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    Summary
    EudraCT Number:2021-000230-33
    Sponsor's Protocol Code Number:HYDROXYSSc
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-08-17
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2021-000230-33
    A.3Full title of the trial
    A DOUBLE BLIND, RANDOMIZED, PLACEBO-CONTROLLED, ADD-ON TRIAL EVALUATING EFFICACY AND SAFETY OF HYDROXYCHLOROQUINE IN EARLY SYSTEMIC SCLEROSIS (SSc)- HYDROXYSSc
    STUDIO CLINICO IN DOPPIO CIECO, RANDOMIZZATO, CONTROLLATO VERSO PLACEBO SULL'EFFICACIA E LA SICUREZZA DELL'ASSOCIAZIONE DELL'IDROSSICLOROCHINA CON LA TERAPIA STANDARD NELLA SCLEROSI SISTEMICA (ScS) PRECOCE - HYDROXYSSc
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    TRIAL EVALUATING EFFICACY AND SAFETY OF HYDROXYCHLOROQUINE VERSUS PLACEBO IN EARLY SYSTEMIC SCLEROSIS (SSc)
    STUDIO SULLA SICUREZZA E SULLA EFFICACIA DELL'IDROSSICLOROCHINA CONTRO PLACEBO NELLA SCLEROSI SISTEMICA (ScS) PRECOCE
    A.3.2Name or abbreviated title of the trial where available
    HYDROXYSSc
    HYDROXYSSc
    A.4.1Sponsor's protocol code numberHYDROXYSSc
    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 SponsorUMBERTO I - POLICLINICO DI ROMA
    B.1.3.4CountryItaly
    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 supportAIFA - Italian Medicines Agency
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationReumatologia-Dipartimento di Scienze Cliniche Internistiche, Anestesiologiche e Cardiovascolari- Policlinico Umberto I-
    B.5.2Functional name of contact pointScleroderma Clinic
    B.5.3 Address:
    B.5.3.1Street AddressViale del Policlinico 155
    B.5.3.2Town/ cityRoma
    B.5.3.3Post code00161
    B.5.3.4CountryItaly
    B.5.4Telephone number0649974641
    B.5.5Fax number064463534
    B.5.6E-mailsclerodermaclinic@gmail.com
    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.2.1.2Country which granted the Marketing AuthorisationItaly
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1963
    D.3 Description of the IMP
    D.3.1Product nameIdrossiclorochina solfato
    D.3.2Product code [N/A]
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNidrossiclorochina solfato
    D.3.9.1CAS number 118-42-3
    D.3.9.2Current sponsor codeidrossiclorochina solfato
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 placeboTablet
    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
    Systemic Sclerosis (SSc) is a rare and orphan disease (DPCM 12 gennaio 2017-GU SG n°65-S.O. n°15 - 18/03/2017), characterized by immunological, vascular and fibrotic abnormalities. The estimated incidence is 18 to 20 cases per million population year and a prevalence of 100 to 300 cases per million population. In Europe the prevalence rate is estimated around 200 per million while in the Italian population around 20000 persons suffer from this form of autoimmune disease.
    La Sclerosi Sistemica (ScS) è una malattia rara (DPCM 12 gennaio 2017-GU SG n ° 65-S.O. N ° 15 - 18/03/2017), caratterizzata da anomalie immunologiche e vascolari e da fibrosi diffusa. L'incidenza stimata è 18-20 casi per milione di abitanti all'anno e la prevalenza è 100-300 casi per milione di abitanti. In Europa il tasso di prevalenza è stimato intorno a 200 per milione mentre nella popolazione italiana circa 20000 persone soffrono di questa forma di malattia autoimmune.
    E.1.1.1Medical condition in easily understood language
    Systemic Sclerosis (SSc) is a rare chronic autoimmune disease characterized by thickening of the skin and / or internal organs and by alteration of the microcirculation.
