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    Summary
    EudraCT Number:2020-003767-25
    Sponsor's Protocol Code Number:APL2-C3G-310
    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:2022-02-25
    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 number2020-003767-25
    A.3Full title of the trial
    A Phase 3, Randomized, Placebo-Controlled, Double-Blinded, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
    Estudio multicéntrico de fase 3, aleatorizado, controlado con placebo, doble ciego, para evaluar la eficacia y la seguridad del pegcetacoplán en pacientes con glomerulopatía C3 primaria o glomerulonefritis membranoproliferativa mediada por inmunocomplejos
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 study to Evaluate the Efficacy and Safety of Pegcetacoplan administered subcutaneously in Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
    Estudio de fase 3 para evaluar la eficacia y la seguridad del pegcetacoplán en pacientes con glomerulopatía C3 primaria o glomerulonefritis membranoproliferativa mediada por inmunocomplejos
    A.3.2Name or abbreviated title of the trial where available
    VALIANT
    A.4.1Sponsor's protocol code numberAPL2-C3G-310
    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 SponsorApellis Pharmaceuticals, Inc.
    B.1.3.4CountryUnited States
    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 supportApellis Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationApellis Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Trial
    B.5.3 Address:
    B.5.3.1Street Address100 5th Avenue, 3rd Floor
    B.5.3.2Town/ cityWaltham
    B.5.3.3Post codeMA 02451
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrials@apellis.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/3/19/2201
    D.3 Description of the IMP
    D.3.1Product namePegcetacoplan
    D.3.2Product code APL-2
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPegcetacoplan
    D.3.9.1CAS number 2019171-69-6
    D.3.9.2Current sponsor codeAPL-2
    D.3.9.4EV Substance CodeSUB195466
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number54
    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 Yes
    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.IMP: 2
    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/3/19/2201
    D.3 Description of the IMP
    D.3.1Product namePegcetacoplan
    D.3.2Product code APL-2
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPegcetacoplan
    D.3.9.1CAS number 2019171-69-6
    D.3.9.2Current sponsor codeAPL-2
    D.3.9.4EV Substance CodeSUB195466
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number54
    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.IMP: 3
    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/3/19/2201
    D.3 Description of the IMP
    D.3.1Product namePegcetacoplan
    D.3.2Product code APL-2
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPegcetacoplan
    D.3.9.1CAS number 2019171-69-6
    D.3.9.2Current sponsor codeAPL-2
    D.3.9.4EV Substance CodeSUB195466
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number54
    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.IMP: 4
    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/3/19/2201
    D.3 Description of the IMP
    D.3.1Product namePegcetacoplan
    D.3.2Product code APL-2
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPegcetacoplan
    D.3.9.1CAS number 2019171-69-6
    D.3.9.2Current sponsor codeAPL-2
    D.3.9.4EV Substance CodeSUB195466
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number54
    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 placeboSolution for injection/infusion
    D.8.4Route of administration of the placeboSubcutaneous 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 (C3G)/immune complex membranoproliferative glomerulonephritis (IC-MPGN)
    glomerulopatía C3 primaria o glomerulonefritis membranoproliferativa mediada por inmunocomplejos
    E.1.1.1Medical condition in easily understood language
    Rare kidney disease which can reoccur after a kidney transplant.
    Enfermedad renal rara que puede reaparecer después de un trasplante de riñón.
    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 SOC
    E.1.2Classification code 10038359
    E.1.2Term Renal and urinary disorders
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    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.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10027168
    E.1.2Term Membranoproliferative glomerulonephritis
    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
    The primary objective of this study is to assess the efficacy of twice-weekly SC doses of pegcetacoplan compared with that of placebo in patients with primary C3G or IC-MPGN on the basis of a reduction in proteinuria.
    Evaluar la eficacia de dosis subcutáneas (SC) dos veces por semana de pegcetacoplán en comparación con placebo en pacientes con glomerulopatía C3 primaria (GC3) o glomerulonefritis membranoproliferativa mediada por complejos inmunitarios (GNMP CI) sobre la base de una reducción de la proteinuria.
