Resistant Epilepsy, DrugPhase 2NCT04406948

Study of Safety and Efficacy of MGCND00EP1 as an Add on Treatment in Children and Adolescents With Resistant Epilepsies

A Phase 2 study testing MGCND00EP1, ECG and EEG in people with Resistant Epilepsy, Drug and Adolescent Epilepsy, run by MGC Pharmaceuticals d.o.o at 2 sites in 2 countries.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record

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Trial summary

EudraCT: 2018-003887-29 Objective:To evaluate the safety and efficacy of: MGCND00EP1 from MGC PHARMACEUTICALS d.o.o. Study Design: Randomized, double blind, placebo controlled parallel grouped study Sample Size: 103 subjects Study Population: Children from 1 year to 18 years of age Comparator Product :Placebo… Read more about this study

Who is it for?: Ages 1 year to 18 years
Conditions the study is for: Resistant Epilepsy, Drug, Adolescent Epilepsy, Children Epilepsy
What is being tested?: MGCND00EP1, ECG and EEG
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 0 participants
Where: 2 sitesin 2 countries
Study dates: 30 May 2024to 30 May 2024
Phase: Phase 2
Are there results?: None posted in the registry

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 1 year to 18 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Patient has documented clinically confirmed diagnosis of epilepsy;
Patient did not respond to at least 2 AED's therapy given in adequate doses;
Patients current therapy is considered inadequate (not completely controlled by AEDs); patients had four or more countable seizures with a motor component per 4 week period;
Patient is aged 1 year - 18 years inclusive at screening age;
Patient took one or more AEDs treatment at dose which has been stable for at least 4 weeks before enrolment;
Females of childbearing potential can only participate in the study if willing to use acceptable, effective methods of contraception during the trial and for three month after end of trial participation as defined in point 7.10 of this protocol;

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Patient/parent is able to read/understand informed consent.
Male patients must either be surgically sterile or he and his female spouse/partner who is of childbearing potential must be willing to use highly effective methods of contraception consisting of 2 forms of birth control (1 of which must be a barrier method) starting at screening and continuing throughout the study.
All medications or interventions for epilepsy (including ketogenic diet and vagus nerve stimulation (VNS) were stable for four weeks prior to screening and participants were willing to maintain a stable regimen throughout the study. The ketogenic diet and VNS treatments are not counted as an AED.

Who cannot join

Known history or presence of clinically significant unstable medical condition other that epilepsy which, in the opinion of the Investigator, would jeopardize the safety of the subject or impact the validity of the study results.
Known history or presence of serious cardiovascular disease
Known or suspected history or family history of: schizophrenia, or other psychotic illness, severe personality disorder or other significant psychiatric disorder.
Known or suspected allergy hypersensitivity or idiosyncratic reaction to cannabinoids or any other drug substances with similar activity or to any of the excipients of the IMP.
Participant has clinically relevant abnormalities in the 12-lead electrocardiogram measured at screening or randomisation.
Patients were currently using or had in the past used recreational or medicinal cannabis or synthetic CBD based medications or preparations within last 3 months or had previous or current treatment with cannabis-based therapy within last 3 months.

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History of drug or alcohol addiction requiring treatment.
History of malabsorption within the last year or presence of clinically significant gastrointestinal disease or surgery that may affect drug bioavailability, including but not limited to cholecystectomy.
Presence of hepatic or renal dysfunction.
Females who: are pregnant (serum hCG level consistent with pregnancy diagnosis); or are lactating;
Participation in a clinical trial that involved administration of an investigational medicinal product within 90 days prior to drug administration, or recent participation in a clinical investigation that, in the opinion of the Investigator, would jeopardize subject safety or the integrity of the study results;
Participant has clinically significant abnormal laboratory values (e.g. liver enzymes);
Participant has clinically significant findings from a physical examination (fever);
In case of ketogenic diet or VNS; the diet need to be stable for at least 4 weeks, and VNS ramping needs to be stable at least 12 weeks before enrolment.

