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    Summary
    EudraCT Number:2020-004053-76
    Sponsor's Protocol Code Number:NL73102.018.20
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-12-10
    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 number2020-004053-76
    A.3Full title of the trial
    Effectiveness of methylphenidate in children and adults with Smith Magenis syndrome and attention-deficit/hyperactivity disorder: An N-of-1 series
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Effectiveness of methylphenidate for treating ADHD in children and adults with Smith Magenis syndrome
    A.3.2Name or abbreviated title of the trial where available
    Methylphenidate for ADHD in Smith Magenis syndrome
    A.4.1Sponsor's protocol code numberNL73102.018.20
    A.7Trial is part of a Paediatric Investigation Plan No
    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.4.1Name of organisation providing support's Heeren Loo
    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 pointEmma Children's Hospital
    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+31641913205
    B.5.6E-maila.m.vaneeghen@amsterdamumc.nl
    B.Sponsor: 2
    B.1.1Name of Sponsor's Heeren Loo
    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 support's Heeren Loo
    B.4.2CountryNetherlands
    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 organisation's Heeren Loo
    B.5.2Functional name of contact pointAdvisium
    B.5.3 Address:
    B.5.3.1Street AddressBerkenweg 11
    B.5.3.2Town/ cityAmersfoort
    B.5.3.3Post code3818 LA
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31641913205
    B.5.6E-mailagnies.van-eeghen@sheerenloo.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 Medikinet
    D.2.1.1.2Name of the Marketing Authorisation holderMedice Arzneimittel Pütter GmbH & Co. KG, Iserlohn, Germany
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 Capsule
    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 Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    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 placeboCapsule
    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
    Smith Magenis syndrome
    ADHD
    E.1.1.1Medical condition in easily understood language
    Smith Magenis syndrome
    Smith Magenis syndrome
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to investigate the effectiveness of methylphenidate for ADHD-symptoms in Smith Magenis syndrome (SMs).
    E.2.2Secondary objectives of the trial
    To determine the effect of methylphenidate on emotion dysregulation and specific personalized goals that are important to the patient and its environment by means of Goal Attainment Scaling (GAS).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - A diagnosis of SMs confirmed with standard genetic testing (such as FISH test, microarray, WES-analysis).
    - Meet DSM-5 criteria for ADHD, and diagnosed with ADHD by a multidisciplinary team consisting of an intellectual disability physician, a psychologist, and a psychiatrist.
    - Minimum age of six years old.
    - Presence of a patient’s caregiver for proxy-reports.
    E.4Principal exclusion criteria
    - Presence of a contra-indication for treatment with methylphenidate.
    - Planned general anaesthesia during the trial.
    - Pregnancy.
    - Breastfeeding females.
    - Females of childbearing potential must be willing to use an effective method of contraception from the time consent is signed until 6 weeks after treatment discontinuation and inform the trial if pregnancy occurs.
    - During treatment with non-selective, irreversible monoamine oxidase (MAO) inhibitors, or within a minimum of 14 days of discontinuing those drugs.
    - Current treatment with serotonergic drugs, acetazolamide, thiazide-diuretic and sodium bicarbonate, sympathicomimetics, tricyclic antidepressants, or anti-psychotics.
    - Current substance or alcohol abuse.
    - Unable to swallow tablets / capsules.
    E.5 End points
    E.5.1Primary end point(s)
    The primary outcome measure is the hyperactivity/inattention subscale of the SDQ, consisting of five items. The response options that are about ‘the recent past’ will be substituted by ‘today’. The SDQ subscale has been psychometrically considered as a valid tool to measure behaviour of people with intellectual disability and applicable to both children and adults.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Daily.
    E.5.2Secondary end point(s)
    Secondary outcome measures are the Dutch shortened version of the Emotion Dysregulation Inventory (EDI) reactivity index consisting of seven items, goal attainment scaling (GAS) to identify and measure individualized goals with regard to the most obstructing symptoms experienced by parents, and the personal questionnaire (PQ) as a symptom list to compare assessments of personalized goals with those measured by GAS. Another study parameter will be the (number of) side effects determined by the side effects checklist of methylphenidate.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The shortened version of the EDI will be evaluated daily. GAS, the PQ, and the side effects checklist of methylphenidate will be evaluated weekly.
    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 No
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 Yes
    E.8.1.7.1Other trial design description
    N-of-1 series
    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 trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    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 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
    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: 6
    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: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 2
    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
    Smith Magenis syndrome is often associated with intellectual disability.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    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 three 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 Decision2020-12-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-12-07
    P. End of Trial
    P.End of Trial StatusOngoing
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