    La Sclerosi Sistemica (ScS) è una malattia rara cronica autoimmune, caratterizzata indurimento ed ispessimento della cute e/o degli organi interni e dall’alterazione della microcircolazione.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10042953
    E.1.2Term Systemic sclerosis
    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 10042953
    E.1.2Term Systemic sclerosis
    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 10042953
    E.1.2Term Systemic sclerosis
    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 10042953
    E.1.2Term Systemic sclerosis
    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 10042953
    E.1.2Term Systemic sclerosis
    E.1.2System Organ Class 100000004859
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy and safety of hydroxychloroquine compared to placebo, at the oral dose of 6 mg/kg daily (up to 400 mg/day), in the treatment of early systemic sclerosis (SSc) associated to SSc standard therapies (immunosuppressive and/or vasoactive).
    Valutare l’efficacia e la sicurezza dell’ aggiunta alla terapia standard della ScS (immunosoppressiva e/o vasoattiva) dell’idrossiclorochina rispetto al placebo, alla dose di 6 mg/kg al giorno (fino a 400 mg/die), nel trattamento della sclerosi sistemica precoce (SSc).
    E.2.2Secondary objectives of the trial
    To evaluate the effectiveness of adding hydroxychloroquine to standard therapy for SSc on ESScGAI, capillaroscopic parameters and CSURI Index, VAS pain, Morning Stiffness (MS), FACIT Fatigue Index and Raynaud Condition Score (RCS); Assess the tolerability and variation of clinical, laboratory and biomarker parameters from baseline.
    Valutare l’efficacia dell’aggiunta dell’idrossiclorochina alla terapia standard per la ScS su ESScGAI, parametri capillaroscopici e CSURI Index, VAS dolore, Morning Stiffness (MS), FACIT Fatigue Index e Raynaud Condition Score (RCS); Valutare la tollerabilità e la variazione dei parametri clinici, di laboratorio e dei biomarcatori rispetto al basale.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Diagnosis of SSc according 2013 ACR/EULAR criteria for classification of SSc (van den Hoogen et al, 2013).
    2. Written Informed Consent (IC)(patient must be able and agree to sign an IC according ICH-GCP guidelines and local laws)
    3. Age >= 18 years
    4. Disease duration <= 5 years from the first non-Raynaud’s symptom
    5. Stable SSc standard treatment within 4 weeks prior to Screening visit.
    1. Diagnosi di Sclerosi Sistemica secondo i criteri classificativi 2013 ACR/EULAR (van den Hoogen et al, 2013).
    2. Consenso informato scritto (il paziente deve essere capace e deve essere d’accordo a firmare il consenso secondo le linee guida ICH-GCP e le leggi locali)