Trial locations and contacts

2 sites in 2 countries.

Country

City, postcode or hospital

2 sites in 2 countries

Israel1 site

Schneider Children's Medical Center of Israel
Petach Tikvah, Central District, 4920235

Other trials in Israel

Slovenia1 site

University Children's Hospital Ljubljana University Medical Centre Ljubljana
Ljubljana, 1000

Other trials in Slovenia

About this study

Official title: Randomized, Double Blind, Placebo Controlled, Parallel Design Phase II b Study of Safety and Efficacy of MGCND00EP1 as an Add on Treatment in Children and Adolescents With Resistant Epilepsies

EudraCT: 2018-003887-29

Objective:To evaluate the safety and efficacy of: MGCND00EP1 from MGC PHARMACEUTICALS d.o.o.

Study Design: Randomized, double blind, placebo controlled parallel grouped study Sample Size: 103 subjects Study Population: Children from 1 year to 18 years of age Comparator Product :Placebo solution, oral IMP Product : MGCND00EP1 (each ml of solution containing 100 mg of cannabidiol and 5 mg of (-)-trans-Δ9- tetrahydrocannabinol as active substance) from MGC PHARMACEUTICALS D.O.O.

According to dosing scheme up to 25 mg/kg BW per day or maximum daily dose 800 mg (whichever smaller) for 6 weeks titration and 6 weeks of treatment, oral administration

Read the detailed description

Subjects on regular therapy with anti-epileptic medications who have evidence from clinical monitoring that current therapy is insufficient, following failure of at least two AEDs for at least during the last 2 months will be enrolled into this study. Upon subjects/parents have consented to participation in the study and after baseline examinations, subjects will continue with current antiepileptic treatment, as clinically needed, for another 28 days at the same dose as before entering the study; drug accountability will be performed for verification of treatment compliance, and diary will be used to record data on epileptic seizures. After that, participating patients will be randomly assigned to MGCND00EP1 or placebo and take it as add on to previous treatment for 6 weeks as titration period and 6 weeks at maintenance dose, and then titrated down during next two weeks. Patients will continue previous anti- epileptic treatment throughout all the periods of the study.

Day one - Screening and Enrollment Visit :

Total 103 patients: Obtain informed consent from legal guardian. Screen potential subjects by inclusion and exclusion criteria. Visit 1: obtain medical and medication history, vital signs, physical and neurological exam, blood and urine tests, ECG, EEG, concomitant medications, questionnaires.

Weeks 1-4 - AED Stabilization Period :

Visit 2: vital signs, weight, physical and neurological exam, blood and urine tests, concomitant medications, questionnaires, monitor AEs, PK blood collection (subset of patients), randomize and dispense study drugs

Patients will be randomized and will either get placebo or MGCND00EP1 (3:1 active:placebo)

Weeks 5-10 - Dose titration period:

Dose escalations (2 mg/kg body weight/day increments), as required, up to 25 mg/kg/day or 800 mg/day, the lower of the two, until stable dose is reached.

Visit 3: vital signs, weight, physical and neurological exam, blood and urine tests, concomitant medications, questionnaires, collect and issue diaries, dispense study drug, monitor AEs

Weeks 11-16 - Maintenance Period :

Visit 4: vital signs,weight, physical and neurological exam, blood and urine tests, concomitant medications, questionnaires, PK sample collection (subset of patients), collect and issue diaries, dispense study drug, EEG, monitor AEs

Weeks 17-18 - Tapering-off and Follow-up Period:

Weekly phone call to determine taper dose at physician's discretion

Visit 5: vital signs,weight, physical and neurological exam, blood and urine tests, concomitant medications, questionnaires, monitor AEs, collect diaries and unused drug

Weeks 19-20 - Follow-up Period:

Weekly phone calls

Visits 6: vital signs,weight, physical and neurological exam, concomitant medications, monitor AEs, collect diaries .