    E.2.2Secondary objectives of the trial
    •To assess the effect of pegcetacoplan on eGFR
    •To assess the effect of pegcetacoplan on additional C3G/IC-MPGN disease–related parameters
    •To evaluate the safety of pegcetacoplan over 52 weeks of treatment
    • Evaluar el efecto del pegcetacoplán en la TFGe
    • Evaluar el efecto del pegcetacoplán sobre los parámetros adicionales relacionados con la enfermedad GC3/GNMP-CI
    • Evaluar la seguridad de pegcetacoplán durante 52 semanas de tratamiento
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Aged at least 18 years; where approved, adolescents (aged 12-17 years) weighing at least 30 kg may also be enrolled.
    2. A diagnosis of primary C3G or IC-MPGN (with or without previous renal transplant).
    3. Evidence of active renal disease, based on one or more of the following:
    a. In adults or adolescents with a baseline renal biopsy, at least 2+ C3c staining on the baseline renal biopsy
    b. In adolescents not providing a baseline renal biopsy, at least one of the following:
    − Serum C5b-9 level above the upper limit of normal during screening
    − Serum C3 below the lower limit of normal (LLN) during screening
    − Presence of an active urine sediment during screening, as evidenced by hematuria with at least 5 red blood cells per high-power field and/or red blood cell casts on routine local or central microscopic analysis of urine
    − Presence of C3 nephritic factor within 6 months of screening, based on central laboratory results or medical history
    4. No more than 50% global glomerulosclerosis or interstitial fibrosis on the baseline biopsy for adult subjects or adolescent subjects providing a baseline biopsy.
    5. At least 1 g/d of proteinuria on a screening 24-hour urine collection and a uPCR of at least 1000 mg/g on at least 2 FMU samples collected during screening.
    6. eGFR ≥30 mL/min/1.73 m2 calculated by the CKD-EPI creatinine equation for adults or the Bedside Schwartz equation for adolescents.
    7. Stable regimen for C3G/IC-MPGN treatment, as described below:
    a. Angiotensin-converting enzyme inhibitor/angiotensin receptor blocker therapy that is stable and optimized, in the opinion of the investigator, for at least 12 weeks prior to randomization
    b. Stable doses of other medications that can affect proteinuria (eg, steroids, mycophenolate mofetil, and/or other allowed immunosuppressants that the patient is receiving for treatment of C3G) for at least 8 weeks prior to the baseline renal biopsy and at least 12 weeks prior to randomization.
    c. If a subject is on prednisone (or other systemic corticosteroid) for C3G/IC-MPGN treatment, the dosage is stable and no higher than 20 mg/d (or equivalent dosage of a corticosteroid other than prednisone) for at least 8 weeks prior to the baseline renal biopsy and at least 12 weeks prior to randomization.
    8. Have received vaccinations against S pneumoniae, N meningitidis (types A, C, W, Y, and B), and H influenzae (type B) within 5 years prior to randomization or agree to receive vaccinations during screening. Vaccination series should be initiated at least 14 days prior to randomization. Vaccination is mandatory unless documented evidence exists that subjects are nonresponders to vaccination.
    9. Female subjects of childbearing potential, defined as any women who have experienced menarche and who are not permanently sterile or postmenopausal, must have negative blood pregnancy tests at screening (and negative urine pregnancy tests on Day 1) and must agree to use protocol-defined methods of contraception from screening through at least 90 days after receiving the last dose of pegcetacoplan.
    10. Male subjects must agree to use protocol-defined methods of contraception and agree to refrain from donating semen from screening through at least 90 days after receiving the last dose of pegcetacoplan.
    11. Subjects above the legal age of consent, in accordance with local regulations, must be willing and able to provide informed consent. The legally authorized representative of subjects under the legal age of consent must be willing and able to provide informed consent; where appropriate, subjects under the legal age of consent must also give their assent to participation in the study.
    12. Willing and able to self-administer pegcetacoplan or have an identified caregiver who can perform the administration.
