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    The EU Clinical Trials Register currently displays   39231   clinical trials with a EudraCT protocol, of which   6427   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).
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    EudraCT Number:2019-002036-90
    Sponsor's Protocol Code Number:Uni-Koeln-3815
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-10-02
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedGermany - PEI
    A.2EudraCT number2019-002036-90
    A.3Full title of the trial
    A randomized, placebo-controlled phase IIa clinical trial to evaluate the efficacy and safety of subcutaneous Dupilumab in localized scleroderma
    Randomisierte, Placebo kontrollierte klinische Phase IIa Studie zur Bewertung der Wirksamkeit und Sicherheit von subkutanem Dupilumab bei zirkumskripter Sklerodermie
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to test effectiveness and safety of the drug Dupilumab in patients with localized scleroderma
    Studie zur Bewertung der Wirksamkeit und Sicherheit des Medikaments Dupilumab bei Patienten mit zirkumskripter Sklerodermie
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberUni-Koeln-3815
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04200755
    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 SponsorUniversity of Cologne
    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.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCTC Cologne
    B.5.2Functional name of contact pointProject Manager
    B.5.3 Address:
    B.5.3.1Street AddressGleueler Str. 269
    B.5.3.2Town/ cityCologne
    B.5.3.3Post code50935
    B.5.4Telephone number004922147888153
    B.5.5Fax number004922147888209
    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 No
    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 Dupilumab
    D.3.4Pharmaceutical form Solution for injection in pre-filled syringe
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDupilumab
    D.3.9.1CAS number 1190264-60-8
    D.3.9.3Other descriptive nameSAR231893
    D.3.9.4EV Substance CodeSUB88511
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 placeboSolution 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
    Localized scleroderma
    Zirkumskripte Sklerodermie
    E.1.1.1Medical condition in easily understood language
    Localized scleroderma
    Zirkumskripte Sklerodermie
    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 21.1
    E.1.2Level LLT
    E.1.2Classification code 10009206
    E.1.2Term Circumscribed scleroderma
    E.1.2System Organ Class 100000004859
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10018124
    E.1.2Term Generalized scleroderma
    E.1.2System Organ Class 100000004859
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10027979
    E.1.2Term Morphea
    E.1.2System Organ Class 100000004859
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate, in comparison with placebo, the efficacy of Dupilumab in patients with Morphea (plaque type) or Generalized localized scleroderma (affecting at least three anatomic sites).
    E.2.2Secondary objectives of the trial
    • To evaluate, in comparison with placebo, the effect of Dupilumab on:
    o Disease activity/skin damage score (mLoSSI/LoSDI) during treatment phase
    o Disease activity/skin damage score (mLoSSI/LoSDI) after end of treatment until 24 weeks after end of treatment
    o Disease activity/skin damage score (mLoSSI/LoSDI) of other existing (non-target) lesions and new lesions
    o The potential for an attenuated gene signature of localized scleroderma (RNA-seq, RT-qPCR)
    o The potential for an attenuated histology (quantity and quality of cellular composition, extracellular matrix composition, altered skin architecture)
    o On the quality of life of patients (DLQI questionnaire)
    • To evaluate the safety profile of Dupilumab (AEs, physical examination, vital signs, clinical laboratory evaluations including hematology, biochemistry and urine analysis)
    • To evaluate immunogenicity of Dupilumab treatment
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject is a male or female ≥18 years of age on the day the study informed consent is signed
    2. Out-patient status
    3. Caucasian
    4. Morphea (plaque type) or Generalized localized scleroderma (affecting at least three anatomic sites)
    5. At least one lesions with lilac ring (active phase of the disease);
    6. Activity of LS within the last 12 month (as defined by progression of size or new developing plaque)
    7. For women of childbearing potential: negative pregnancy test at Visit 1
    8. For women of childbearing potetnial: Use of effective method of contraceptionfrom 4 weeks prior to enrolment, throughout the study treatment until 12 weeks after the last IMP dose
    9. Written informed consent signed
    E.4Principal exclusion criteria
    1. Systemic immunosuppressive therapy or UV therapy less than 3 months before enrolment.
    2. Participation in another trial of IMPs or devices parallel to, or less than 6 months before or previous participation in this trial
    3. Pregnancy or breastfeeding mother
    4. Diagnosis of other significant chronic inflammatory or autoimmune disorders.
    5. Topical immunosuppressive therapy less than 1 month before enrollment
    6. Concurrent phototherapy
    7. Known infection with helminths (helminthose)
    8. Any condition or laboratory abnormality that, in the judgment of the investigator, would put the subject at unacceptable risk for participation in the study or may interfere with the assessments included in the study. E.g. uncontrolled psychiatric illness or history of clinical relevant drug abuse.