Conditions: Resistant Epilepsy, Drug, Adolescent Epilepsy, Children Epilepsy, Children and Adolescents With Resistant Epilepsies
Being tested: MGCND00EP1, Placebo, ECG, EEG, Blood and urine collection
Keywords: epilepsy, resistant epilepsy, adolescents, children
Medical subject headings: Drug Resistant Epilepsy, Epilepsy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

MGCND00EP1Drug
Also called Cannabis oil
Patients will take cannabis oil during the study
PlaceboDrug
Patient will take carrier oil during the study
ECGDiagnostic test
A standard 12-lead ECG will be recorded using digital ECG recording equipment provided to the investigational site. The ECG has to be performed prior to laboratory samplings at time points indicated in the Schedule of Assessments.
The ECG recording will be reviewed by investigator and case of need consultation with cardiologist will be performed. The investigator has the final decision on the clinical significance of the ECG results.
EEGDiagnostic test
An EEG is an electrophysiological monitoring method that records the electrical activity and measures voltage fluctuations resulting from ionic current within the neurons of the brain. In clinical contexts, EEG refers to the recording of the brain's spontaneous electrical activity over a period of time.
Blood and urine collectionDiagnostic test
Also called blood tests
safety blood tests - hematology\blood count and biochemistry standard blood tests urinalysis - urine test analysis

Study groups in NCT04406948
Study group	Role	What they receive
MGCND00EP1	Experimental	Participants who will assigned to receive add on MGCND00EP1 will receive carrier oil containing THC and CBD in ratio 20:1, (10% of cannabidiol and 0.5 % and (-)-trans-Δ9-tetrahydrocannabinol) . Titration Dose: 1 to 2 mg/kg body weight/day. dose will be increased every week by 2 mg/kg body weight/day up to a maximum 25 mg/kg body weigh/day or maximum daily dose 800 mg (the smaller of those 2 values) (divided into two daily doses). After titration, patients will be receiving a stable maintenance dose of IMP (up to 25 mg/kg BW per day or maximum daily dose 800 mg (whichever smaller)) for 6 weeks period. During forth treatment period participants will commence a 2 weeks down-titration taper period, followed by 4 weeks observational follow up period of previous standard AE treatment without IMP. Receives: Drug: MGCND00EP1; Diagnostic Test: ECG; Diagnostic Test: EEG; Diagnostic Test: Blood and urine collection
PLACEBO	Placebo comparator	Participants who are assigned to receive add on PLACEBO will be administered the carrier oil (without the active ingredients). Titration Dose: 1 to 2 mg/kg body weight/day. dose will be increased every week by 2 mg/kg body weight/day up to a maximum 25 mg/kg body weigh/day or maximum daily dose 800 mg (the smaller of those 2 values) (divided into two daily doses). After titration, patients will be receiving a stable maintenance dose of IMP (up to 25 mg/kg BW per day or maximum daily dose 800 mg (whichever smaller)) for 6 weeks period. During forth treatment period participants will commence a 2 weeks down-titration taper period, followed by 4 weeks observational follow up period of previous standard AE treatment without IMP. Receives: Drug: Placebo; Diagnostic Test: ECG; Diagnostic Test: EEG; Diagnostic Test: Blood and urine collection

What is being measured

The primary outcome is the main question the study is designed to answer.

