Full‑spectrum cannabis extract for refractory epilepsy in tuberous sclerosis
Treatment With Full-spectrum Cannabis Extract of Refractory Epilepsy Associated With Tuberous Sclerosis Complex (TSC)
This trial tests whether a full‑spectrum cannabis extract (YCJ-01) can reduce seizures in people with tuberous sclerosis complex who have hard‑to‑control epilepsy.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 84 (estimated) |
| Ages | 2 Years to 65 Years |
| Sex | All |
| Sponsor | Oils4Cure Industry-sponsored |
| Locations | 1 site (Madrid) |
| Trial ID | NCT07403266 on ClinicalTrials.gov |
What this trial studies
This Phase 3 interventional trial compares treatment with YCJ-01 to placebo in patients with refractory epilepsy secondary to tuberous sclerosis complex. Participants are assigned to receive YCJ-01 or placebo and seizure counts during treatment will be compared with a baseline period, with a primary focus on change in monthly seizures in the second month of treatment. Safety is monitored through adverse events, serious adverse events, events leading to withdrawal, vital signs, and laboratory tests. Response rate is measured as the proportion achieving at least a 50% reduction in seizures.
Who should consider this trial
Good fit: People aged 2 to 65 with a clinical or genetic diagnosis of tuberous sclerosis complex who have refractory epilepsy despite standard treatments and who had at least four observable seizures in the four weeks before starting treatment are the intended participants.
Not a fit: Patients whose seizures are well controlled, who have fewer than four observable seizures in the baseline period, or whose seizures are due to causes other than TSC are unlikely to benefit from this intervention.
Why it matters
Potential benefit: If successful, YCJ-01 could reduce seizure frequency and improve seizure control for people with refractory TSC‑related epilepsy.
How similar studies have performed: Cannabidiol formulations have shown seizure reductions in TSC, but full‑spectrum cannabis extracts like YCJ-01 are less well studied and represent a related but not identical approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria:Patients of any sex between 2 and 65 years of age (both included) Diagnosis confirmed by the TSC investigator (by clinical criteria and/or genetic study). Refractory epilepsy secondary to TSC, defined as that not responding successfully to traditional AEDs (2 or more), ketogenic diet, vagal nerve stimulator, and/or whose patients are not candidate for epilepsy surgery or persist with seizures after surgery. Patients with a minimum of 4 epileptic seizures or more within 4 weeks prior to the initiation of the assigned treatment in the trial, with observable external signs (loss of consciousness or motor component). Stability in AEDs doses, and in ketogenic diet/programming of the device associated with the vagal nerve stimulator with no changes for at least 4 weeks prior to the initiation of the assigned treatment. Patients who are in treatment with 3 or less AEDs at the time of signing the informed consent. For the purposes of assessing eligibility, Clobazam will not be counted as AED Willingness by patients or caregivers/family members (in case of patients who are minors or under legal guardianship) to complete the seizure diary. In case of women of child-bearing age, for safety, those who agree to follow the required contraceptive measures from the signing of the informed consent until three months after stop the intake of the investigational medicinal product. Patients who have signed the informed consent either by themselves or through a legal representative. \- Exclusion Criteria:Patients who are receiving treatment on corticoids at the time of signing the informed consent. Pregnancy. Breastfeeding women To have participated in another clinical trial, unless at least 5 half- lives of the investigational product have elapsed, or 12 weeks, if it is a product with cannabis oil. Patients who have received products with cannabis oil in the last 12 weeks. Patients who did not correctly follow the AED treatment in the 4 weeks prior to the intervention assigned in the trial. Patients who changed medication or AED dose, ketogenic diet, or the vagal stimulator during the 4 weeks prior to the initiation of the intervention assigned in the trial. Cardiac, renal, and hepatic failure, pancreatic insufficiency, or hematologic dysfunction with values above the normal limits of creatinine and urea; values 2 times the normal limits of transaminases, lipases, and serum amylase; platelets \< 80000/mm3, and white blood cell count \<3000/mm3. Uncontrolled severe medical condition such as: hepatic disease, increased bilirubin levels more than twice the normal limit, cirrhosis, chronic hepatitis (hepatitis B or C), uncontrolled diabetes (defined as blood glucose \>150 mg%), (chronic or acute) active infections or uncontrolled severe infections, or active bleeding. Patients or family/caregivers (in case of patients who are minors or under legal guardianship) who does not agree to comply with the requirements and trial visits or present a high risk of non-compliance with the protocol, according to the treating physician. Allergy to any of the components of the investigational medicinal product/placebo. Family (considering only first-degree blood relatives) or personal history of schizophrenia. Personal history of suicide attempts. \-
Where this trial is running
Madrid
- Hospital Ruber Internacional — Madrid, Spain (Recruiting)
Study contacts
- Study coordinator: Gil Nagel, Antonio Gil Nagel, Antonio, Doctor
- Email: ensayosepi@neurologiaclinica.es
- Phone: +34 913875000
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.