EpilepsyPhase 2CBD_RENCT05803434
Cannabidiol in Children and Young Adults With Rare Disease-associated Severe Epilepsy
A Phase 2 study testing Cannabidiol oral solution in people with Epilepsy and Rare Diseases, run by Meyer Children's Hospital IRCCS at 1 site in Italy.
Trial summary
This is a pilot, open-label, phase II study. The main objective of the study is to demonstrate that Cannabidiol (CBD), used in addition to current anti-seizure medications (ASMs) reduces the number and/or severity of motor (generalized, focal, or both) seizures in children and young adults with rare disease-associated… Read more about this study
- Who is it for?
- Ages 2 to 25 years
- Conditions the study is for
- Epilepsy, Rare Diseases
- What is being tested?
- Cannabidiol oral solution
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteItaly
- Study dates
- 14 Mar 2024to 1 Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or female;
- Children (age 2-18 years) and young adults (18-25 years), as of the day of the Screening Visit;
- Subject with rare disease-associated severe epilepsy. Subject has been certified by the National Health System as affected by a rare disease listed in https://www.malattierare.gov.it
- Patient has severe epilepsy, with at least 4 motor (generalized, focal, or both) seizures per month during baseline period, despite 2 or more current or prior ASMs;
- Previous treatment with at least 2 ASMs;
- Currently taking at least 1 other ASMs or between one and four ASMs, with a stable antiseizure treatment for the previous 4 weeks (including ketogenic diet and vagal nerve stimulation);
- Subject's parent/caregiver has been informed of the nature of the study and informed consent has been obtained from the legally responsible parent/guardian;
- Subject's parent/caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability in the opinion of the investigator
Who cannot join
- Age <2 years;
- Known hypersensitivity to CBD or any of the excipients in the study formulation;
- Progressive neurological disease;
- Clinically significant unstable medical conditions other than epilepsy that may place patient's safety at risk;
- Any other significant disease or disorder which, in the opinion of the investigator, may either put the patient at risk because of participation in the study, may influence the result of the study, or affect the patient's ability to participate in the study;
- Impaired hepatic function at screening defined as any of the following: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3 times the upper limit of normal (ULN) and total bilirubin (TBL) greater than 2 times the ULN;
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- Subject taking more than four concurrent ASMs;
- Subject has taken corticotropins in the six months prior to screening;
- Subjects taking felbamate, and they have been taking it for less than one year prior to screening;
- Inadequate supervision by parents and/or caregivers as judged by the investigator;
- Subject has been part of a clinical trial involving another investigational medicinal product in the previous six months;
- Current or past use of recreational or medicinal cannabis, or cannabinoid-based medications, within the three months prior to screening and is unwilling to abstain for the duration for the study;
- Female patients who are pregnant;
- Female patients of childbearing potential or male patient whose partner is of childbearing potential, unless willing to ensure that they or their partner use a highly effective method of birth control during the study and for three months thereafter.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Open-label Pilot Study to Assess the Efficacy and Safety of Cannabidiol Oral Solution as an Adjunctive Treatment for Children and Young Adults With Rare Disease-associated Severe Epilepsy
This is a pilot, open-label, phase II study. The main objective of the study is to demonstrate that Cannabidiol (CBD), used in addition to current anti-seizure medications (ASMs) reduces the number and/or severity of motor (generalized, focal, or both) seizures in children and young adults with rare disease-associated severe epilepsy.
Secondary objectives include assessment of safety and tolerability, changes in behaviour, cognition and sleep, pharmacokinetic interaction with concurrent ASMs.
- Conditions
- Epilepsy, Rare Diseases
- Being tested
- Cannabidiol oral solution
- Keywords
- drug resistant epilepsy, rare epilepsy, cannabidiol
- Medical subject headings
- Epilepsy, Rare Diseases, Drug Resistant Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cannabidiol oral solutionDrug
Cannabidiol will be administered orally twice daily into equally divided doses. The starting dose is 2.5 mg/kg twice daily. The dose can be gradually increased to 5 mg/kg twice daily, which is the recommended maintenance dose, up to a maximum dose of 10 mg/kg twice daily, according to tolerability and clinical response.
Following titration, subjects will continue treatment over a 20-week maintenance period. The total treatment duration from the beginning of the titration period till the end of the maintenance period will be 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Cannabidiol treatment | Experimental | Not described Receives: Drug: Cannabidiol oral solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in number of generalized and/or focal motor-onset seizure frequency
percentage change per 28 days from the 4-week baseline period in generalized and/or focal motor-onset seizure frequency during the 24-week treatment period
Measured: 24 weeks
Change in severity of generalized and/or focal motor-onset seizure frequency
a score will be established for each patient, based on review and comparison of all baseline-EEG/7-weeks control-EEG and baseline-EEG/15-weeks control-EEG, with values ranging from 0 (= worsened EEG), to a maximum of 2 (= improved); 1 will be assigned if the EEG trace is unmodified
Measured: 24 weeks
Secondary outcomes (10)
Incidence of adverse events
Adverse events reporting according to Common Terminology Criteria for Adverse Events (CTCAE) from 1 (mild) to 5 (death)
Measured: 24 weeks
Body weight
Measurement of body weight for tolerability monitoring
Measured: 24 weeks
Maximum Plasma Concentraion [Cmax] of concurrent ASMs
blood levels of concurrent ASMs will be taken at baseline and every 4 weeks
Measured: 24 weeks
Number of subjects considered treatment responders
Number of subjects with a ≥25%, ≥50% ≥75% reduction in motor (generalized, focal, or both) seizures from baseline
Measured: 24 weeks
Number of subjects who are free of motor (generalized, focal, or both) seizures
Number of subjects who are free of motor (generalized, focal, or both) seizures
Measured: 24 weeks
Longest period of seizure freedom
Longest period of seizure freedom
Measured: 24 weeks
Number of patients experiencing a >25% worsening, -25 to +25% no change, 25-50% improvement, 50-75% improvement or >75% improvement in total seizures from baseline
Number of patients experiencing a >25% worsening, -25 to +25% no change, 25-50% improvement, 50-75% improvement or >75% improvement in total seizures from baseline
Measured: 24 weeks
Changes from baseline in number of inpatient hospitalizations due to epilepsy
Changes from baseline in number of inpatient hospitalizations due to epilepsy
Measured: 24 weeks
Change in severity of seizures will be assessed using a pediatric adaptation of the Chalfont Seizure Severity Scale
Change in severity of seizures will be assessed using a pediatric adaptation of the Chalfont Seizure Severity Scale (from 1 minimum severity to >100 max severity)
Measured: 24 weeks
Change from baseline to 6-months after treatment initiation in number of seizure-free days
Change from baseline to 6-months after treatment initiation in number of seizure-free days
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Apr 2023
- Study start14 Mar 2024
- Primary completion1 Apr 2026
- Study completion1 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for EpilepsyStudies of the same condition, open and under way.
- More trials run by Meyer Children's Hospital IRCCSThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Meyer Children's Hospital IRCCS
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05803434
- Sponsor's protocol ID
- CBD_RE
- Acronym
- CBD_RE
- Status last verified
- Oct 2026
- First submitted
- 28 Feb 2023
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label Pilot Study to Assess the Efficacy and Safety of Cannabidiol Oral Solution as an Adjunctive Treatment for Children and Young Adults With Rare Disease-associated Severe Epilepsy
- ClinicalTrials.gov ID
- NCT05803434
- Acronym / sponsor ID
- CBD_RE / CBD_RE
- Conditions
- Epilepsy, Rare Diseases
- Keywords
- drug resistant epilepsy, rare epilepsy, cannabidiol
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Meyer Children's Hospital IRCCSUniversity, hospital or other
- Study start
- 14 Mar 2024 (actual)
- Primary completion
- 1 Apr 2026 (actual)
- Study completion
- 1 Dec 2026 (estimated)
- First posted
- 7 Apr 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a pilot, open-label, phase II study. The main objective of the study is to demonstrate that Cannabidiol (CBD), used in addition to current anti-seizure medications (ASMs) reduces the number and/or severity of motor (generalized, focal, or both) seizures in children and young adults with rare disease-associated severe epilepsy.