The proportion of patients showing a >50% reduction in frequency of seizures at week 12 of the study, in the treatment versus placebo groups
study drug overall efficiency as a seizure reducing treatment compared to placebo group
Measured: 12 weeks
Change in number of epileptic seizures as documented by patient diaries (Visit 2 level compared to Visit 3 level and Visit 4) in treatment and placebo group.
study drug overall efficiency as a seizure reducing treatment
Measured: 16 weeks

Secondary outcomes (8)

The incidence of adverse events will be summarized by organ class, severity and duration on weeks 12 and 18 of the study.
Adverse events assessment
Measured: 12-18 weeks
Any change in physical examination, vital signs, lab tests results, ect will be collected and analyzed.
Improvement in vital signs, lab test results and physical examination
Measured: 18 weeks
Change in duration of epileptic seizures as documented by patient diaries (Visit 2 level compared to Visit 3 level and Visit 4 level compared to Visit 2 level) by treatment group.
seizure frequency assessment
Measured: 12-18 weeks
Change in score from Quality of Life in Childhood Epilepsy Questionnaire 55 (QOLCE-55 questionnaire) scoring 0-100 points, higher scoring indicates better condition. scoring will be compared between Visit 2 level and Visit 4 level.
QoL questionnaire assessment
Measured: 18 weeks
Change in Clinical Global Impressions Scale (CGI scale) scoring 1-7 points, low scoring indicates better condition. Visit 2 level compared to Visit 4 level by treatment group.
CGI improvement by treatment group
Measured: 18 weeks
Percentage of MGCND00EP1-treated patients who will develop a response to MGCND00EP1 (response will be defined as a reduction of seizures frequency by at least 25 % ) as compared between Visit 2 level and Visit 4 level
response to MGCND00EP1
Measured: 18 weeks
Proportion of seizure-free patients between the placebo and treatment group on week 12 of treatment (including titration).
Proportion of seizure-free patients
Measured: 12 weeks
Change in form of new seizures and emergency of new forms will be monitored during the trial
New seizure or seizure form emergency
Measured: 18 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

30 May 202430 May 2024

First posted29 May 2020
Study start30 May 2024
Primary completion30 May 2024
Study completion30 May 2024

Why it stopped: Failure to overcome bureaucratic obstacles within timeframe.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (39)Papers the sponsor has linked to this study.
Other trials for Resistant Epilepsy, DrugStudies of the same condition, open and under way.
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: Randomised
Intervention model: Parallel groups
Model description: * Treatment with current antiepileptic therapy 28 days - Various combinations of anti-epileptic (AE) medications * Add on treatment 42 days (6 weeks) - titration period - MGCND00EP1 or placebo in addition to AE medications. * Add on treatment 42 days (6 weeks) Maintenance stable treatment period - MGCND00EP1 or placebo in addition to AE medications * Add on treatment 14 days (2 weeks)- taper - down titration period - MGCND00EP1 or placebo in addition to AE medications * Follow up 28 days - Standard/ previous AE treatment
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04406948
Sponsor's protocol ID: MGC-002
Status last verified: May 2024
First submitted: 17 May 2020
Last updated: 3 Jun 2024
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Publications

