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    Summary
    EudraCT Number:2020-002132-67
    Sponsor's Protocol Code Number:GWEP17005
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2021-10-22
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2020-002132-67
    A.3Full title of the trial
    An Open-Label, Randomized Trial to Assess the Safety, Pharmacokinetics, and Exploratory Efficacy of Adjunctive Cannabidiol Oral Solution (GWP42003-P) Compared with Standard of Care Antiseizure Medication, in Patients Age 1 Month to Less than 12 Months of Age with Tuberous Sclerosis Complex who Experience Inadequately-Controlled Seizures
    P/0047/2020
    P/0350/2020
    P/0033/2021
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety, Pharmacokinetics, and Exploratory Efficacy Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) Compared With Standard of Care Antiseizure Medication, in Patients Age 1 Month to <12 Months of Age With Tuberous Sclerosis Complex Who Experience Inadequately-controlled Seizures
    A.4.1Sponsor's protocol code numberGWEP17005
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04485104
    A.5.4Other Identifiers
    Name:INDNumber:120055
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/136/2017
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGW Research Ltd
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportGW Research Ltd
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGW Research Ltd
    B.5.2Functional name of contact pointGW Research Ltd Switchboard
    B.5.3 Address:
    B.5.3.1Street AddressSovereign House, Vision Park, Chivers Way
    B.5.3.2Town/ cityHiston, Cambridge
    B.5.3.3Post codeCB24 9BZ
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+441223266800
    B.5.5Fax number+441223235667
    B.5.6E-mailinfo@gwpharm.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Cannabidiol Oral Solution, is known as Epidyolex 100 mg/mL oral solution and is the approved name in the EU. Epidiolex (cannabidiol) oral solution is the approved name in the US.
    D.2.1.1.2Name of the Marketing Authorisation holderGW Pharma (International) B.V.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1959
    D.3 Description of the IMP
    D.3.1Product nameCannabidiol (CBD)
    D.3.2Product code GWP42003-P
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product Yes
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product Yes
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Tuberous Sclerosis Complex (TSC)
    E.1.1.1Medical condition in easily understood language
    Genetic disease of benign tumour growth with multiple symptoms including seizures
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10045138
    E.1.2Term Tuberous sclerosis
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level PT
    E.1.2Classification code 10080584
    E.1.2Term Tuberous sclerosis complex
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part A: To evaluate the safety and tolerability of GWP42003-P compared with standard of care (SOC) antiseizure medication (ASM) assessed during the 17-week treatment period.
    Part B: To evaluate the long-term safety and tolerability of GWP42003-P.
    E.2.2Secondary objectives of the trial
    Part A:
    To investigate the exposure of GWP42003-P and its major metabolites following multiple doses of GWP42003-P.
    To evaluate the exploratory efficacy of GWP42003-P in reducing the frequency of countable seizures when compared to SOC.
    To evaluate the exploratory effects of GWP42003-P on quality of life compared with SOC.
    Part B:
    To evaluate the exploratory effects of GWP42003-P on long-term quality of life.
    To evaluate the long-term exploratory efficacy of GWP42003-P in reducing the frequency of countable seizures.
    To investigate the exposure of GWP42003-P and its major metabolites following multiple doses of GWP42003-P.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    For inclusion in the trial, patients must fulfill ALL of the following criteria:
    1. Must be 1 month to < 12 months of age at the time of initial informed consent.
    2. Parent(s)/legal representative is/are willing and able to give informed consent for participation in the trial.
    3. Parent(s)/legal representative is/are willing and able (in the investigator’s opinion) to comply with all trial requirements (including accurate ePRO diary completion).
    4. Caregiver completes at least 75% of ePRO and paper seizure diary entries during the 28 days of the baseline period (≥ 22 days of entries).
    5. Must have a clinical diagnosis of TSC according to the investigator and as defined by 2012 International Tuberous Sclerosis Complex Consensus Conference and ILAE.
    6. Multichannel (minimum 8-channel) 8- to 24 hour VEEG, to be read prior to Part A Visit 3 by the investigator and an independent reviewer, for confirmation of diagnosis of inadequately-controlled seizures is required in Part A. In certain cases, the VEEG may be collected from the patient’s medical record if suitable (i.e., meet all of the following criteria):
    a. A suitable VEEG meets all the following criteria:
    i. Multichannel (minimum 8-channel)
    ii. 8- to 24-hour recording
    iii. Completed within 1 year of Part A Visit 1
    iv. Consistent with the patient’s current seizures (in the investigator’s opinion)
    v. Can be reviewed by the investigator and an independent reviewer prior to randomization
    vi. Consistent with a diagnosis of inadequately-controlled seizures
    b. If a suitable VEEG is not available in the patient’s medical record a multichannel (minimum 8-channel) 8- to 24-hour VEEG will be completed and read, prior to Visit 3, by the investigator and an independent reviewer to confirm consistency with a diagnosis of inadequately-controlled seizures.
