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    Summary
    EudraCT Number:2020-004589-21
    Sponsor's Protocol Code Number:CLNP023B12301
    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:2021-08-11
    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 number2020-004589-21
    A.3Full title of the trial
    A multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in complement 3 glomerulopathy
    Estudio multicéntrico, aleatorizado, doble ciego, de grupos paralelos y controlado con placebo para evaluar la eficacia y la seguridad de iptacopán (LNP023) en la glomerulopatía C3.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of efficacy and safety of iptacopan in complement 3 glomerulopathy patients
    Estudio de la eficacia y la seguridad de iptacopán en pacientes con glomerulopatía C3
    A.4.1Sponsor's protocol code numberCLNP023B12301
    A.7Trial is part of a Paediatric Investigation Plan Yes
    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 vía de les Corts Catalanes, 764
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08013
    B.5.3.4CountrySpain
    B.5.4Telephone number+3493 0353036
    B.5.5Fax number+3493 2479903
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/18/2104
    D.3 Description of the IMP
    D.3.1Product nameiptacopan
    D.3.2Product code LNP023
    D.3.4Pharmaceutical form Capsule, hard
    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 INNiptacopan
    D.3.9.2Current sponsor codeLNP023
    D.3.9.3Other descriptive name LNP023 HYDROCHLORIDE SALT
    D.3.9.4EV Substance CodeSUB180361
    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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    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
    complement 3 glomerulopathy
    glomerulopatía C3
    E.1.1.1Medical condition in easily understood language
    Kidney disorder
    trastorno renal
    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 PT
    E.1.2Classification code 10077827
    E.1.2Term C3 glomerulopathy
    E.1.2System Organ Class 10038359 - Renal and urinary 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 demonstrate the superiority of iptacopan compared to placebo in reducing proteinuria.
    Demostrar la superioridad de iptacopán en comparación con placebo en la reducción de la proteinuria
    E.2.2Secondary objectives of the trial
    - To demonstrate the superiority of iptacopan vs. placebo in improving eGFR.
    - To demonstrate the superiority of iptacopan vs. placebo in the proportion of participants who achieved a composite renal endpoint.
    - To demonstrate the effect of iptacopan vs placebo in reducing glomerular inflammation in the kidney.
    - To assess the effect of iptacopan compared to placebo in improvement of patient reported fatigue.
    - To evaluate the safety and tolerability of iptacopan compared to placebo
    - Demostrar la superioridad de iptacopán frente a placebo en la mejora la TFGe
    - Demostrar la superioridad de iptacopán frente a placebo en el porcentaje de participantes que hayan alcanzado la variable renal compuesta
    - Demostrar el efecto de iptacopán frente a placebo en la reducción de la inflamación glomerular en el riñón
    - Evaluar el efecto de iptacopán en comparación con placebo en la mejora de la fatiga
    comunicada por el paciente
    - Evaluar la seguridad y la tolerabilidad de iptacopán en comparación con placebo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male and female participants age >/= 18 and </= 60 years at screening.
    -Diagnosis of C3G as confirmed by renal biopsy within 12 months prior to enrollment
    - Prior to randomization, all participants must have been on a maximally recommended or tolerated dose of an angiotensin converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB) for at least 90 days. The doses of other antiproteinuric medications including mycophenolic acids, corticosteroids, and mineralocorticoid receptor antagonists should be stable for at least 90 days prior to randomization.
    - Reduced serum C3 (defined as less than 0.85 x lower limit of the central laboratory normal range) at Screening.
    - UPCR >/= 1.0 g/g sampled from the first morning void urine sample at Day -75 and Day -15.
    - Estimated GFR (using the CKD-EPI formula) or measured GFR >/= 30 ml/min/1.73m2 at Screening and Day -15.
    - Vaccination against Neisseria meningitidis infection prior to the start of study treatment. Vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given, if available and according to local regulations.
    - Participantes de ambos sexos entre >/=18 años y </=60 años en la selección
    - Diagnóstico de GC3 confirmada por biopsia renal durante los 12 meses previos
    al reclutamiento
    - Antes de la aleatorización, todos los participantes deben haber tomado una
    dosis máxima recomendada o tolerada de un inhibidor de la enzima
    convertidora de la angiotensina (IECA) o de un antagonista de los receptores
    de la angiotensina (ARA) durante al menos 90 días. Las dosis de otros fármacos
    antiproteinúricos, incluyendo ácidos micofenólicos, corticosteroides y
    antagonistas de los receptores mineralocorticoideos, deberían ser estables
    durante al menos 90 días antes de la aleatorización.
    - C3 en suero reducido (definido como menos de 0,85 x límite inferior del intervalo normal del laboratorio central) en la selección.
    - CPC en orina ≥1,0 g/g tomado de una muestra de la primera micción de la mañana del día -75 y el día -15.
    - TFG estimada (utilizando la fórmula CKD-EPI) o TFG medida >/=30 ml/min/1,73 m2 en la selección y el día -15.
    - Vacuna contra la infección por Neisseria meningitidis antes de comenzar el
    tratamiento del estudio. Se deberá administrar la vacuna contra las infecciones por Streptococcus
    pneumoniae y Haemophilus influenzae, si están disponibles y según la
    normativa local
    E.4Principal exclusion criteria
    - Participants who have received any cell or organ transplantation, including kidney transplantation.