Berg AT, Zelko FA, Levy SR, Testa FM. Age at onset of epilepsy, pharmacoresistance, and cognitive outcomes: a prospective cohort study. Neurology. 2012 Sep 25;79(13):1384-91. doi: 10.1212/WNL.0b013e31826c1b55. Epub 2012 Sep 12.
PubMed 22972641
Devinsky O, Vickrey BG, Cramer J, Perrine K, Hermann B, Meador K, Hays RD. Development of the quality of life in epilepsy inventory. Epilepsia. 1995 Nov;36(11):1089-104. doi: 10.1111/j.1528-1157.1995.tb00467.x.
PubMed 7588453
Donner EJ. Opportunity gained, opportunity lost: treating pharmacoresistant epilepsy in children. Epilepsia. 2013 May;54 Suppl 2:16-8. doi: 10.1111/epi.12178.
PubMed 23646965
Cilio MR, Thiele EA, Devinsky O. The case for assessing cannabidiol in epilepsy. Epilepsia. 2014 Jun;55(6):787-90. doi: 10.1111/epi.12635. Epub 2014 May 22.
PubMed 24854434
Maa E, Figi P. The case for medical marijuana in epilepsy. Epilepsia. 2014 Jun;55(6):783-6. doi: 10.1111/epi.12610. Epub 2014 May 22.
PubMed 24854149
Sharp GB, Samanta D, Willis E. Options for pharmacoresistant epilepsy in children: when medications don't work. Pediatr Ann. 2015 Feb;44(2):e43-8. doi: 10.3928/00904481-20150203-11.
PubMed 25658218
Bent S. Herbal medicine in the United States: review of efficacy, safety, and regulation: grand rounds at University of California, San Francisco Medical Center. J Gen Intern Med. 2008 Jun;23(6):854-9. doi: 10.1007/s11606-008-0632-y. Epub 2008 Apr 16.
PubMed 18415652
Russo EB. History of cannabis and its preparations in saga, science, and sobriquet. Chem Biodivers. 2007 Aug;4(8):1614-48. doi: 10.1002/cbdv.200790144.
PubMed 17712811
Zuardi AW, Crippa JA, Hallak JE, Moreira FA, Guimaraes FS. Cannabidiol, a Cannabis sativa constituent, as an antipsychotic drug. Braz J Med Biol Res. 2006 Apr;39(4):421-9. doi: 10.1590/s0100-879x2006000400001. Epub 2006 Apr 3.
PubMed 16612464
Devinsky O, Marsh E, Friedman D, Thiele E, Laux L, Sullivan J, Miller I, Flamini R, Wilfong A, Filloux F, Wong M, Tilton N, Bruno P, Bluvstein J, Hedlund J, Kamens R, Maclean J, Nangia S, Singhal NS, Wilson CA, Patel A, Cilio MR. Cannabidiol in patients with treatment-resistant epilepsy: an open-label interventional trial. Lancet Neurol. 2016 Mar;15(3):270-8. doi: 10.1016/S1474-4422(15)00379-8. Epub 2015 Dec 24.
PubMed 26724101
Cortesi M, Fusar-Poli P. Potential therapeutical effects of cannabidiol in children with pharmacoresistant epilepsy. Med Hypotheses. 2007;68(4):920-1. doi: 10.1016/j.mehy.2006.09.030. Epub 2006 Nov 16. No abstract available.
PubMed 17112679
Dan B. Cannabinoids in paediatric neurology. Dev Med Child Neurol. 2015 Nov;57(11):984. doi: 10.1111/dmcn.12926. No abstract available.
PubMed 26443475
Press CA, Knupp KG, Chapman KE. Parental reporting of response to oral cannabis extracts for treatment of refractory epilepsy. Epilepsy Behav. 2015 Apr;45:49-52. doi: 10.1016/j.yebeh.2015.02.043. Epub 2015 Apr 3.
PubMed 25845492
Devinsky O, Cilio MR, Cross H, Fernandez-Ruiz J, French J, Hill C, Katz R, Di Marzo V, Jutras-Aswad D, Notcutt WG, Martinez-Orgado J, Robson PJ, Rohrback BG, Thiele E, Whalley B, Friedman D. Cannabidiol: pharmacology and potential therapeutic role in epilepsy and other neuropsychiatric disorders. Epilepsia. 2014 Jun;55(6):791-802. doi: 10.1111/epi.12631. Epub 2014 May 22.
PubMed 24854329
Bergamaschi MM, Queiroz RH, Zuardi AW, Crippa JA. Safety and side effects of cannabidiol, a Cannabis sativa constituent. Curr Drug Saf. 2011 Sep 1;6(4):237-49. doi: 10.2174/157488611798280924.
PubMed 22129319

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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Study at a glance

