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    Summary
    EudraCT Number:2020-003596-17
    Sponsor's Protocol Code Number:CMAS825D12201
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-07-27
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2020-003596-17
    A.3Full title of the trial
    A three-period multicenter study, with a randomized withdrawal, double-blinded, placebo-controlled design in Period 2 to evaluate the clinical efficacy, safety and tolerability of MAS825 in NLRC4-GOF patients
    Estudio multicéntrico de tres periodos con un diseño de retirada aleatorizada, doble ciego y controlado con placebo en el periodo 2 para evaluar la eficacia clínica, la seguridad y la tolerabilidad de MAS825 en pacientes con mutación con ganancia de función en NLRC4
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to evaluate the efficacy and safety of MAS825 in NLRC4-GOF patients
    Estudio para evaluar la eficacia y la seguridad de MAS825 en pacientes con mutación con ganancia de función en NLRC4
    A.4.1Sponsor's protocol code numberCMAS825D12201
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04641442
    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 SponsorNovartis Farmacéutica, S.A.
    B.1.3.4CountrySpain
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Farmacéutica S.A.
    B.5.2Functional name of contact pointTrial Monitoring Organization (TMo)
    B.5.3 Address:
    B.5.3.1Street AddressGran Via de les Corts Catalanes, 764
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08013
    B.5.3.4CountrySpain
    B.5.4Telephone number+3490 0353036
    B.5.6E-maileecc.novartis@novartis.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameMAS825
    D.3.2Product code MAS825
    D.3.4Pharmaceutical form Concentrate for solution for injection/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 INNMAS825, INN not available
    D.3.9.3Other descriptive nameMAS825
    D.3.9.4EV Substance CodeSUB217990
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number1
    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 placeboSolution for injection/infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    NLRC4-GOF, AIFEC (autoinflammation with infantile enterocolitis)
    NLRC4-GOF, AIECI (autoinflamación con enterocolitis infantil)
    E.1.1.1Medical condition in easily understood language
    Autoinflammation with infantile enterocolitis
    Autoinflamación con enterocolitis infantil
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 23.1
    E.1.2Level PT
    E.1.2Classification code 10084306
    E.1.2Term Autoinflammation with infantile enterocolitis
    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 determine the efficacy of MAS825 in prevention of flares in NLRC4-GOF patients
    Determinar la eficacia de MAS825 en la prevención de brotes en pacientes con mutación GOF en NLRC4
    E.2.2Secondary objectives of the trial
    - To evaluate the safety and tolerability in patients with NLRC4-GOF
    - Evaluate the serological markers of MAS825
    - Evaluate efficacy of MAS825 to improve clinical status of NLRC4-GOF patients
    - Evaluate efficacy of MAS825 to achieve serological remission
    - Evaluate the effect of MAS825 on concomitant glucocorticoid administration
    - Evaluate effect of MAS825 on the time to first flare in patients with NLRC4-GOF
    - Evaluate the efficacy of MAS825 to improve signs and symptoms of NLRC4-GOF
    - Evaluate effect of MAS825 on patient reported outcomes in patients with NLRC4-GOF over time
    - Evaluar la seguridad y la tolerabilidad de MAS825 en pacientes con mutación GOF en NLRC4.
    -Evaluar marcadores serológicos de MAS825
    - Evaluar la eficacia de MAS825 para mejorar el estado clínico de los pacientes con mutación GOF en NLRC4.
    -Evaluar la eficacia de MAS825 para alcanzar remisión serológica.
    -Evaluar los efectos de MAS825 en la administración concomitante de glucocorticoides.
    -Evaluar el efecto de MAS825 en el tiempo hasta el primer brote en pacientes con mutación GOF en NLRC4.
    -Evaluar la eficacia de MAS825 en la mejora de los signos y síntomas de los pacientes con mutación GOF en NLRC4.