    9. Known hypersensitivity to any components of the IMP
    10. Treatment with a live (attenuated) vaccine within 3 months prior to enrollment
    11. Histroy of malignancy (except patients with complteels reated in situ carcinoma of the cervix, completely treated and resolved non-metastatic suamous or basal cell carcinoma or the skin)
    12. Known diagnosis of active tuberculosis or non-tuberculous mycobacterial infections or latens untreated tuberculosis unless it is well documented by a specialist that the patient has been adequately treated.
    13. Known diagnosis of HIV, HBV or HCV infection
    14. Reguklar use (more than 2 visits per week) or a tanning booth/parlor
    15. Known diagnosis of asthma
    E.5 End points
    E.5.1Primary end point(s)
    To determine the efficacy of Dupilumab treatment response of patients with LS will be assessed by multiple measures which have been developed and validated in previous clinical trials and which are used within the ENLS (European Network for Localized Scleroderma) for the assessment of patients with LS (7). Personnel involved in data acquisition during the trial will be specifically trained in the assessment procedures.
    At baseline visit (V1) a single existing lesion with the highest mLoSSI activity index will be defined as target lesion.
    Treatment response is determined by change in mLoSSI (size of lesion, erythema, skin thickening) or LoSDI (dermal athrophy, subcutaneous atrophy, dyspigmentation) of the target lesion; score reduction by 50% after 24 weeks (EoT V14) compared to baseline (V1) is defined as treatment response.
    E.5.1.1Timepoint(s) of evaluation of this end point
    EoT V14
    E.5.2Secondary end point(s)
    • mLoSSI of target lesion during treatment and during follow-up
    • LoSDI (dermal athrophy, subcutaneous atrophy, dyspigmentation) of target lesion during treatment, at EoT and during follow-up
    • Count of all existing non-target and new lesions on the entire integument during treatment, at EoT and during follow-up
    • mLoSSI and LoSDI of all existing non-target lesions and new lesions on the entire integument during treatment, at EoT and during follow-up
    • Gene signature of localized scleroderma after Dupilumab treatment: RNA-seq, RT-qPCR of tissue biopsies obtained from the lesion (lilac ring, optional: center) and healthy skin before (baseline visit V1) and after treatment (EoT V14)
    • Histology of localized scleroderma after Dupilumab treatment: tissue biopsies obtained from the lesion (lilac ring, optional: center) and healthy skin before (baseline visit/V1) and after treatment (EoT V14). Analysis of quantity and quality of cellular composition, extracellular matrix composition, altered skin architecture)
    • DermatoLogy Quality of life Index (DLQI)
    • Safety profile of Dupilumab:
    - Adverse events (AEs)/treatment-emergent adverse events (TEAEs)
    - Physical examination and body weight
    - Vital signs
    - Clinical laboratory evaluations including hematology, biochemistry
    • Anti-drug antibody levels (optional)
    • Anti-nuclear antibodies (ANAs) levels
    • Serum cytokine levels
    E.5.2.1Timepoint(s) of evaluation of this end point
    EoT V14 and/or FU visits V15 and V16
    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. 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 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 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 concerned3
    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
    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
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    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: 42
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state45
    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 end of IMP treatment the subject will be treated according to local standard of care. No additional care due to trial participation is expected to be required.
    Nach Abschluss der IMP-Behandlung wird der Patient nach dem lokalen Standard behandelt. Es wird erwartet, dass keine zusätzliche Behandlung aufgrund der Teilnahme an der Studie erforderlich ist.
    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 Decision2019-12-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-11-29
    P. End of Trial
    P.End of Trial StatusOngoing
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