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    Summary
    EudraCT Number:2016-004223-23
    Sponsor's Protocol Code Number:NI-0501-06
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-06-09
    Trial results View 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 ConcernedSpain - AEMPS
    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)
    Estudio piloto abierto, multicéntrico y de un solo grupo para evaluar la seguridad, tolerabilidad, farmacocinética y eficacia de administraciones intravenosas de NI-0501, un anticuerpo monoclonal anti-interferón gamma (anti-IFNγ), en pacientes con artritis idiopática juvenil de inicio sistémico (AIJiS) que desarrollan síndrome de activación del macrófago/ HLH secundaria (SAM/HLHs).
    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)
    Estudio para investigar una nueva medicacion, NI-0501, en niños con la enfermedad artritis idiopática juvenil (AIJiS) que desarrollan syndrome de activacion del macrofago/HLH secundaria (SAM/HLHs)
    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.1.3.4CountrySwitzerland
    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.4.2CountrySwitzerland
    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.5.3.4CountrySwitzerland
    B.5.5Fax number+41228397142
    B.5.6E-mailNI-0501.clinscience@novimmune.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.2.5The 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.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEmapalumab
    D.3.9.2Current sponsor codeNI-0501
    D.3.9.3Other descriptive nameNI-0501
    D.3.9.4EV Substance CodeSUB32461
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 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
    Macrophage activation syndrome / Secondary hemophagocytic lymphohistiocytosis (MAS/sHLH) in patients with Systemic Juvenile Idiopathic Arthritis (sJIA)
    Síndrome de activación de macrofagos / linfohistiocitosis hemofagocítica secundaria (MAS/sHLH) en pacientes con artritis idiopática juvenile sistesmica (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.
    Una afeccion rara y potencialmente mortal, caracterizada por una hiperinflamacion incontrolada en el fondo de una enfermedad reumatologica.
    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 20.0
    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
    •Describir el perfil de farmacocinética (FC) de NI-0501 en pacientes con AIJiS con SAM.
    •Confirmar la pauta posológica propuesta de NI-0501.
    •Evaluar el perfil de seguridad y tolerabilidad de administraciones intravenosas de NI-0501.
    •Evaluar de forma preliminar la eficacia de NI-0501.
    •Evaluar los niveles de biomarcadores relevantes.
    •Evaluar la inmunogenicidad de NI-0501.
    E.2.2Secondary objectives of the trial
    Not applicable
    No aplica
    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.
    •Pacientes de ambos sexos con una edad < 18 años.
    •AIJiS confirmada o sospecha elevada de AIJiS.
    •Diagnóstico de SAM activo confirmado por el reumatólogo responsable del tratamiento.
    •Paciente que presenta una respuesta inadecuada al tratamiento i.v. con glucocorticoides a dosis elevadas.
    inicio del tratamiento i.v. con glucocorticoides a dosis elevadas.
    •Consentimiento informado proporcionado por el paciente (según se requiera según la normativa local) o por el(los) representante(s) legal(es) del paciente con el asentimiento de los pacientes que estén legalmente capacitados para otorgarlo, según proceda.
    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.
    •Diagnóstico de sospecha o confirmación de HLH primario o HLH debido a una enfermedad neoplásica.
    •Pacientes tratados con tocilizumab, canakinumab o inhibidores del FNT durante un plazo correspondiente a 5 veces su semivida definida.
    •Infecciones por micobacterias activas (típicas y atípicas), Histoplasma Capsulatum, Shigella, Salmonella, Campylobacter y Leishmania .
    •Sospecha clínica de tuberculosis latente.
    •Serología positiva para anticuerpos frente al VIH.
    •Presencia de neoplasia maligna.
    •Haber recibido una vacuna de BCG en las 12 semanas anteriores a la selección.
    •Haber recibido vacunas vivas o vivas atenuadas (diferentes de BCG) en las 6 semanas anteriores a la selección.
    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.
    La seguridad y la tolerabilidad :
    •Incidencia, gravedad, relación causal y resultado de los AA (graves y no graves), prestando especial atención a las infecciones.
    •Evolución de los parámetros analíticos, especialmente el hemograma completo, las PFH, los marcadores inflamatorios (ferritina y PCR) y los parámetros de coagulación.
    •Número de pacientes retirados por motivos de seguridad.
    Farmacocineticos y Farmacodinámicos:
    • Perfil PK de NI-0501
    •Niveles de IFNγ libre circulante antes de la dosis y de IFNγ total tras el cominezo con NI-0501
    •Niveles (en caso de que haya) de anticuerpos circulantes contra NI-0501 para determinar la inmunogenicidad.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At multiple timepoints over 8 weeks
    E.5.2Secondary end point(s)
    Efficacy:
    •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.
    Eficacia:
    •Número de pacientes que alcanzan remisión del SAM en la semana 8 después del comienzo del tratamiento con NI-0501.
    •Tiempo hasta la remisión del SAM.
    •Número de pacientes en los que pueden reducirse progresivamente los glucocorticoides en cualquier momento durante el estudio i) hasta la misma dosis (o inferior) que se administraba antes de la aparición del SAM (en aquellos pacientes que ya reciben tratamiento para el AIJiS) o ii) hasta el 50 % (o menos) de la dosis administrada al inicio del tratamiento con NI-0501 (en aquellos pacientes que presentan SAM al inicio de la AIJiS).
    •Tiempo hasta la reducción de la dosis de glucocorticoides (tal como se ha definido anteriormente).
    •Tiempo de supervivencia.
    •Número de pacientes retirados del estudio por falta de eficacia.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At multiple timepoints over 8 weeks
    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.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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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
    LVLS
    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.1.1.4.1Number of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number 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.3.3.6.1Details of subjects incapable of giving consent
    Pediatric population
    Población pediátrica
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Pediatric population
    poblacion pediatrica
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    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
    Los pacientes recibirán el tratamiento estandar utilizado en el centro del estudio
    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 Decision2017-09-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-09-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-05-19
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