    -Evaluar el efecto de MAS825 en los resultados comunicados por el paciente con mutación GOF en NLRC4 a lo largo del tiempo.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Male and female patients weighing at least 3 kg
    2.Written informed consent by parent(s)/legal guardian(s) for the pediatric patients and assent by the pediatric patient (depending on local requirements) must be obtained before any study-specific
    assessment is performed. For adult patients, written informed consent by patients capable of giving consent, or, when the patient is not capable of giving consent, by his or her legal/authorized representative (if allowed according to local requirements).
    3.Patients with genetic diagnosis of NLRC4-GOF
    4.Clinical history and investigations consistent with autoinflammation with infantile enterocolitis (AIFEC/NLRC4-GOF)
    5.At first treatment, evidence of active disease
    1.Pacientes de ambos sexos que pesen por lo menos 3 kg
    2.Se debe obtener el consentimiento informado por escrito de los progenitores/tutores legales de los pacientes pediátricos y el asentimiento de los pacientes pediátricos (según los requisitos locales) antes de que se realice cualquier evaluación específica del estudio. Para los pacientes adultos, el consentimiento informado por escrito firmado por los pacientes que puedan dar su consentimiento, o, cuando el paciente no pueda dar su consentimiento, debe hacerlo su representante legal/autorizado (si está autorizado por los requisitos locales).
    3. Pacientes con diagnóstico genético de mutación GOF en NLRC4
    4.Historia clínica y pruebas relacionadas con autoinflamación con enterocolitis infantil (AIECI/mutación GOF en NLRC4)
    5.En el primer tratamiento, la evidencia de la enfermedad activa
    E.4Principal exclusion criteria
    1.History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or to any of the excipients.
    2.Signs and symptoms, in the judgment of the investigator, of clinically significant systemic recurrent and/or evidence of active bacterial, fungal, parasitic or viral infections.
    - COVID-19 specific: If in line with health and governmental authority guidance, it is highly recommended that testing to exclude COVID-19 using PCR or comparable approved methodology be completed within 1 week prior to first dosing.
    3.Any conditions or significant medical problems, which in the opinion of the investigator places the patient at unacceptable risk for MAS825 therapy
    4.Previous treatment with anti-rejection and/or immunomodulatory drugs within the past 28 days or 5 half-lives (whichever is the longer) for immunomodulatory therapeutic antibodies (or as listed in the prohibited medications section) prior to MAS825 treatment with the exceptions of glucocorticoids, cyclosporin and targeted binding or blocking therapies.
    5.A positive HIV test result at Screening. Evidence of prior testing within 3 months is sufficient.
    6.A positive Hepatitis B surface antigen (HBsAg) or Hepatitis C test result. Evidence of prior testing within 3 months is sufficient.
    7.Presence of tuberculosis infection as defined by a positive TB test at Screening. Evidence of prior testing within 3 months is sufficient.
    8.Live vaccinations within 1 month prior to MAS825 treatment, during the trial, and up to 3 months following the last dose.
    9.Female patients of child-bearing potential (or Tanner stage 2 or above) who are or might become sexually active, agree to use highly effective contraceptive methods to prevent pregnancy while on MAS825 therapy
    1. Antecedentes de hipersensibilidad a alguno de los fármacos del estudio o a fármacos de clases químicas similares o a cualquiera de sus excipientes.
    2. Signos y síntomas, según el investigador, de infecciones bacterianas, por hongos, parasitarias o víricas sistémicas recurrentes, y clínicamente significativas o evidencia de estas infecciones activas.
    - Acerca de la COVID-19: En consonancia con las pautas de las autoridades sanitarias y gubernamentales, se recomienda encarecidamente que se realicen pruebas para descartar COVID-19 mediante PCR o una metodología aprobada comparable durante la semana anterior a la primera dosis.
    3. Cualquier condición o problema médico significativo que según el criterio del investigador ponga al paciente en un riesgo inaceptable para el tratamiento con MAS825.
