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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04406948
Recruitment Status : Not yet recruiting
First Posted : May 29, 2020
Last Update Posted : May 29, 2020
Sponsor:
Information provided by (Responsible Party):
MGC Pharmaceuticals d.o.o

Brief 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 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


Condition or disease Intervention/treatment Phase
Resistant Epilepsy, Drug Adolescent Epilepsy Children Epilepsy Children and Adolescents With Resistant Epilepsies Drug: MGCND00EP1 Drug: Placebo Diagnostic Test: ECG Diagnostic Test: EEG Diagnostic Test: Blood and urine collection Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 103 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention 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 (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Estimated Study Start Date : November 2020
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : December 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Epilepsy

Arm Intervention/treatment
Experimental: MGCND00EP1

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.

Drug: MGCND00EP1
Patients will take cannabis oil during the study
Other Name: Cannabis oil

Diagnostic Test: ECG

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.


Diagnostic Test: EEG
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.

Diagnostic Test: Blood and urine collection
safety blood tests - hematology\blood count and biochemistry standard blood tests urinalysis - urine test analysis
Other Name: blood tests

Placebo Comparator: PLACEBO

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.

Drug: Placebo
Patient will take carrier oil during the study

Diagnostic Test: ECG

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.


Diagnostic Test: EEG
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.

Diagnostic Test: Blood and urine collection
safety blood tests - hematology\blood count and biochemistry standard blood tests urinalysis - urine test analysis
Other Name: blood tests




Primary Outcome Measures :
  1. 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

  2. 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


Secondary Outcome Measures :
  1. 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 ]
    Adverse events assessment

  2. Any change in physical examination, vital signs, lab tests results, ect will be collected and analyzed. [ Time Frame: 18 weeks ]
    Improvement in vital signs, lab test results and physical examination

  3. 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 ]
    seizure frequency assessment

  4. 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 ]
    QoL questionnaire assessment

  5. 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 ]
    CGI improvement by treatment group

  6. 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 ]
    response to MGCND00EP1

  7. Proportion of seizure-free patients between the placebo and treatment group on week 12 of treatment (including titration). [ Time Frame: 12 weeks ]
    Proportion of seizure-free patients

  8. Change in form of new seizures and emergency of new forms will be monitored during the trial [ Time Frame: 18 weeks ]
    New seizure or seizure form emergency



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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;
  • 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.

Exclusion Criteria:

  • 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.
  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04406948


Contacts
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Contact: Nadia Lisovoder +972529573063 nadyal@galilee-cbr.com

Locations
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Israel
Schneider Children's Medical Center of Israel
Petach Tikvah, Central District, Israel, 4920235
Contact: Dror Kraus    +972523302002      
Slovenia
University Children's Hospital Ljubljana University Medical Centre Ljubljana
Ljubljana, Slovenia, 1000
Contact: David Neubauer, MD, PhD    +386 1 522 9231      
Sponsors and Collaborators
MGC Pharmaceuticals d.o.o
Investigators
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Study Director: Rubi Zomer MGC Pharmacuticals
Additional Information:
Publications:
Goldstein B. Cannabis in the treatment of pediatric epilepsy. O'Shaugnessy's 2016:7-9.
O'Connell BK, Gloss D, Devinsky O. Cannabinoids in treatment-resistant epilepsy: A review. Epilepsy Behav. 2017 Feb 8. pii: S1525-5050(16)30625-4. doi: 10.1016/j.yebeh.2016.11.012. [Epub ahead of print]
Sativex smpc , 20.09.2018

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Responsible Party: MGC Pharmaceuticals d.o.o
ClinicalTrials.gov Identifier: NCT04406948    
Other Study ID Numbers: MGC-002
First Posted: May 29, 2020    Key Record Dates
Last Update Posted: May 29, 2020
Last Verified: May 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by MGC Pharmaceuticals d.o.o:
epilepsy
resistant epilepsy
adolescents
children
Additional relevant MeSH terms:
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Epilepsy
Drug Resistant Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases