Epidiolex (cannabidiol) / Jazz - LARVOL DELTA

Retrospective Multicenter Chart Review Study of Adjunctive Cannabidiol for Seizures Associated with Lennox-Gastaut Syndrome, Dravet Syndrome and Tuberous Sclerosis Complex. (PubMed, Neurol Ther) - "In patients with LGS, DS, or TSC-associated epilepsy, adjunctive CBD was associated with a reduction in seizure frequency across age groups. CBD demonstrated tolerability consistent with its known profile, and 67% of patients remained on treatment at 12 months."

Journal • Retrospective data • Anesthesia • CNS Disorders • Epilepsy

Phytocannabinoids for Reducing Chronic Chemotherapy-Induced Peripheral Neuropathy in Breast and Colon Cancer Survivors (clinicaltrials.gov) - P2 | N=120 | Recruiting | Sponsor: City of Hope Medical Center | Not yet recruiting ➔ Recruiting | Trial completion date: Oct 2026 ➔ Jan 2028 | Trial primary completion date: Oct 2026 ➔ Jan 2028

Enrollment open • Trial completion date • Trial primary completion date • Breast Cancer • Colon Cancer • Colorectal Cancer • Oncology • Pain • Solid Tumor

A 1:1 combination of cannabidiol and Δ9-tetrahydrocannabinol inhibit toll-like receptor 7- and 8-mediated inflammation in human immune cells. (PubMed, Eur J Pharmacol) - "In this study we focused on two clinically relevant plant-derived (phyto) cannabinoids, cannabidiol (CBD) and Δ9-tetrahydrocannabinol (THC), given that cannabinoid-based therapeutics containing these compounds are currently available in the form of sativex® (nabiximols) and epidiolex®. Data presented herein indicate that CBD and THC, particularly when delivered in a 1:1 combination, can act as TLR7/8 immunomodulatory drugs to dampen inflammation in macrophages and PBMCs. This study provides evidence that phytocannabinoids target TLR7/8-induced viral signalling on endosomal compartments to control inflammation in immune cells."

Journal • Immunology • Infectious Disease • Inflammation • CXCL10 • IRF7 • TLR7 • TLR8 • TNFA

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

Enrollment open • CNS Disorders • Orthopedics • Urinary Incontinence • Urology

Epidiolex® for Anxiety in Pediatric Epilepsy (clinicaltrials.gov) - P4 | N=20 | Completed | Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | Recruiting ➔ Completed | Trial completion date: Apr 2026 ➔ May 2025

Trial completion • Trial completion date • CNS Disorders • Epilepsy • Mood Disorders • Pediatrics • Psychiatry

Differential Effects of THC and CBD on Brain Network Timing (OHBM 2025) - " 39 healthy adults (48.7% female, mean age 29.28±7.13) were given 10mg THC (Syndros), 600mg CBD (Epidiolex), both THC and CBD, or placebo across four study visits. These findings suggest that the mechanism of action of THC on brain network function may be through alteration of the timing and stability of brain connectivity. Such a mechanism is consistent with slower cognitive and sensorimotor response times. Investigation of how THC alters the timing of synchronous brain connections may facilitate a unified explanation of perceptual and cognitive alterations of brain network function induced by THC, with greater brain network stability and impaired set shifting and traversal of distinct cognitive states."

CNS Disorders • Cognitive Disorders

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

Enrollment open • Genetic Disorders • Hematological Disorders • Inflammation • Pain • Sickle Cell Disease

A Randomised Controlled Trial of Cannabidiol (CBD) for the treatment of Cannabis Use Disorder (CUD) (ANZCTR) - P3 | N=250 | Active, not recruiting | Sponsor: University of Sydney | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Psychiatry • Substance Abuse

Is highly purified cannabidiol a treatment opportunity for drug-resistant epilepsy in subjects with typical Rett syndrome and CDKL5 deficiency disorder? (PubMed, Epilepsia Open) - "This study explored the use of highly purified Cannabidiol (CBD, Epidiolex®) as an add-on therapy for individuals with drug-resistant epilepsy due to Rett Syndrome (RTT) or CDKL5 Deficiency Disorder (CDD). Twenty-seven participants received CBD alongside their usual ASMs. After a median treatment duration of 14 months, 66.6% experienced fewer seizures, with some showing over 75% reduction. Side effects were generally mild, mainly sleepiness or irritability. Notably, caregivers reported improvements in attention, responsiveness, sleep, and motor function. While results were not statistically significant, they suggest CBD may benefit seizure control and quality of life in RTT and CDD patients."

Journal • Anorexia • CNS Disorders • Developmental Disorders • Epilepsy • Genetic Disorders • Insomnia • Movement Disorders • Sleep Disorder • CDKL5

Oral formulations for cannabidiol: Improved absolute oral bioavailability of biodegradable cannabidiol self-emulsifying drug delivery systems. (PubMed, Colloids Surf B Biointerfaces) - "These findings underscore the potential of SEDDS as an effective oral delivery system for CBD, capable of achieving higher CBD plasma concentrations than Epidiolex. Additionally, the biodegradable PG-based SEDDS demonstrated improved absolute bioavailability compared to Epidiolex, emphasizing the importance of formulation design in optimizing oral drug delivery systems."