From the registry record, last updated by the sponsor on 3 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Randomized, Double Blind, Placebo Controlled, Parallel Design Phase II b Study of Safety and Efficacy of MGCND00EP1 as an Add on Treatment in Children and Adolescents With Resistant Epilepsies
ClinicalTrials.gov ID: NCT04406948
Acronym / sponsor ID: MGC-002
Conditions: Resistant Epilepsy, Drug, Adolescent Epilepsy, Children Epilepsy, Children and Adolescents With Resistant Epilepsies
Keywords: epilepsy, resistant epilepsy, adolescents, children
Phase: Phase 2
Study type: Interventional
Recruitment status: WithdrawnReason stopped: Failure to overcome bureaucratic obstacles within timeframe.
Enrolment: 0 participants (actual)
Lead sponsor: MGC Pharmaceuticals d.o.oIndustry (pharma, biotech, devices)
Study start: 30 May 2024 (actual)
Primary completion: 30 May 2024 (actual)
Study completion: 30 May 2024 (actual)
First posted: 29 May 2020 (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.

EudraCT: 2018-003887-29

Objective:To evaluate the safety and efficacy of: MGCND00EP1 from MGC PHARMACEUTICALS d.o.o.

According to dosing scheme up to 25 mg/kg BW per day or maximum daily dose 800 mg (whichever smaller) for 6 weeks titration and 6 weeks of treatment, oral administration

Detailed description

Day one - Screening and Enrollment Visit :

Weeks 1-4 - AED Stabilization Period :

Patients will be randomized and will either get placebo or MGCND00EP1 (3:1 active:placebo)

Weeks 5-10 - Dose titration period:

Dose escalations (2 mg/kg body weight/day increments), as required, up to 25 mg/kg/day or 800 mg/day, the lower of the two, until stable dose is reached.

Visit 3: vital signs, weight, physical and neurological exam, blood and urine tests, concomitant medications, questionnaires, collect and issue diaries, dispense study drug, monitor AEs

Weeks 11-16 - Maintenance Period :

Weeks 17-18 - Tapering-off and Follow-up Period:

Weekly phone call to determine taper dose at physician's discretion

Visit 5: vital signs,weight, physical and neurological exam, blood and urine tests, concomitant medications, questionnaires, monitor AEs, collect diaries and unused drug

Weeks 19-20 - Follow-up Period:

Weekly phone calls

Visits 6: vital signs,weight, physical and neurological exam, concomitant medications, monitor AEs, collect diaries .

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2
Model description: * Treatment with current antiepileptic therapy 28 days - Various combinations of anti-epileptic (AE) medications * Add on treatment 42 days (6 weeks) - titration period - MGCND00EP1 or placebo in addition to AE medications. * Add on treatment 42 days (6 weeks) Maintenance stable treatment period - MGCND00EP1 or placebo in addition to AE medications * Add on treatment 14 days (2 weeks)- taper - down titration period - MGCND00EP1 or placebo in addition to AE medications * Follow up 28 days - Standard/ previous AE treatment

Arm	Type	Receives
MGCND00EP1	Experimental	Drug: MGCND00EP1, Diagnostic Test: ECG, Diagnostic Test: EEG, Diagnostic Test: Blood and urine collectionParticipants who will assigned to receive add on MGCND00EP1 will receive carrier oil containing THC and CBD in ratio 20:1, (10% of cannabidiol and 0.5 % and (-)-trans-Δ9-tetrahydrocannabinol) . Titration Dose: 1 to 2 mg/kg body weight/day. dose will be increased every week by 2 mg/kg body weight/day up to a maximum 25 mg/kg body weigh/day or maximum daily dose 800 mg (the smaller of those 2 values) (divided into two daily doses). After titration, patients will be receiving a stable maintenance dose of IMP (up to 25 mg/kg BW per day or maximum daily dose 800 mg (whichever smaller)) for 6 weeks period. During forth treatment period participants will commence a 2 weeks down-titration taper period, followed by 4 weeks observational follow up period of previous standard AE treatment without IMP.
PLACEBO	Placebo comparator	Drug: Placebo, Diagnostic Test: ECG, Diagnostic Test: EEG, Diagnostic Test: Blood and urine collectionParticipants who are assigned to receive add on PLACEBO will be administered the carrier oil (without the active ingredients). Titration Dose: 1 to 2 mg/kg body weight/day. dose will be increased every week by 2 mg/kg body weight/day up to a maximum 25 mg/kg body weigh/day or maximum daily dose 800 mg (the smaller of those 2 values) (divided into two daily doses). After titration, patients will be receiving a stable maintenance dose of IMP (up to 25 mg/kg BW per day or maximum daily dose 800 mg (whichever smaller)) for 6 weeks period. During forth treatment period participants will commence a 2 weeks down-titration taper period, followed by 4 weeks observational follow up period of previous standard AE treatment without IMP.

