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    Summary
    EudraCT Number:2019-000735-61
    Sponsor's Protocol Code Number:SP-NAC19
    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:2020-08-04
    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 number2019-000735-61
    A.3Full title of the trial
    N- acetylcysteine treatment for skin picking in children and young adults with PWS: a randomized, controlled, cross-over trial.
    Behandeling met N-acetylcysteïne voor skin picking gedrag in kinderen en jongvolwassenen met PWS: een gerandomiseerde, gecontroleerde, cross-over studie
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Treatment with N-acetylcysteine for skin picking in children and young adults with PWS
    Behandeling met N-acetylcysteïne voor skin picking in kinderen en jongvolwassenen met PWS
    A.4.1Sponsor's protocol code numberSP-NAC19
    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 SponsorDutch Growth Research Foundation
    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 supportDutch Growth Research Foundation
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDutch Growth Research Foundation
    B.5.2Functional name of contact pointProject leader Prader-Willi Studies
    B.5.3 Address:
    B.5.3.1Street AddressWestzeedijk 106
    B.5.3.2Town/ cityRotterdam
    B.5.3.3Post code3016 AH
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31102251533
    B.5.6E-mailpraderwilli@kindengroei.nl
    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 Fluimucil (acetylcysteine)
    D.2.1.1.2Name of the Marketing Authorisation holderZambon Group
    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.3.1Product nameFluimucil
    D.3.2Product code R05CB01
    D.3.4Pharmaceutical form Effervescent tablet
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Prader-Willi syndrome
    Prader-Willi syndroom
    E.1.1.1Medical condition in easily understood language
    Prader-Willi syndrome
    Prader-Willi syndroom
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10036476
    E.1.2Term Prader-Willi syndrome
    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 effects of N-acetylcysteine treatment on skin picking behavior, measured by the change in total number and size of the skin picking laesions in children and young adults with PWS
    E.2.2Secondary objectives of the trial
    To investigate the effects of N-acetylcysteine treatment in children and young adults with PWS on:
    - Other repetitive compulsive behaviour, e.g. nail biting and repeating or sorting and organizing behaviour
    - Cortisol levels, measured by hair cortisol levels
    - Quality of life
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    To be eligible to participate in this study, a subject must meet all the following criteria:
    - Prader-Willi syndrome (genetically proven)
    - Age between 6 - 24.99 years
    - Skin picking behaviour for at least one year, according the following DSM-5 criteria:
    1. Repeated picking of the skin and damaging it.
    2. Multiple attempts to stop the behaviour.
    3. Picking of the skin provokes significant stress or limitations in social, professional functioning, or limits other important areas of functioning.
    4. Picking of the skin is not attributed to the direct effect of drugs (e.g. cocaine) or to any other medical condition (e.g. scabies).
    5. Picking of the skin is not explained by another mental disorder (e.g. psychosis or body dysmorphic disorder).
    E.4Principal exclusion criteria
    A potential subject who meets any of the following criteria will be excluded from participation in this study:
    - Severe psychiatric problems.
    - Previous treatment with N-acetylcysteine in the last 3 months.
    - Current or recent (past 3 months) DSM-5 substance abuse or dependence.
    - Initiation of pharmacotherapy, psychotherapy, or behaviour therapy from a mental health perspective in the last 3 months.
    - Treatment with investigational medication or depot neuroleptics within 3 months.
    - Need for medication other than NAC with possible psychotropic effects or unfavourable interactions with NAC.
    - Asthma (given the possible worsening).
    - Active peptic ulcer considering the possible gastrointestinal- and vomiting side effects.
    E.5 End points
    E.5.1Primary end point(s)
    The main study parameter will be the difference of the changes in number and size of the skin picking lesions, assessed by counting and measuring the lesions in cm.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At start, 3 months, 6 months and 9 months.
    E.5.2Secondary end point(s)
    Change in:
    - Skin picking behavior, measured with the Skin Picking Symptom Assessment Scale
    - Repetitive compulsive behaviour, measured by the Repetitive Behaviour Scale questionnaire
    - Hair cortisol levels
    - Quality of life, measured with the Pediatric Quality of Life Inventory
    - Safety parameters (laboratory parameters and medical assessments)
    E.5.2.1Timepoint(s) of evaluation of this end point
    At start, 3 months, 6 months and 9 months.
    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) Yes
    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 Yes
    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 treatment
    E.8.2.4Number of treatment arms in the trial2
    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 years0
    E.8.9.1In the Member State concerned months9
    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: 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) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 7
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    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
    Children and young adults with intellectual disability (IQ 50-85)
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state35
    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)
    If a subject had positive effects of treatment, we will aim to continue treatment in this subject
    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 Decision2020-08-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-04-06
    P. End of Trial
    P.End of Trial StatusOngoing
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