Journal

CAN-SAD: Investigating the Effects of Cannabidiol on Social Anxiety Disorder (clinicaltrials.gov) - P4 | N=50 | Recruiting | Sponsor: Massachusetts Institute of Technology | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Mood Disorders • Psychiatry • Social Anxiety Disorder

Protocol for EpiCom: A phase 3b/4 study of behavioral outcomes following adjunctive cannabidiol for the management of tuberous sclerosis complex-associated neuropsychiatric disorders (TAND). (PubMed, PLoS One) - P4 | "A plant-derived highly purified pharmaceutical formulation of cannabidiol (CBD; Epidiolex®/Epidyolex® oral solution) is approved for seizures associated with TSC...EpiCom will assess the changes in behavioral and other outcomes associated with TAND and seizure outcomes, including overall symptom severity and treatment retention, following adjunctive CBD treatment in individuals with TSC-associated seizures. The results will inform future studies evaluating pharmacotherapy for behavioral outcomes in similar populations."

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

Efficacy of Epidiolex in Patients With Electrical Status Epilepticus of Sleep (ESES). (clinicaltrials.gov) - P1 | N=16 | Completed | Sponsor: Northwell Health | Recruiting ➔ Completed | N=34 ➔ 16

Enrollment change • Trial completion • Epilepsy

CREWS: Effects of Cannabidiol on Stress and Nicotine Withdrawal (clinicaltrials.gov) - P1 | N=90 | Not yet recruiting | Sponsor: Johns Hopkins University

New P1 trial • Nicotine Addiction • Tobacco Addiction

An open-label phase I comparator-controlled clinical trial to assess tolerability and pharmacokinetics of IHL-675 A a fixed dose combination of cannabidiol plus hydroxychloroquine in healthy volunteers. (PubMed, Sci Rep) - P1 | "Plasma concentrations of CBD, HCQ and their major metabolites, 7-OH-CBD, and 7-COOH-CBD, and desethylhydroxychloroquine (DHCQ), desethylchloroquine (DCQ), and bisdesethylhydroxy-chloroquine (BDCQ) were assessed at predefined timepoints across the monitoring period and PK parameters were determined and compared between treatments using noncompartmental methods and analysis of variance (ANOVA) of log-transformed values exposure PK parameters...Compared to the reference listed drugs for CBD (Epidiolex 150 mg) and HCQ (Plaquenil 200 mg), there was a slightly increased exposure to CBD and its metabolites for IHL-675 A, and slightly decreased exposure to HCQ. These results support the continued clinical development of IHL-675 A.Trial Registration: ACTRN12622000289718."

Clinical • Journal • P1 data • PK/PD data • Immunology • Inflammatory Arthritis • Rheumatoid Arthritis • Rheumatology

The Regulatory Environment Surrounding Cannabis Medicines in the EU, the USA, and Australia. (PubMed, Pharmaceutics) - " Only Epidyolex™ has been approved by these three agencies...There are few regulator-approved cannabis-based medicines available and, evidence supporting the therapeutic use of consumer products is very limited. A complex regulatory and legislative scenario hinders research on and development of cannabis-based medicines, leaving a market gap filled with unregulated products that are potentially misleading regarding their therapeutic claims."

Journal

Cannabidiol (CBD) Treatment for Severe Problem Behaviors in Autistic Boys: A Randomized Clinical Trial. (PubMed, J Autism Dev Disord) - P2 | "We conducted a double-blind, placebo-controlled crossover study of plant-derived CBD (Epidiolex®) to determine safety, tolerability, and behavior effects in autistic boys...A prominent placebo effect demonstrates the importance of placebo control in treatment studies. Medications taken for behavior may reduce blood levels of CBD and may affect outcome measures.Trial Registration https://clinicaltrials.gov NCT04517799 start date 6/1/2020."

Clinical • Journal • Autism Spectrum Disorder • Genetic Disorders

Caregiver-reported non-seizure and seizure outcomes with cannabidiol and clobazam in patients aged ≥2 years with Lennox-Gastaut syndrome or Dravet syndrome: A subgroup analysis of the BECOME survey. (PubMed, Seizure) - "Consistent with the overall BECOME survey, most caregivers of patients with LGS/DS aged ≥2 years taking concomitant clobazam reported improvements in seizure and non-seizure outcomes since initiating CBD treatment."

Journal • CNS Disorders • Epilepsy

Ananda Pharma on phase I trial progress - ICYMI (Proactiveinvestors) - "Ananda Pharma Plc...and CEO Melissa Sturgess talked with Proactive about the company’s progress towards beginning its first clinical trial in Australia for its cannabinoid-based drug candidate...The trial will compare the pharmacokinetic profile of Ananda’s candidate with Epidiolex, the only CBD drug currently approved by regulators....The Australian Therapeutic Goods Administration has acknowledged Ananda’s first trial."

New P1 trial • CNS Disorders

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