    7. Currently taking ≥ 1 ASMs at a dose which remains stable 2 weeks prior to randomization (Visit 3) and remain stable during the treatment period. Where required for patient safety, adjustments of concomitant ASMs or addition of new ASM may be permitted following discussion with the medical monitor.
    a. ACTH or high dose corticosteroids for the treatment of infantile spasms are counted as ASMs.
    8. Has seizures which are not adequately controlled through their current ASMs, defined as ≥ 1 seizure reported on the paper and ePRO seizure diary during the baseline period.
    9. Parent(s)/legal representative is/are willing to allow the responsible authorities to be notified of participation in the trial, if mandated by local law.
    10. Parent(s)/legal representative is/are willing to allow the patient’s primary care practitioner (if they have one) and consultant (if they have one) to be notified of participation in the trial if the primary care practitioner/consultant is different from the investigator.
    Part A Only:
    11. All medications or interventions for epilepsy must remain stable 2 weeks prior to Visit 3. Where required for patient safety, adjustments of concomitant ASMs or addition of a new ASM may be permitted following discussion with the medical monitor.
    12. The caregiver(s) are willing to maintain a stable regimen of ASMs throughout the trial.
    Part B Only:
    13. Has completed Visit 12 of Part A, if randomized to GWP42003-P and SOC ASM or completed at least 26 days of Part A if randomized to SOC ASM.
    14. Was compliant with all requirements of Part A (e.g., dosing, paper and ePRO seizure diary, patient visits/procedures), in the opinion of the investigator and sponsor.
    15. Investigator considers continued treatment in a 1-year extension trial represents a favorable risk-benefit assessment for the patient.
    E.4Principal exclusion criteria
    Part A and B:
    1. Has clinically significant unstable medical condition other than epilepsy.
    2. Has had clinically significant symptoms or a clinically significant illness within the 4 weeks prior to Visit 1, other than epilepsy, which in the opinion of the investigator could affect seizure frequency.
    3. Has undergone general anesthesia in the 4 weeks prior to Visit 1.
    4. Has undergone surgery for epilepsy within 6 months prior to Visit 1.
    5. Has taken felbamate prior to Visit 1.
    6. Has taken valproic acid within 4 weeks prior to Visit 1.
    7. Has tumor growth which, in the opinion of the investigator, could affect patient safety.
    8. Has clinically significant abnormal laboratory values, in the investigator’s opinion, at screening/baseline.
    9. Has clinically significant abnormalities in the ECG measured at screening. Including, QT interval corrected for heart rate with Bazett’s formula (QTcB), of > 460 msec on ECG.
    10. Has any concurrent cardiovascular conditions, which will, in the investigator’s opinion, interfere with the ability to assess their ECGs.
    11. Has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP such as sesame seed oil.
    12. Has significantly impaired hepatic function prior to randomization, defined as:
    o Serum ALT or AST > 3 × ULN and (TBL > 2 × ULN or international normalized ratio [INR] > 1.5).
    o Elevated ALT or AST should be discussed with the medical monitor prior to randomization; the medical monitor may allow for a confirmatory re-draw prior to randomization.
    13. Has received another IMP within 4 weeks prior to Visit 1.
    14. Caregiver is currently giving or has given recreational or medicinal cannabis, cannabinoid-based medications (including Sativex®) or CBD (including Epidiolex® [GWP42003 P]) to the patient within the 4 weeks prior to Visit 1 and is unwilling to abstain from doing so for the duration of the trial.
    15. Mother (if breastfeeding) is currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex®) or CBD (including Epidiolex® [GWP42003-P]) within the 4 weeks prior to Visit 1 and is unwilling to abstain from doing so for the duration of the trial.
    16. Has any other clinically significant disease or disorder which, in the opinion of the investigator, may either put the patient, other patients, or site staff at risk because of participation in the trial, may influence the result of the trial, or may affect the patient’s ability to take part in the trial.
    17. Any clinically significant abnormalities identified following a physical examination of the patient that, in the opinion of the investigator, would jeopardize the safety of the patient if they took part in the trial.