    - Rapidly progressive crescentic glomerulonephritis defined as a 50% decline in the eGFR within 3 months with renal biopsy findings of glomerular crescent formation seen in at least 50% of glomeruli.
    - Renal biopsy showing interstitial fibrosis/tubular atrophy (IF/TA) of more than 50%.
    - Monoclonal gammopathy of undetermined significance (MGUS) confirmed by the measurement of serum free light chains or other investigation as per local standard of care.
    - Participants with an active systemic bacterial, viral or fungal infection within 14 days prior to study treatment administration.
    - The presence of fever >/= 38°C (100.4°F) within 7 days prior to study treatment administration.
    - A history of recurrent invasive infections caused by encapsulated organisms, e.g., N. meningitidis and S. pneumoniae.
    - The use of inhibitors of complement factors (e.g., Factor B, Factor D, C3 inhibitors, anti Complement 5 (C5) antibodies, C5a receptor antagonists) within 6 months prior to the Screening visit.
    - The use of immunosuppressants (except mycophenolic acids),
    cyclophosphamide or systemic corticosteroids at a dose >7.5 mg/day (or equivalent for a similar medication) within 90 days of study drug administration.
    - Participantes que hayan recibido cualquier trasplante de células o de órganos,
    incluyendo trasplante de riñón.
    - Glomerulonefritis rápidamente progresiva con formación de semilunas (definida
    como una disminución del 50 % en la TFGe en 3 meses) con hallazgos en la
    biopsia renal de formación de semilunas glomerulares en al menos un 50 % de
    los glomérulos.
    - Biopsia renal que muestre fibrosis intersticial/atrofia tubular (FI/AT) de más del
    50 %.
    - Gammapatía monoclonal de significado indeterminado (GMSI) confirmada
    mediante la medición de cadenas ligeras libres en suero u otros medios
    siguiendo el tratamiento de referencia local.
    - Participantes con infección bacteriana, vírica o por hongos activa y sistémica
    durante los 14 días anteriores a la administración del tratamiento del estudio
    - Presencia de fiebre >/=8 °C durante los 7 días anteriores a la administración del
    tratamiento del estudio.
    - Antecedentes de infecciones invasivas recurrentes causadas por organismos
    encapsulados, p. ej., meningococo o neumococo.
    - Uso de inhibidores de factores del complemento (p. ej., factor B, factor D,
    inhibidores C3, anticuerpos anticomplemento 5 (C5), antagonistas de los
    receptores C5a) durante los 6 meses anteriores a la visita de selección.
    - Uso de inmunosupresores (excepto ácidos micofenólicos), ciclofosfamida o
    corticosteroides sistémicos a una dosis >7,5 mg/día (o equivalente para una
    medicación similar) durante los 90 días anteriores a la administración del
    fármaco del estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Log-transformed ratio to baseline in UPCR (sampled from a 24-hour urine collection)
    Cociente transformado logarítmicamente del cociente proteína en orina (tomado de una recogida de orina de 24 horas) desde la basal.
    E.5.1.1Timepoint(s) of evaluation of this end point
    6 months
    6 meses
    E.5.2Secondary end point(s)
    - Change from baseline in eGFR
    - A participant meets the requirements of the composite renal endpoint if he/she satisfies: (1) a stable or improved eGFR compared to the baseline visit (</=15% reduction in eGFR), and (2) a >/=50% reduction in UPCR compared to the baseline visit.
    - Change from baseline in disease total activity score in a renal biopsy.
    - Change in the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT Fatigue) score.
    - Occurrence of clinically significant vital signs, ECGs, and safety laboratory measurements, as well as adverse events (AEs), AEs of special interest, and study drug discontinuation due to an AE.
    -Cambio en la TFGe desde la basal
    -Un participante cumple los requisitos de la variable renal compuesta si cumple los siguientes criterios a los 6 meses: (1) una TFGe estable o mejorada respecto a la visita basal (reducción </=15 % en la TFGe) y (2) una reducción >/=50 % en el CPC en orina respecto a la visita basal
    -Cambio en la puntuación total de la actividad de la enfermedad en una biopsia renal desde la basal.
    -Cambio en la puntuación obtenida en la Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue).
    -Valoración las constantes vitales clínicamente significativas, los electrocardiogramas (ECG) y los valores analíticos de seguridad , así como los acontecimientos adversos (AA), los AA de especial interés y la discontinuación del fármaco del estudio debido a un AA (o a cualquier problema de seguridad).
    E.5.2.1Timepoint(s) of evaluation of this end point
    6 months
    6 meses
    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 No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    tolerability
    tolerabilidad
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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
    Brazil
    Canada
    China
    India
    Israel
    Japan
    Turkey
    United States
    Belgium
    France
    Germany
    Italy
    Netherlands
    Spain
    Switzerland
    United Kingdom
    Czechia
    Argentina
    Greece
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days24
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    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: 68
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 28
    F.4.2.2In the whole clinical trial 68
    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)
    Participants will have the option to continue open-label iptacopan treatment by transitioning to the C3G Extension study (protocol CLNP023B12001B).
    Los participantes tendrán la opción de continuar con el tratamiento con
    iptacopán abierto pasando al estudio de GC3 de extensión (protocolo CLNP023B12001B).
    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 Decision2021-08-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusOngoing
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