    4. Tratamiento anterior con fármacos antirrechazo o inmunomoduladores durante los últimos 28 días o 5 vidas medias (aquello que sea más largo) de los anticuerpos terapéuticos inmunomoduladores (o según lo enumerado en el apartado de medicación prohibida) antes del tratamiento con MAS825 con las excepciones de glucocorticoides, ciclosporina y o las terapias de bloqueo o unión.
    5.Resultado positivo de la prueba de VIH en la selección. Una evidencia de una prueba realizada durante los 3 meses anteriores es suficiente.
    6. Resultado positivo de la prueba del antígeno de superficie de la hepatitis B (HBsAg) o de la hepatitis C. Una evidencia de una prueba realizada durante los 3 meses anteriores es suficiente.
    7. Presencia de infección por tuberculosis según una prueba de TB positiva en la selección. Una evidencia de una prueba realizada durante los 3 meses anteriores es suficiente.
    8. Administración de vacunas vivas durante el mes anterior al tratamiento con MAS825, durante el ensayo y hasta los 3 meses posteriores a la última dosis.
    9. Pacientes con posibilidad de quedarse embarazadas (o en estadio 2 de Tanner o superior) que son o podrían ser sexualmente activas, aceptar el uso de métodos anticonceptivos altamente eficaces para prevenir el embarazo mientras estén en tratamiento con MAS825.
    E.5 End points
    E.5.1Primary end point(s)
    Occurrence of disease flare in patients with MAS825 treated patients compared with placebo during Period 2 assessed by Physician's Global Assessment and inflammatory markers
    Aparición de brote de enfermedad en pacientes tratados con MAS825 en comparación con placebo durante el Período 2 siendo evaluado por la Evaluación global del médico y los marcadores inflamatorios
    E.5.1.1Timepoint(s) of evaluation of this end point
    Period 2
    Período 2
    E.5.2Secondary end point(s)
    - Number and severity of safety assessments and adverse events
    - Confirmation of serological markers of MAS825
    - PGA and inflammatory markers at Day 29, end of Period 1 and 2
    - Serological remission via inflammatory markers
    - Glucocorticoid therapy <0.2mg/kg by end of period 1
    - Time to first flare during period 2
    - Physician Severity Assessment of Disease Signs and Symptoms scale
    - Patient' / Parent's global assessment of disease activity (PPGA) scale
    - Número y gravedad de evaluaciones de seguridad y eventos adversos
    - Confirmación de marcadores serológicos de MAS825
    - PGA y marcadores inflamatorios en el Día 29, fin de los Períodos 1 y 2
    - Remisión serológica via marcadores inflamatorios
    -Terapia con glucocorticoides<0.2mg/kg al final del período 1
    - Tiempo al primer brote durante el período 2
    - Evaluación del médico de la escala gravedad de los signos y síntomas de la enfermedad.
    - Evaluación global de la escala paciente/progenitor (EGPP) de la actividad de la enfermedad
    E.5.2.1Timepoint(s) of evaluation of this end point
    Multiple time points up to End of Study visit
    Múltiples puntos temporales hasta visita de Fin de Estudio
    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 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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability, immunogenicity
    Tolerabilidad, inmunogenicidad
    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 No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Secuencial; retirada de la randomización
    Sequential; randomized withdrawal
    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 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 Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Japan
    United States
    France
    Italy
    Spain
    Czechia
    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
    último paciente última visita
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days16
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months4
    E.8.9.2In all countries concerned by the trial days9
    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: 7
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Yes
    F.1.1.2.1Number of subjects for this age range: 1
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 2
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 1
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 1
    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 patients
    Pacientes pediátricos
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 4
    F.4.2.2In the whole clinical trial 8
    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)
    Please see Protocol Section 6.1.4
    Por favor ver Sección 6.1.4 del Protocolo
    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 Decision2022-03-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-09-28
    P. End of Trial
    P.End of Trial StatusOngoing
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