    18. Has previously been randomized into this trial.
    19. Has plans to travel outside their country of residence during the trial, unless the patient has confirmation that the IMP is permitted in the destination country and all stops along the way.
    Part B Only:
    20. Has had clinically significant symptoms or a clinically significant illness in the 4 weeks prior to Part B, Visit 1 other than epilepsy, which in the opinion of the investigator could affect seizure frequency.
    21. Has undergone surgery for epilepsy during Part A.
    22. Has taken felbamate during Part A.
    23. Has taken valproic acid during Part A.
    24. Has clinically significant abnormalities in the ECGs in Part A. Including, QT interval corrected for heart rate with Bazett’s formula (QTcB), of > 460 msec on ECG.
    25. Has significantly impaired hepatic function during Part A, defined as:
    o Serum ALT or AST > 3 × ULN and (TBL > 2 x ULN or INR > 1.5).
    o Elevated ALT or AST should be discussed with the medical monitor prior to rollover in Part B; the medical monitor may allow for a confirmatory redraw prior to rollover.
    26. Caregiver is currently giving or has given recreational or medicinal cannabis, cannabinoid-based medications (including Sativex®) or CBD other than the IMP (GWP42003-P) to the patient during Part A and is unwilling to abstain from doing so for the duration of the trial.
    27. Mother (if breastfeeding) is currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex®) or CBD (including Epidiolex® [GWP42003-P]) during Part A and is unwilling to abstain from doing so for the duration of the trial.
    28. Has previously been enrolled in Part B of this trial.
    E.5 End points
    E.5.1Primary end point(s)
    Part A:
    The safety profile of GWP42003-P compared with SOC ASM will be assessed by measuring:
    o Adverse events (AEs) (frequency, type, and severity).
    o Vital signs.
    o Physical examination.
    o 12-lead electrocardiogram (ECG).
    o Clinically significant changes in laboratory parameters.
    o Emergence of new seizure types.
    Part B:
    The safety profile of GWP42003-P will be assessed by measuring:
    o AEs (frequency, type, and severity).
    o Vital signs.
    o Physical examination.
    o 12-lead ECG.
    o Clinically significant changes in laboratory parameters.
    o Emergence of new seizure types.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the study according to the schedule of assessments in the protocol
    E.5.2Secondary end point(s)
    Part A:
    • Trough, 3-hour and 6-hour postdose plasma concentrations of cannabidiol (CBD) and its major metabolites following multiple doses of GWP42003 P.
    • Percent change from baseline in caregiver and investigator reported total countable seizure frequency, as recorded on seizure diaries.
    • Proportion of patients with a ≥ 50% reduction in caregiver and investigator reported total countable seizure frequency, from the baseline period compared to Part A treatment period, as recorded on seizure diaries.
    • Worsening and improvement from the baseline period compared to Part A treatment period of countable seizure frequency as recorded by caregivers and investigators on seizure diaries:
    o Worsening: > 25% change
    o No Change Increase: ≤ 25% to ≥ 0% change
    o No Change Decrease: < 0% to > -25% change
    o Improvement category 1: ≤ -25% to > -50% change
    o Improvement category 2: ≤ -50 to > -75% change
    o Improvement category 3: ≤ -75% change
    • Seizure free status.
    • Change in seizure frequency from baseline to Part A End of Treatment as captured by multichannel (minimum of 8 channels) video VEEG with minimum 8 to 24 hours of recording.
    • Change in EEG seizure burden (e.g., seizure type(s), severity and frequency recorded during EEGs), baseline compared to Part A End of Treatment.
    • Where possible, correlation of seizures recorded during multichannel VEEG with countable clinical seizures recorded by caregivers and investigators on seizure diaries.
    • Change from baseline in scores of ITQOL-SF47 questionnaire at Part A End of Treatment.
    Part B:
    • Change from baseline in scores of ITQOL-SF47 score at Part B End of Treatment.
    •Percentage change in total countable seizure frequency, as recorded by caregivers and investigators on seizure diaries for each 12-week period from baseline to Part B End of Treatment.
    • Seizure free status.
    • Trough, 3-hour and 6-hour postdose plasma concentrations of CBD and its major metabolites following multiple doses of GWP42003-P.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the study according to the schedule of assessments in the protocol
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Standard of Care Antiseizure Medication
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LPLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months7
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 15
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 15
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Paediatric population from 1 month to <12 months of age
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 15
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Participant and Investigator may consider continued treatment in a 1-year extension trial (Part B) if a favorable risk-benefit assessment for the participant.
    Routine standard of care will be continued after a participant has ended participation in the trial.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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