Secondary objectives include assessment of safety and tolerability, changes in behaviour, cognition and sleep, pharmacokinetic interaction with concurrent ASMs.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cannabidiol treatment | Experimental | Drug: Cannabidiol oral solution |
Who may qualify
- Ages
- 2 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Male or female;
- Children (age 2-18 years) and young adults (18-25 years), as of the day of the Screening Visit;
- Subject with rare disease-associated severe epilepsy. Subject has been certified by the National Health System as affected by a rare disease listed in https://www.malattierare.gov.it
- Patient has severe epilepsy, with at least 4 motor (generalized, focal, or both) seizures per month during baseline period, despite 2 or more current or prior ASMs;
- Previous treatment with at least 2 ASMs;
Key exclusion criteria (14 listed)
- Age <2 years;
- Known hypersensitivity to CBD or any of the excipients in the study formulation;
- Progressive neurological disease;
- Clinically significant unstable medical conditions other than epilepsy that may place patient's safety at risk;
- Any other significant disease or disorder which, in the opinion of the investigator, may either put the patient at risk because of participation in the study, may influence the result of the study, or affect the patient's ability to participate in the study;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cannabidiol oral solutionCannabidiol will be administered orally twice daily into equally divided doses. The starting dose is 2.5 mg/kg twice daily. The dose can be gradually increased to 5 mg/kg twice daily, which is the recommended maintenance dose, up to a maximum dose of 10 mg/kg twice daily, according to tolerability and clinical response. Following titration, subjects will continue treatment over a 20-week maintenance period. The total treatment duration from the beginning of the titration period till the end of the maintenance period will be 24 weeks. | Drug | Cannabidiol treatment |
Primary outcome
Change in number of generalized and/or focal motor-onset seizure frequency
Time frame: 24 weeks
percentage change per 28 days from the 4-week baseline period in generalized and/or focal motor-onset seizure frequency during the 24-week treatment period
Change in severity of generalized and/or focal motor-onset seizure frequency
Time frame: 24 weeks
a score will be established for each patient, based on review and comparison of all baseline-EEG/7-weeks control-EEG and baseline-EEG/15-weeks control-EEG, with values ranging from 0 (= worsened EEG), to a maximum of 2 (= improved); 1 will be assigned if the EEG trace is unmodified
10 secondary outcomes
Incidence of adverse events
Time frame: 24 weeks
Body weight
Time frame: 24 weeks
Maximum Plasma Concentraion [Cmax] of concurrent ASMs
Time frame: 24 weeks
Number of subjects considered treatment responders
Time frame: 24 weeks
Number of subjects who are free of motor (generalized, focal, or both) seizures
Time frame: 24 weeks
Longest period of seizure freedom
Time frame: 24 weeks
Number of patients experiencing a >25% worsening, -25 to +25% no change, 25-50% improvement, 50-75% improvement or >75% improvement in total seizures from baseline
Time frame: 24 weeks
Changes from baseline in number of inpatient hospitalizations due to epilepsy
Time frame: 24 weeks
Change in severity of seizures will be assessed using a pediatric adaptation of the Chalfont Seizure Severity Scale
Time frame: 24 weeks
Change from baseline to 6-months after treatment initiation in number of seizure-free days
Time frame: 24 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cannabidiol oral solution in people with Epilepsy and Rare Diseases, run by Meyer Children's Hospital IRCCS at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 9 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in number of generalized and/or focal motor-onset seizure frequency (time frame: 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2026.