Who may qualify

Ages: 1 year to 18 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (9 listed)

Patient has documented clinically confirmed diagnosis of epilepsy;
Patient did not respond to at least 2 AED's therapy given in adequate doses;
Patients current therapy is considered inadequate (not completely controlled by AEDs); patients had four or more countable seizures with a motor component per 4 week period;
Patient is aged 1 year - 18 years inclusive at screening age;
Patient took one or more AEDs treatment at dose which has been stable for at least 4 weeks before enrolment;

Key exclusion criteria (14 listed)

Known history or presence of clinically significant unstable medical condition other that epilepsy which, in the opinion of the Investigator, would jeopardize the safety of the subject or impact the validity of the study results.
Known history or presence of serious cardiovascular disease
Known or suspected history or family history of: schizophrenia, or other psychotic illness, severe personality disorder or other significant psychiatric disorder.
Known or suspected allergy hypersensitivity or idiosyncratic reaction to cannabinoids or any other drug substances with similar activity or to any of the excipients of the IMP.
Participant has clinically relevant abnormalities in the 12-lead electrocardiogram measured at screening or randomisation.

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
MGCND00EP1Also: Cannabis oilPatients will take cannabis oil during the study	Drug	MGCND00EP1
PlaceboPatient will take carrier oil during the study	Drug	PLACEBO
ECGA standard 12-lead ECG will be recorded using digital ECG recording equipment provided to the investigational site. The ECG has to be performed prior to laboratory samplings at time points indicated in the Schedule of Assessments. The ECG recording will be reviewed by investigator and case of need consultation with cardiologist will be performed. The investigator has the final decision on the clinical significance of the ECG results.	Diagnostic test	MGCND00EP1, PLACEBO
EEGAn EEG is an electrophysiological monitoring method that records the electrical activity and measures voltage fluctuations resulting from ionic current within the neurons of the brain. In clinical contexts, EEG refers to the recording of the brain's spontaneous electrical activity over a period of time.	Diagnostic test	MGCND00EP1, PLACEBO
Blood and urine collectionAlso: blood testssafety blood tests - hematology\blood count and biochemistry standard blood tests urinalysis - urine test analysis	Diagnostic test	MGCND00EP1, PLACEBO

Primary outcome

The proportion of patients showing a >50% reduction in frequency of seizures at week 12 of the study, in the treatment versus placebo groups
Time frame: 12 weeks
study drug overall efficiency as a seizure reducing treatment compared to placebo group
Change in number of epileptic seizures as documented by patient diaries (Visit 2 level compared to Visit 3 level and Visit 4) in treatment and placebo group.
Time frame: 16 weeks
study drug overall efficiency as a seizure reducing treatment

