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    Summary
    EudraCT Number:2019-001220-35
    Sponsor's Protocol Code Number:RC31-19-0045
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-04-23
    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 ConcernedFrance - ANSM
    A.2EudraCT number2019-001220-35
    A.3Full title of the trial
    A randomized double-blinded pilot study of the efficacy and safety of dupilumab versus placebo in patients with Netherton syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A pilot study of the efficacy and safety of dupilumab versus placebo in patients with Netherton syndrome
    A.3.2Name or abbreviated title of the trial where available
    NS-Dupi
    A.4.1Sponsor's protocol code numberRC31-19-0045
    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 SponsorCHU de Toulouse
    B.1.3.4CountryFrance
    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 supportSANOFI AVENTIS
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCHU de Toulouse
    B.5.2Functional name of contact pointALGANS
    B.5.3 Address:
    B.5.3.1Street Address2, rue Viguerie
    B.5.3.2Town/ cityToulouse
    B.5.3.3Post code31059
    B.5.3.4CountryFrance
    B.5.4Telephone number+330561777204
    B.5.5Fax number+330561778411
    B.5.6E-mailalgans.n@chu-toulouse.fr
    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 Dupixent
    D.2.1.1.2Name of the Marketing Authorisation holderSANOFI AVENTIS
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameDUPILUMAB
    D.3.2Product code SAR231893
    D.3.4Pharmaceutical form Suspension for injection in pre-filled syringe
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous 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 No
    D.3.11.9Recombinant medicinal product Yes
    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 placeboSuspension for injection in pre-filled syringe
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Netherton syndrome
    E.1.1.1Medical condition in easily understood language
    Netherton syndrome
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10062909
    E.1.2Term Netherton's 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 assess the effect of dupilumab versus placebo on the severity of the disease in patients with moderate to severe NS as determined by the evolution at week 16 vs. baseline using a specific disease severity score (NASA).
    E.2.2Secondary objectives of the trial
    - To assess the effect of dupilumab versus placebo on the evolution of severity of the disease in patients with moderate to severe NS at each visit versus baseline, using a specific disease severity score and other clinical parameters.
    - Effect on the bacterial or viral cutaneous secondary infections
    - Effect on the reduction of dermocorticosteroid intake
    - Effect on the evolution of quality of life at week 16 and week 28 versus baseline.
    - Effect on the evolution of systemic Th2 sensitization (total IgE blood levels and specific IgE) at week 16 versus baseline.
    - Effect on the evolution of skin inflammation on skin biopsies at week 16 versus baseline.
    - Effect on the evolution of protease activity on skin biopsies at week 16 versus baseline.
    - Effect on the evolution of microbiome analysis at week 16 versus baseline.
    - Effect on the evolution of the TEWL (transepidermal water loss), at week 16 versus baseline.
    - To assess the safety during the study period.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Adult patients (≥18 years) affiliated to a social insurance protection regimen.
    -Clinical diagnosis of NS and absent or marked reduction of LEKTI staining.
    -Moderate to severe forms: NASA (Netherton Area Severity Assessment score) score ≥ 5/12 at inclusion.
    -Patients able to understand the study procedures including the ability to complete patient-based self-assessment questionnaires.
    -Patients who agree to sign the written informed consent.
    E.4Principal exclusion criteria
    - Hypersensitivity to dupilumab or its excipients.
    - Modification of the usual treatment within 2 weeks before inclusion.
    - Treatment with topical calcineurin inhibitors 1 week before inclusion.
    - Treatment with oral immunosuppressant , oral retinoids or phototherapy within 4 weeks before inclusion.
    - Treatment with immunomodulating biologics 16 weeks before inclusion.
    - Treatment with another investigational drug within 8 weeks before inclusion.
    - Treatment with a systemic antibiotic within 1 week before inclusion.
    - Active skin infection requiring the use of a systemic therapy within 2 weeks before the inclusion.
    - Any other condition that according to the investigator will impair the ability to evaluate treatment effect.
    - Known or suspected history of immunosuppression, including history of invasive opportunistic infections
    - Current infections including infection with helminthes.
    - Pregnant or breastfeeding women, or women planning to become pregnant or breastfeed during the study.
    - Mental or physical incapacity to fill in the questionnaires.
    E.5 End points
    E.5.1Primary end point(s)
    The severity of the disease will be evaluated by measuring the evolution at week 16 vs. baseline of the Netherton Area Severity Assessment score (NASA).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 0, Week 2, 4, 6, 8, 10, 12, 14, 16, 28.
    E.5.2Secondary end point(s)
    - Clinical efficacy
    - Bacterial or viral skin infections
    - Quantity of dermocorticosteroids
    - QOL : will be evaluated using the DLQI (Dermatology Life Quality Index (33)) and the IQoL-32 (specific ichthyosis QOL score (34)).
    - Systemic Th2 sensitization
    - Skin inflammation
    - Protease activity
    - Microbiome qualitative and quantitative analysis
    - TEWL: will be assessed using a Tewameter
    - Safety of dupilumab
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 0, Week 2, 4, 6, 8, 10, 12, 14, 16, 28.
    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 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) No
    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 Yes
    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 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 Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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 years1
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months3
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 24
    F.1.3Elderly (>=65 years) Information not present in EudraCT
    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 Yes
    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
    persons who are under tutorship
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 24
    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)
    None
    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 Decision2019-06-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-10-08
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-11-15
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