Epidiolex (cannabidiol) / Jazz - LARVOL DELTA

A PILOT STUDY EVALUATING CANNABIDIOL ORAL SOLUTION IN BILATERAL TOTAL KNEE ARTHROPLASTY (ASRA-SPRING 2025) - P4 | "[1-2] In studies using animal models of acute and chronic pain, CBD relieves hyperalgesia and edema associated with inflammation, and reduces allodynia and anxiety associated with nerve injury.[2] Epidiolex® is the only CBD formulation approved by the Food and Drug Administration (FDA) and is indicated for the treatment of seizures associated with Lennox-Gastaut Syndrome, Dravet Syndrome, and tuberous sclerosis complex...The primary outcome was cumulative 72-hour opioid consumption, calculated from procedure end, in morphine milligram equivalents (MMEs)...Though not powered to detect statistical significance, the approximate 30% lower opioid requirements in patients who received 400mg oral cannabidiol solution suggests promising efficacy of this dose, but inconclusive results for 800 mg. Additional analyses of our pilot data will help elucidate tolerability of CBD and reasons for an apparent lack of dose response, including potential biphasic effects of CBD at..."

Clinical • Anesthesia • CNS Disorders • Epilepsy • Inflammation • Orthopedics • Pain • Psychiatry

Epidiolex in Obsessive Compulsive Disorder and Related Disorders (clinicaltrials.gov) - P2 | N=4 | Active, not recruiting | Sponsor: University of Chicago | N=15 ➔ 4 | Recruiting ➔ Active, not recruiting

Enrollment change • Enrollment closed • Mood Disorders • Movement Disorders • Obsessive-Compulsive Disorder • Psychiatry • Tourette Syndrome

Adjunctive use of cannabidiol in pediatric drug-resistant epilepsy: A retrospective multicenter analysis. (PubMed, Epilepsy Behav) - "CBD has proven to be an effective adjunctive treatment for medically refractory epilepsy, showing significant efficacy across various epilepsy etiologies and genetic backgrounds. Its ability to reduce seizure frequency and the burden of anti-seizure medications (ASMs), especially in syndromes that are traditionally difficult to manage, highlights its value as an additional therapeutic option."

Journal • Retrospective data • CNS Disorders • Epilepsy • Pediatrics

SPICE: Cannabidiol in Sickle Cell Disease (clinicaltrials.gov) - P1/2 | N=52 | Not yet recruiting | Sponsor: Icahn School of Medicine at Mount Sinai

New P1/2 trial • Genetic Disorders • Hematological Disorders • Inflammation • Pain • Sickle Cell Disease

OPERA: Long-Term Follow-Up of a Cohort of Participants Prescribed Epidyolex in France in a Real-life Setting (clinicaltrials.gov) - P=N/A | N=158 | Active, not recruiting | Sponsor: Jazz Pharmaceuticals | Completed ➔ Active, not recruiting | Trial completion date: Jul 2024 ➔ Jul 2026 | Trial primary completion date: Jul 2024 ➔ Jul 2026

Enrollment closed • Trial completion date • Trial primary completion date • CNS Disorders • Epilepsy

CANN-SWITCH: A Study to Investigate the Transition of Children From 'Artisanal" Cannabidiol (CBD) to Epidiolex (clinicaltrials.gov) - P4 | N=25 | Not yet recruiting | Sponsor: Elizabeth Donner

New P4 trial • CNS Disorders • Epilepsy

CAN-SAD: Investigating the Effects of Cannabidiol on Social Anxiety Disorder (clinicaltrials.gov) - P4 | N=50 | Not yet recruiting | Sponsor: Massachusetts Institute of Technology | Trial completion date: Sep 2025 ➔ Sep 2026 | Trial primary completion date: May 2025 ➔ May 2026

Trial completion date • Trial primary completion date • CNS Disorders • Mood Disorders • Psychiatry • Social Anxiety Disorder

EpiCom: A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex (clinicaltrials.gov) - P4 | N=79 | Active, not recruiting | Sponsor: Jazz Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Epilepsy • Mental Retardation • Psychiatry

Unraveling Infantile Epileptic Spasms in the Context of Primary Mitochondrial Disease (P2-6.004). (PubMed, Neurology) - "Treatment strategies included ACTH, Vigabatrin, prednisone, Topamax, Clobazam, Epidiolex, Keppra and Ketogenic diet, leading to resolution of spasms in 8 patients (57%) within 1 to 24 months. Catenaccio has received research support from NIH. Amy Goldstein has received personal compensation in the range of $500-$4,999 for serving as a speaker with UMDF."