8 secondary outcomes

The incidence of adverse events will be summarized by organ class, severity and duration on weeks 12 and 18 of the study.
Time frame: 12-18 weeks
Any change in physical examination, vital signs, lab tests results, ect will be collected and analyzed.
Time frame: 18 weeks
Change in duration of epileptic seizures as documented by patient diaries (Visit 2 level compared to Visit 3 level and Visit 4 level compared to Visit 2 level) by treatment group.
Time frame: 12-18 weeks
Change in score from Quality of Life in Childhood Epilepsy Questionnaire 55 (QOLCE-55 questionnaire) scoring 0-100 points, higher scoring indicates better condition. scoring will be compared between Visit 2 level and Visit 4 level.
Time frame: 18 weeks
Change in Clinical Global Impressions Scale (CGI scale) scoring 1-7 points, low scoring indicates better condition. Visit 2 level compared to Visit 4 level by treatment group.
Time frame: 18 weeks
Percentage of MGCND00EP1-treated patients who will develop a response to MGCND00EP1 (response will be defined as a reduction of seizures frequency by at least 25 % ) as compared between Visit 2 level and Visit 4 level
Time frame: 18 weeks
Proportion of seizure-free patients between the placebo and treatment group on week 12 of treatment (including titration).
Time frame: 12 weeks
Change in form of new seizures and emergency of new forms will be monitored during the trial
Time frame: 18 weeks

Results and safety

The study was withdrawn before enrolling participants, so there are no results.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2024.
Background: PubMed 22972641Berg AT, Zelko FA, Levy SR, Testa FM. Age at onset of epilepsy, pharmacoresistance, and cognitive outcomes: a prospective cohort study. Neurology. 2012 Sep 25;79(13):1384-91. doi: 10.1212/WNL.0b013e31826c1b55. Epub 2012 Sep 12.
Background: PubMed 7588453Devinsky O, Vickrey BG, Cramer J, Perrine K, Hermann B, Meador K, Hays RD. Development of the quality of life in epilepsy inventory. Epilepsia. 1995 Nov;36(11):1089-104. doi: 10.1111/j.1528-1157.1995.tb00467.x.
Background: PubMed 23646965Donner EJ. Opportunity gained, opportunity lost: treating pharmacoresistant epilepsy in children. Epilepsia. 2013 May;54 Suppl 2:16-8. doi: 10.1111/epi.12178.
Background: PubMed 24854434Cilio MR, Thiele EA, Devinsky O. The case for assessing cannabidiol in epilepsy. Epilepsia. 2014 Jun;55(6):787-90. doi: 10.1111/epi.12635. Epub 2014 May 22.
Background: PubMed 24854149Maa E, Figi P. The case for medical marijuana in epilepsy. Epilepsia. 2014 Jun;55(6):783-6. doi: 10.1111/epi.12610. Epub 2014 May 22.
Background: PubMed 25658218Sharp GB, Samanta D, Willis E. Options for pharmacoresistant epilepsy in children: when medications don't work. Pediatr Ann. 2015 Feb;44(2):e43-8. doi: 10.3928/00904481-20150203-11.
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Background: PubMed 16612464Zuardi AW, Crippa JA, Hallak JE, Moreira FA, Guimaraes FS. Cannabidiol, a Cannabis sativa constituent, as an antipsychotic drug. Braz J Med Biol Res. 2006 Apr;39(4):421-9. doi: 10.1590/s0100-879x2006000400001. Epub 2006 Apr 3.
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nice.org-( dne: 14.februarja 2016)Link provided by the sponsor.
Individual participant dataSharing planned: No

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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.

Rubi Zomer study director

MGC Pharmacuticals

2 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing MGCND00EP1, ECG and EEG in people with Resistant Epilepsy, Drug and Adolescent Epilepsy, run by MGC Pharmaceuticals d.o.o at 2 sites in 2 countries.

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 9 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: withdrawn, as of the sponsor's last update on 3 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Israel, Slovenia. 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. Some participants in this study may receive a placebo. 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?

The study was withdrawn before enrolling participants, so there are no results.

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?

The proportion of patients showing a >50% reduction in frequency of seizures at week 12 of the study, in the treatment versus placebo groups (time frame: 12 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?

On the ClinicalTrials.gov record for NCT04406948, which also lists 39 linked publications.

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 3 Jun 2024.