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    EudraCT Number:2016-004223-23
    Sponsor's Protocol Code Number:NI-0501-06
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-01-25
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2016-004223-23
    A.3Full title of the trial
    A pilot, open-label, single arm, multicenter study to evaluate safety, tolerability, pharmacokinetics and efficacy of intravenous administrations of NI-0501, an anti-interferon gamma (anti-IFNγ) monoclonal antibody, in patients with systemic Juvenile Idiopathic Arthritis (sJIA) developing Macrophage Activation Syndrome/secondary HLH (MAS/sHLH)
    Studio pilota, aperto, a braccio singolo, multicentrico per valutare sicurezza, tollerabilita’, farmacocinetica ed efficacia di somministrazioni endovenose di NI-0501, anticorpo monoclonale anti-interferone gamma (anti-IFNγ) in pazienti affetti da artrite giovanile idiopatica sistemica (sJIA) che sviluppano sindrome da attivazione macrofagica / linfoistiocitosi emofagocitica secondaria (MAS/sHLH)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate a new medication, NI-0501, in children with the disease systemic Juvenile Idiopathic Arthritis (sJIA) developing Macrophage Activation Syndrome/secondary Hemophagocytic Lymphohistiocytosis (MAS/sHLH)
    Studio per valutare un nuovo farmaco, NI-0501, in bambini affetti dalla malattia Artrite Giovanile Idiopatica Sistemica (sJIA) che sviluppano Sindrome da Attivazione Macrofagica / linfoistiocitosi emofagocitica secondaria (MAS/sHLH)
    A.4.1Sponsor's protocol code numberNI-0501-06
    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 SponsorNovimmune SA
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportNovimmune SA
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovimmune SA
    B.5.2Functional name of contact pointClinical Science
    B.5.3 Address:
    B.5.3.1Street Address14 Chemin des Aulx
    B.5.3.2Town/ cityPlan-les-Ouates
    B.5.3.3Post code1228
    B.5.5Fax number+41228397142
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/10/749
    D.3 Description of the IMP
    D.3.2Product code NI-0501
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous 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. 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 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 Yes
    D. medicinal product typeMonoclonal antibody
    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
    Macrophage activation syndrome / Secondary hemophagocytic lymphohistiocytosis (MAS/sHLH) in patients with Systemic Juvenile Idiopathic Arthritis (sJIA)
    Sindrome da attivazione macrofagica / linfoistiocitosi emofagocitica secondaria (MAS/sHLH) in pazienti con Artrite giovanile idiopatica sistemica (sJIA)
    E.1.1.1Medical condition in easily understood language
    A rare, life-threatening condition, characterized by uncontrolled hyper-inflammation on the background of a rheumatologic disease.
    Malattia rara, pericolosa per la vita, caratterizzata da una infiammazione intensa incontrollata, che si sviluppa nel contesto di una malattia reumatica
    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 19.1
    E.1.2Level LLT
    E.1.2Classification code 10053867
    E.1.2Term Macrophage activation syndrome
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • To describe the pharmacokinetic profile of NI-0501 in sJIA patients with MAS
    •To confirm the proposed dosing regimen of NI-0501
    •To evaluate the safety and tolerability profile of intravenous administrations of NI-0501
    •To preliminary assess the efficacy of NI-0501
    •To assess the levels of relevant biomarkers
    •To assess the immunogenicity of NI-0501
    •Descrivere il profilo famacocinetico di NI-0501
    •Confermare il regime terapeutico proposto per NI-0501
    •Valutare il profilo di sicurezza e tollerabilità di somministrazioni endovenose (e.v.) di NI-0501
    •Valutare preliminarmente l’efficacia di NI-0501
    •Valutare i livelli di rilevanti biomarcatori
    •Valutare l’immunogenicita’ di NI-0501
    E.2.2Secondary objectives of the trial
    Not applicable
    Non applicabile
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Patients of both genders, aged <18 years
    •Confirmed sJIA or high presumption of sJIA.
    •Diagnosis of active MAS confirmed by the treating rheumatologist
    •Patient presenting an inadequate response to high dose i.v. glucocorticoid treatment
    •Informed consent provided by the patient (as required by local law), or by the patient’s legally authorized representative(s) with the assent of patients who are legally capable of providing it, as applicable
    •Pazienti di entrambi i sessi di eta’ <18 anni
    •Diagnosi di sJIA, precedentemente confermata o altamente probabile.
    •Diagnosi di MAS attiva, confermata dal reumatologo curante
    •Risposta inadeguata a terapia e.v. con corticosteroidi ad alto dosaggio
    •Consenso informato firmato dal paziente (secondo legislazione locale), o dal/dai suo/suoi tutore/i legale, unitamente all’assenso del paziente che sia legalmente in grado di fornirlo, se applicabile
    E.4Principal exclusion criteria
    •Diagnosis of suspected or confirmed primary HLH or HLH consequent to a neoplastic disease.
    •Patients treated with Tocilizumab, Canakinumab or TNF inhibitors within 5 times of their defined half-life.
    •Active mycobacteria (typical and atypical), Histoplasma Capsulatum, Shigella, Salmonella, Campylobacter and Leishmania infections.
    •Clinical suspicion of latent tuberculosis.
    •Positive serology for HIV antibodies.
    •Presence of malignancy.
    •Receipt of a BCG vaccine within 12 weeks prior to screening.
    •Receipt of live or attenuated live vaccines (other than BCG) within 6 weeks prior to screening.
    •Diagnosi, confermata o sospetta, di HLH primaria o di HLH secondaria ad una patologia neoplastica.
    •Trattamento con Tocilizumab, Canakinumab, inibitori del TNF nel periodo temporale di 5 volte la loro emivita.
    •Infezioni micobatteriche attive (tipiche ed atipiche), infezioni attive da Histoplasma Capsulatum, Shigella, Salmonella, Campylobacter e Leishmania.
    •Sospetto clinico di tubercolosi latente
    •Serologia positiva agli anticorpi anti-HIV.
    •Patologie neoplastiche maligne
    •Vaccinazione antitubercolare (bacillo di Calmette e Guérin, BCG) nelle 12 settimane antecedenti la visita di screening.
    •Vaccinazione per mezzo di agenti vivi o attenuati (diversi dal BCG) nelle 6 settimane prima della visita di screening
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability:
    •Incidence, severity, causality and outcomes of AEs (serious and non-serious), with particular attention being paid to infections.
    •Evolution of laboratory parameters, in particular CBC, LFTs, inflammatory markers (ferritin and CRP) and coagulation parameters.
    •Number of patients withdrawn from the study due to safety reasons.

