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    Summary
    EudraCT Number:2021-003250-23
    Sponsor's Protocol Code Number:78162.018.21
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2022-08-25
    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
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2021-003250-23
    A.3Full title of the trial
    Cannabidiol (Epidyolex) for behavioural problems in patients with Tuberous Sclerosis Complex, Sanfilippo and Fragile X Syndrome: an N-of-1 series
    Cannabidiol (Epidyolex) voor gedragsproblemen bij patiënten met Tubereuze Sclerosis Complex, Sanfilippo en Fragiele X Syndroom: een N-of-1 serie
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Effectiveness of CBD on behavioural problems in patients with TSC, FXS and Sanfilippo
    Effect van CBD-olie op gedragsproblemen bij patiënten met Tubereuze Sclerosis Complex, Fragiele X-syndroom en Sanfilippo
    A.3.2Name or abbreviated title of the trial where available
    CBD4RARE
    CBD4RARE
    A.4.1Sponsor's protocol code number78162.018.21
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAmsterdam UMC
    B.1.3.4CountryNetherlands
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAmsterdam UMC
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmsterdam UMC
    B.5.2Functional name of contact pointDr A.M. van Eeghen
    B.5.3 Address:
    B.5.3.1Street AddressMeibergdreef 9
    B.5.3.2Town/ cityAmsterdam
    B.5.3.3Post code1105 AZ
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31205669111
    B.5.6E-mailonderzoeksbureauvkc@amsterdamumc.nl
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleComparator
    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 (Epidyolex)
    D.2.1.1.2Name of the Marketing Authorisation holderGW Pharma (International) B.V., Databankweg 26 3821AL Amersfoort, The Netherlands
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral solution
    D.8.4Route of administration of the placeboOral use
    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), Sanfilippo syndrome, Fragile X syndrome (FXS)
    E.1.1.1Medical condition in easily understood language
    Tuberous Sclerosis Complex (TSC), Sanfilippo syndrome, Fragile X syndrome (FXS)
    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 20.0
    E.1.2Level SOC
    E.1.2Classification code 10010331
    E.1.2Term Congenital, familial and genetic disorders
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level HLGT
    E.1.2Classification code 10027424
    E.1.2Term Metabolic and nutritional disorders congenital
    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
    To investigate the effectiveness of CBD on irritability and other behavioural problems in children and adults with TSC, Sanfilippo and FXS, and autism.
    E.2.2Secondary objectives of the trial
    The secondary objective is to examine the effects of CBD on aggression, hyperactivity, anxiety, mood, patient-centered outcome measures (PCOMs), disease-specific outcome measures, seizure frequency, and side effects.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Minimum age of 6 years old.
    - Clinically definite diagnosis of TSC, Sanfilippo or FXS (modified Gomez criteria or positive genetic test (such as FISH test, microarray, WES-analysis)).
    - Suffering from severe behavioural manifestation with a minimum score of 4 on the CGI scale.
    - All medications or interventions for epilepsy and behavioural manifestations must have been stable dosed for one month prior to screening and the participant is willing to maintain the current regimen throughout the trial.
    - Presence of a consistently available patient caregiver for proxy-reports.
    E.4Principal exclusion criteria
    • Use of valproate should be stable three months prior to enrolment;
    • Any known or suspected hypersensitivity to cannabinoids or any of the excipients of the Investigational Medicinal Product (IMP), such as sesame oil;
    • Participant is currently using or has in the past used recreational or medicinal cannabis, or cannabinoid-based medications, within three months prior to screening and is unwilling to abstain for the duration of the study;
    • Treatment with CBD or other cannabinoid within the previous two months;
    • History or current evidence of significantly impaired liver function, defined as 1) Alanine aminotransferase (ALT) and / or aspartate aminotransferase (AST) > 5 × upper limit of normal (ULN); 2) ALT or AST > 3 × ULN with concomitant total bilirubin > 2.0 × ULN; or 3) ALT or AST ≥ 3 × ULN with the appearance of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia;
    • Pregnancy or breastfeeding;
    • Glaucoma;
    • Participant has undergone general anaesthesia in the four weeks prior to randomization;
    • Use of any interfering medication within 30 days prior to enrolment or planning to take interfering medication during the trial;
    • Planned changes in use of anti-epileptics;
    • Subjects who have had changes in non-exclusionary psychotropic medications within 4 weeks of initiation of trial;
    • Any planned major surgery within the duration of the trial;
    • Expected inability to take blood samples due to anxiety or resistance.;
    • Unable to swallow the study drug (or placebo);
    • The patient is unwilling or, in the investigator’s opinion, unable to adhere to the requirements of the study;
    • Any condition or abnormality which may, in opinion of the investigator, compromise the safety of patients.
    E.5 End points
    E.5.1Primary end point(s)
    To examine the effectiveness of CBD on behavioural problems using the irritability subscale of the Aberrant Behavior Checklist (ABC) in children and adults with TSC, Sanfilippo syndrome and FXS.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Weekly
    E.5.2Secondary end point(s)
    • To examine the effectiveness of CBD on the Behaviour problems using total ABC score in children and adults with TSC, Sanfilippo syndrome and FXS;
    • To examine the effectiveness of CBD on anxiety, depression and mood using the Anxiety, Depressions and Mood Scale (ADAMS) in children and adults with TSC, Sanfilippo syndrome and FXS;
    • To examine the effectiveness of CBD on autism symptoms using the Social Communication Questionnaire (SCQ) and Social Responsiveness Scale (SRS-2) (when applicable) in children and adults with TSC, Sanfilippo syndrome and FXS;
    • To examine the effectiveness of CBD on sensory processing using the Short Sensory Profile (SP-NL) (when applicable) in children and adults with TSC, Sanfilippo syndrome and FXS;
    • To examine the effectiveness of CBD on functional / developmental outcomes using the Goal Attainment Scaling (GAS) and Personal Questionnaire (PQ) in children and adults with TSC, Sanfilippo syndrome and FXS;
    • To examine the effectiveness of CBD on symptom severity and treatment response using the Clinical Global Impression (CGI) scale in children and adults with TSC, Sanfilippo syndrome and FXS;
    • To examine the effectiveness of CBD on parental stress using the Opvoedingsbelasting Vragenlijst (OBVL) (when applicable) in children and adults with TSC, Sanfilippo syndrome and FXS;
    • To examine the effectiveness of CBD on quality of life using syndrome-specific outcome measures or Pediatric Quality of Life Inventory (PedsQL) in children and adults with TSC, Sanfilippo syndrome and FXS;
    • To examine the effectiveness of CBD on seizure frequency using a seizure diary in children and adults with TSC, Sanfilippo syndrome and FXS.
    E.5.2.1Timepoint(s) of evaluation of this end point
    End of (interventional) periods
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    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: 18
    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) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 9
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 9
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 12
    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
    All three diseases are associated with intellectual disability.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    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)
    After completing the cycles of the N-of-1 design, patients will discuss the results (personalized treatment response) with the treating physician to determine either discontinuation or continuation with the same or a different dosage of treatment. After 6 months, a final optional assessment will take place.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-08-25
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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