frequency, with 37.8% of patients experiencing at least a 50% reduction in seizures after 6 months.

While adverse events (AEs) were reported in nearly half of the patients, mainly including somnolence and asthenia, the overall efficacy of CBD was comparable to previous studies, suggesting that a gradual increase in dosage may enhance patient safety without compromising therapeutic benefits.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Slow Titration of Cannabidiol Add-On in Drug-Resistant Epilepsies Can Improve Safety With Maintained Efficacy in an Open-Label Study. D'Onofrio, G., Kuchenbuch, M., Hachon-Le Camus, C., et al.[2020]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7439947/

Emerging Use of Epidiolex (Cannabidiol) in Epilepsy - PMC In addition to demonstrating efficacy in terms of improved seizure control and QoL, CBD has shown to be tolerable, with the most common (≥10%) ADRs including ...

2. aesnet.org aesnet.org/abstractslisting/impact-of-cannabidiol-on-seizures-and-anxiety-symptoms-in-pediatric-epilepsy-patients--a-preliminary-analysis

Impact-of-Cannabidiol-on-Seizures-and-Anxiety-Symptoms-in ... In this ongoing study, we examine the effect of Epidiolex on seizure reduction and on the improvement of anxiety symptoms in pediatric epilepsy patients.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05324449

NCT05324449 | Epidiolex® for Anxiety in Pediatric Epilepsy This is a prospective open label study assessing the efficacy and tolerability of pharmaceutical grade cannabidiol--Epidiolex (R), for the treatment of anxiety ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10166640/

Clinical Outcome Data of Children Treated with Cannabis ... Primary outcomes were ≥50% reduction in seizure frequency, changes in the Impact of Pediatric Epilepsy Score (IPES), and incidence of adverse events. Results ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1525505025001659

Adjunctive use of cannabidiol in pediatric drug-resistant ... Highlights · CBD significantly reduced seizure frequency in pediatric DRE patients. · 21 % of patients had a 51–75 % reduction in seizures after CBD treatment.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10949818/

The efficacy and safety of cannabidiol (CBD) in pediatric ... The analysis revealed promising efficacy outcomes. Notably, CBD demonstrated substantial reductions in seizure frequency, with some patients ...

7. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1525505023002494

Review The use of cannabinoids in children with epilepsy Conclusion. The studies showed good methodological rigor and characterized the use of CBD as effective and safe in the treatment of children with epilepsy. If ...

8. jamanetwork.com jamanetwork.com/journals/jamanetworkopen/fullarticle/2803957

Adverse Events of Cannabidiol Use in Patients With Epilepsy Results Nine studies were included. Overall incidences of 9.7% in the CBD group and 4.0% in the control group were found for any grade AEs.

9. neurology.org neurology.org/doi/10.1212/WNL.0000000000202697

Real-world Outcomes of Cannabidiol For The treatment ... Following CBD therapy in patients identified to have genetic etiology of epilepsy, 3 patient(23%)had no improvement, 4(30.7%)had mild improvement, and 6(46.1%) ...