    Pharmacokinetics and pharmacodynamics:
    •PK profile of NI-0501.
    •Levels of circulating free IFNγ at predose, and total IFNγ after initiation of NI-0501.
    •Levels of the main IFNγ-induced chemokines and other potential disease biomarkers.
    •Levels (if any) of circulating antibodies against NI-0501 to determine immunogenicity.
    Sicurezza e tollerabilita’:
    •Incidenza, severita’, causalita’ ed esito degli eventi avversi (AEs, seri e non-seri) con attenzione particolare alle infezioni.
    •Evoluzione dei valori di laboratorio, in particolare emocromo completo, test di funzionalita’ epatica, markers infiammatori (ferritina e PCR) e parametri di coagulazione
    •Numero di pazienti ritirati dallo studio per ragioni di sicurezza.

    Farmacocinetica e Farmacodinamica:
    •Profilo farmacocinetico di NI-0501.
    •Livelli di IFNγ circolante libero predose, ed IFNγ totale dopo somministratzione di NI-0501.
    •Livelli delle principali chemochine indotte dall’ IFNγ e di altri potenziali biomarcatori della malattia.
    •Livelli (se presenti) di anticorpi anti-NI-0501 al fine di determinarne l’immunogenicita’.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At multiple timepoints over 8 weeks
    A diversi tempi di osservazione nelle 8 settimane dello studio
    E.5.2Secondary end point(s)
    •Number of patients achieving MAS remission by Week 8 after initiation of NI-0501 treatment.
    •Time to MAS remission.
    •Number of patients for whom at any time during the study glucocorticoids can be tapered i) to the same (or lower) dose being administered before the occurrence of MAS ((in those patients who are already treated for sJIA) or ii) by 50% (or less) of the dose administered at NI-0501 treatment start (in those patients who present with MAS at sJIA onset).
    •Time to glucocorticoids tapering (as above described).
    •Survival time.
    •Number of patients withdrawn from the study due to lack of efficacy.
    •Numero di pazienti nei quali verra’ raggiunta la remissione della MAS entro la settimana 8 dall’inizio del trattamento con NI-0501.
    •Tempo al raggiungimento della remissione della MAS.
    •Numero di pazienti per i quali, in qualunque momento dello studio, sia possibile ridurre la dose di glucocorticoidi i) alla stessa dose (o inferiore) somministrata prima dell’insorgenza della MAS (nei pazienti gia’ in terapia per la sJIA) oppure ii) di almeno il 50% rispetto alla dose somministrata all’inizio del trattamento con NI-0501 (nei pazienti in cui la MAS rappresenta la prima manifestazione della sJIA).
    •Tempo alla riduzione della dose di glucocorticoidi (come descritto sopra).
    •Analisi di sopravvivenza.
    •Numero di pazienti che escono dallo studio per mancanza di efficacia

    E.5.2.1Timepoint(s) of evaluation of this end point
    At multiple timepoints over 8 weeks
    A diversi tempi di osservazione nelle 8 settimane dello studio
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    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 No
    E.8.1.1Randomised No
    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 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 No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 EEA7
    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
    Ultima visita dell’ultimo paziente
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    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: 5
    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) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F. of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 2
    F.1.2Adults (18-64 years) No
    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 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. of subjects incapable of giving consent
    Pediatric population
    Popolazione pediatrica
    F.3.3.7Others Yes
    F. of other specific vulnerable populations
    Pediatric population
    Popolazione pediatrica
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 5
    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)
    Patients will receive standard of care treatment used at the study center
    I pazienti riceveranno i trattamenti standard utilizzati dal centro
    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 Decision2017-04-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-12-14
    P. End of Trial
    P.End of Trial StatusCompleted
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