Journal • Cardiovascular • CNS Disorders • Epilepsy • Metabolic Disorders • Pediatrics • PDHA1

Trial of Cannabidiol to Treat Severe Behavior Problems in Children With Autism (clinicaltrials.gov) - P2 | N=42 | Active, not recruiting | Sponsor: University of California, San Diego | Phase classification: P2/3 ➔ P2 | Trial completion date: Dec 2023 ➔ Dec 2025 | Trial primary completion date: Jun 2023 ➔ Jun 2025

Phase classification • Trial completion date • Trial primary completion date • Autism Spectrum Disorder • Genetic Disorders

Optimization of seizure prevention by cannabidiol (CBD). (PubMed, Transl Neurosci) - "Our data suggest that route and CBD formulations affect its efficacy in the prevention of epileptic seizures. Inhaled broad spectrum CBD showed a potential superior effect compared to other delivery routes and CBD formulations in the prevention of epileptic seizures, which warrants further research."

Journal • CNS Disorders • Epilepsy

Antiepileptic activity and potential mechanism of full-spectrum hemp extract. (PubMed, Fundam Res) - "Although the approval of Epidiolex has ignited hope for patients, there is still a large gap in the field of anti-seizure research...Although the approval of Epidiolex has ignited hope for patients, there is still a large gap in the field of anti-seizure research...The results indicated that cannabichromene and cannabinol were involved in the anti-seizure process, especially CBC showed a strong allosteric enhancement effects on CBD binding site of the GABAA receptor, which implied that the GABAA receptor seemed to be the primary anti-epileptic target of HE. This article not only presents the great potential of HE as a candidate for new anti-epileptic drugs with less psychoactive, but also provides a valuable contribution to subsequent mechanism research and drug development on epilepsy."

Journal • CNS Disorders • Epilepsy • Mental Retardation • Psychiatry

Role of CBD in Regulating Meal Time Anxiety in Anorexia Nervosa (clinicaltrials.gov) - P1 | N=40 | Active, not recruiting | Sponsor: University of California, San Diego | Trial completion date: Jun 2025 ➔ Nov 2025 | Trial primary completion date: Jun 2025 ➔ Nov 2025

Trial completion date • Trial primary completion date • Anorexia • Mood Disorders • Psychiatry

Formulation and Evaluation of Solid Self-Nanoemulsifying Drug Delivery System of Cannabidiol for Enhanced Solubility and Bioavailability. (PubMed, Pharmaceutics) - "The pharmacokinetic data of Sprague Dawley rats indicated that a single oral dose of L-SNEDDS and spray drying SNEDDS led to a quicker absorption and a higher Cmax of CBD compared to the two oil-based controls (CBD-sesame oil (similar to Epidiolex®) and CBD-MCT), which is favorable for the application of CBD products. SNEDDS is a prospective strategy for enhancing the solubility and oral bioavailability of CBD, and solid SNEDDS offers flexibility for developing more CBD-loaded solid formulations. Moreover, SNEDDS provides new concepts and methods for other poorly water-soluble drugs."

Journal

Study of Cannabidiol in Sanfilippo Syndrome (clinicaltrials.gov) - P2/3 | N=35 | Not yet recruiting | Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | Initiation date: Jan 2025 ➔ Jun 2025 | Trial primary completion date: Jan 2026 ➔ Jun 2026

Trial initiation date • Trial primary completion date • Hunter Syndrome • Lysosomal Storage Diseases

Jazz Pharmaceuticals to Showcase Research Demonstrating Treatment Benefits of Xywav (calcium, magnesium, potassium, and sodium oxybates) Oral Solution and Epidiolex (cannabidiol) at American Academy of Neurology Annual Meeting (PRNewswire) - "Jazz Pharmaceuticals plc...today announced that seven abstracts from across its neuroscience portfolio will be featured at the 77th Annual American Academy of Neurology Meeting (AAN) being held April 5-9, 2025, in San Diego...Highlights at the 2025 AAN Annual Meeting include:Two presentations showcasing updated results from the open-label, single-arm, Phase 4 DUET trial of adults with narcolepsy or IH, which evaluated the effectiveness and safety of Xywav on key sleep outcomes, daytime symptoms, and functional impacts, including cognitive complaints, work productivity, and daily activities. Novel analysis of real-world Epidiolex (cannabidiol) treatment patterns from U.S. specialty pharmacy data found the overall probability of persistence at one year was nearly 70% (69.9%) among new patients and underscores the importance of dose optimization."