    3. Età >= 18 anni
    4. Durata di malattia <= 5 anni dal primo sintomo non-Raynaud
    5. Terapia standard per la SSc stabile entro le 4 settimane dalla visita di screening.
    E.4Principal exclusion criteria
    1. Known hypersensytivity to the study drug (active substance or excipients) or derivatives 4-aminoquinolines
    2. Age < 18 years
    3. Body weight < 45 kg
    4. Anticoagulant and/or antiplatelet therapy
    5. History of retynopathy and/or maculopathy
    6. History of periferic neuropathy
    7. History of severe miopathy (other than SSc related)
    8. History of hypoglycemia
    9. History of bradycardia (HR<50) or ventricular arrhythmias
    10. Deficiency of glucose-6-phosphate dehydrogenase
    11. Unstable SSc or SSc with end-stage organ involvement at Screening or Visit 1
    12. Pregnant or breast feeding women
    13. Other contraindicated clinical and / or laboratory conditions
    1. Allergia nota al farmaco in studio (sostanza attiva o eccipienti) o ai derivati 4-aminochinolonici
    2. Età < 18 anni
    3. Peso < 45 kg
    4. Terapia anticoagulante e/o antiaggregante
    5. Storia di maculopatia e/o retinopatia 6. Storia di neuropatia periferica
    7. Storia di severa miopatia (non correlata alla ScS)
    8. Storia di ipoglicemia
    9. Storia di bradicardia (FC<50), di aritmie ventricolari
    10. Deficit di glucosio-6 fosfato deidrogenasi
    11. ScS instabile o ScS con coinvolgimento d’organo terminale alla visita di screening o alla Visita 1.
    12. Gravidanza o allattamento
    13. Altre condizioni cliniche e/o di laboratorio controindicate
    E.5 End points
    E.5.1Primary end point(s)
    To evaluate efficacy of HCQ add-on compared to placebo by assessing changes from baseline of American College of Rheumatology Combined Response Index for Systemic Sclerosis (CRISS)(Khanna D 2009) at week 52.
    Primary Safety endpoint:
    Valutare l’efficacia dell’ aggiunta dell’idrossiclorochina rispetto al placebo sulla variazione dal baseline del American College of Rheumatology Combined Response Index for Systemic Sclerosis (CRISS)(Khanna D 2009)
    E.5.1.1Timepoint(s) of evaluation of this end point
    52 weeks
    52 settimane
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints: To evaluate efficacy of HCQ add-on by assessing change from baseline of:
    1. European Systemic Sclerosis Global Disease Activity Index (ESScGDAI) at week 52
    2. Visual Analogue Scale (VAS) for pain at weeks 26 and 52
    3. Morning stiffness (MS) duration (in minutes) at weeks 26 and 52
    4. The FACIT fatigue Index at week 26 and 52
    5. Raynaud Condition Score (RCS) scale at week 26 and 52
    6. Naifold Capillaroscopy (NC) main parameters and CSURI (Capillaroscopic Skin Ulcer Risk Index) at week 52; Safety of treatment in terms of treatment-emergent adverse events (TEAEs) associated with treatment with HCQ in combination with standard therapy for SSc and changes in vital signs, weight, body mass index, physical examinations, laboratory safety tests, electrocardiograms (ECGs); Exploratory biological endpoints: to evaluate the effects of HCQ add-on compared to placebo by assessing changes from baseline in CXCL4 the levels or CXCL4-DNA complexes and in IFN-I concentrations in peripheral blood of SSc patients.
    Valutare l’efficacia su ESScGAI, parametri capillaroscopici e CSURI Index a 52 settimane, VAS dolore, Morning Stiffness (MS), FACIT Fatigue Index e Raynaud Condition Score (RCS) a 26 e 52 settimane.; Sicurezza del trattamento in termini di treatment-emergent adverse events (TEAEs) associati con l’aggiunta dell’idrossiclorochina alla terapia standard per la SSc e di variazioni rispetto al basale dei parametri vitali, peso, body mass index, esame obiettivo, esami di laboratorio di sicurezza e dell'ECG.; Endpoint biologici esplorativi: valutare gli effetti dell’aggiunta dell’idrossiclorochina rispetto al placebo determinando le variazioni dal baseline dei livelli di CXCL4 o dei complessi CXCL4-DNA e della concentrazione di IFN-I nel sangue periferico dei pazienti con SSc
    E.5.2.1Timepoint(s) of evaluation of this end point
    26 e 52 weeks; 52 weeks; 52 weeks
    26 e 52 settimane; 52 settimane; 52 settimane
    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 No
    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 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 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 Information not present in EudraCT
    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
    The trial ends after 4 additional weeks of follow-up from LVLS
    La sperimentazione si conclude al termine delle 4 settimane di follow up aggiuntive dalla 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 months26
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months26
    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: 100
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 51
    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 state151
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 151
    F.4.2.2In the whole clinical trial 151
    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 4 weeks from the end of the of the study, patients will undergo a follow up visit to assess the safety endpoints.
    Dopo 4 settimane dal termine dello studio, i pazienti saranno sottoposti a una visita di follow-up per valutare gli endpoint di sicurezza.
    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 Decision2021-09-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-01-26
    P. End of Trial
    P.End of Trial StatusOngoing
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