P4 data • Real-world • Epilepsy • Narcolepsy • Sleep Disorder

Mechanisms of Cannabidiol in Persons With MS: the Role of Sleep and Pain Phenotype (clinicaltrials.gov) - P2 | N=166 | Recruiting | Sponsor: Tiffany J. Braley, MD, MS | Trial completion date: Jun 2026 ➔ Jun 2027 | Trial primary completion date: Jun 2026 ➔ Jun 2027

Trial completion date • Trial primary completion date • CNS Disorders • Multiple Sclerosis • Pain

Real-world experience of cannabidiol in conjunction with clobazam for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome: Results from a retrospective multicentre chart review in Germany. (PubMed, Epilepsy Behav) - "In this chart review of patients with severe treatment refractory LGS or DS receiving adjunctive CBD and CLB concomitantly, a reduction in seizure frequency and sustained treatment retention was observed for up to 12 months across age groups in real-world clinical practice. CBD discontinuations exclusively due to AEs were infrequent and the AE profile was generally aligned to that previously observed."

Journal • Real-world evidence • Retrospective data • Anesthesia • CNS Disorders • Epilepsy • Pediatrics

Tuberous Sclerosis Complex (TSC)–Associated Neuropsychiatric Disorder (TAND) Outcomes Following Add-on Cannabidiol (CBD) Treatment: 3-Month Analysis of Open-Label Phase 3b/4 Trial EpiCom (AAN 2025) - P4 | "Objective:To investigate behavioral and co-occurring outcomes after initiation of add-on CBD treatment in patients with TSC who experience seizures (NCT05864846).Background:Epidiolex® is a plant-derived highly purified pharmaceutical formulation of CBD approved for treatment of seizures associated with TSC; anecdotal evidence suggests neuropsychiatric improvements...After initiating CBD, improvements were seen on TAND-SQ and ABC subscales and in severity of psychiatric and behavioral problems reported by caregivers and clinicians."

Clinical • P3 data • CNS Disorders • Epilepsy • Mental Retardation • Psychiatry

Unraveling Infantile Epileptic Spasms in the Context of Primary Mitochondrial Disease (AAN 2025) - "Treatment strategies included ACTH, Vigabatrin, prednisone, Topamax, Clobazam, Epidiolex, Keppra and Ketogenic diet, leading to resolution of spasms in 8 patients (57%) within 1 to 24 months. IESS can be the presenting seizure type in patients with PMD, highlighting the need for comprehensive metabolic and genetic evaluation in cryptogenic cases of IESS. While most patients with PMD have medically refractory spasms, some may achieve seizure freedom with monotherapy. Thus, ACTH or Vigabatrin are recommended as effective first-line treatments."

Cardiovascular • CNS Disorders • Epilepsy • Metabolic Disorders • PDHA1

Nurse-Reported Outcomes of Cannabidiol (CBD) Treatment in the Long-Term Care (LTC) Setting: Results From the BEhavior, COgnition, and More With Epidiolex® in the Long-Term Care Setting (BECOME-LTC) Survey (AAN 2025) - "The BECOME-LTC survey results suggest benefits of CBD treatment among LTC and group home residents with epilepsy."

CNS Disorders • Epilepsy

Caregiver-Reported Real-world Use of Cannabidiol (CBD) and Effects on Seizures and Caregiver Burden: Results From the CARE-EpiC Survey (AAN 2025) - "Objective:The Caregiver Analysis of Real-world Epidiolex® in Epilepsy Context (CARE-EpiC) study was conducted to understand real-world implications of CBD treatment from the caregiver's perspective.Background:Epidiolex®, a plant-derived highly purified pharmaceutical formulation of CBD, is approved for treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC); non-seizure benefits of CBD have also been reported...CBD initiation was associated with positive changes not only in dependents' well-being but also in caregiver experiences. Results showed that in caring for their dependents' physical, emotional, and behavioral needs, caregivers may require additional support that might not be captured in traditional burden of illness and cost-effectiveness analyses."

Clinical • Real-world • Real-world evidence • CNS Disorders • Epilepsy

Investigation of the Pharmacokinetic Profile of CBD (clinicaltrials.gov) - P1 | N=24 | Completed | Sponsor: Icahn School of Medicine at Mount Sinai | Active, not recruiting ➔ Completed

Trial completion

Pharmacokinetic variability and use of therapeutic drug monitoring of cannabidiol in patients with refractory epilepsy. (PubMed, Epilepsia) - "This observational study with TDM data revealed extensive pharmacokinetic variability of CBD in patients with refractory epilepsy. The results demonstrate the need for close follow-up and use of TDM, including biochemical markers of toxicity, for individualized treatment with CBD."

Journal • PK/PD data • CNS Disorders • Epilepsy

CBD for Lower Urinary Tract Dysfunction in Spinal Cord Injury (clinicaltrials.gov) - P2 | N=20 | Not yet recruiting | Sponsor: University of Wisconsin, Madison

New P2 trial • CNS Disorders • Orthopedics • Urinary Incontinence • Urology