    1. Tener al menos 18 años; cuando esté aprobado; adolescentes (de 12 a 17 años) que pesen al menos 30 kg también pueden ser enrolados.
    2. Un diagnóstico de GC3 primaria o GNMP-CI (con o sin trasplante renal previo).
    3. Pruebas de enfermedad renal activa, en base a uno o más de los siguientes criterios:
    a. En adultos o adolescentes con biopsia renal inicial, tinción de al menos 2+ para C3c en la biopsia renal inicial recolectada durante la selección.
    b. En adolescentes que no se someten a una biopsia renal inicial, al menos uno de los siguientes:
    Nivel sérico C5b-9 por sobre el límite superior de la normalidad durante la selección 3 sérico por debajo del límite inferior de la normalidad durante la selección
    Presencia de un sedimento urinario activo durante la selección, según se evidencia por hematuria con al menos 5 glóbulos rojos por campo de gran aumento y/o cilindros de glóbulos rojos en análisis de orina microscópico local o central de rutina
    Presencia de factor nefrítico C3 dentro de los 6 meses de la selección, con base en resultados de laboratorio central o antecedentes médicos
    4. No más del 50 % de glomeruloesclerosis global o fibrosis intersticial en la biopsia inicial en sujetos adultos o en sujetos adolescentes que proporcionen una biopsia inicial
    5. Al menos 1 g/día de proteinuria en una recolección de orina de 24 horas en la selección y una uPCR de al menos 1000 mg/g en al menos 2 recolecciones puntuales de la primera de orina de la mañana durante la selección.
    6. TFGe ≥30 mL/min/1,73 m2 calculada con la ecuación de creatinina de la Colaboración entre la Enfermedad Renal Crónica y la Epidemiología en adultos o la ecuación de Bedside Schwartz en adolescentes.
    7. Régimen estable para el tratamiento de GC3/GNMP-CI según se describe a continuación:
    a. Tratamiento con inhibidor de la enzima convertidora de angiotensina/bloqueador de los receptores de la angiotensina que se mantenga estable y mejorado, según la opinión del investigador, durante al menos 12 semanas antes de la aleatorización
    b. Dosis estables de otros medicamentos que puedan afectar la proteinuria (por ejemplo, esteroides, micofenolato de mofetilo y/u otros inmunosupresores permitidos que el paciente esté recibiendo para el tratamiento de C3G) durante al menos 8 semanas antes de la biopsia renal inicial y al menos 12 semanas antes de la aleatorización.
    8. Haber recibido vacunas contra S. pneumoniae, N. meningitidis (tipos A, C, W, Y, y B) y H. influenzae (tipo B) en los 5 años anteriores a la aleatorización o aceptar recibir vacunas durante la selección. Las vacunaciones deben realizarse al menos 14 días antes de la aleatorización. La vacunación es obligatoria, a menos que existan pruebas documentadas de que los sujetos no responden a la vacunación.
    9. Las mujeres en edad fértil, definidas como las mujeres que han experimentado la menarquia y que no son permanentemente estériles o posmenopáusicas, deben tener pruebas de embarazo en sangre negativas en la selección (y pruebas de embarazo en orina negativas el día 1) y deben aceptar el uso de métodos de anticoncepción definidos por el protocolo desde la selección hasta al menos 90 días después de recibir la última dosis de pegcetacoplán.
    10. Los hombres deben aceptar el uso de métodos anticonceptivos definidos por el protocolo y abstenerse de donar semen desde la selección hasta al menos 90 días después de recibir la última dosis de pegcetacoplán.
    11. Los sujetos que superen la edad legal de consentimiento, de acuerdo con las regulaciones locales, deben estar dispuestos y ser capaces de otorgar su consentimiento informado. El representante legalmente autorizado de los sujetos menores de la edad legal de consentimiento debe estar dispuesto y ser capaz de otorgar su consentimiento informado; cuando corresponda, los sujetos menores de la edad legal de consentimiento también deben dar su consentimiento para participar en el estudio.
    12. Estar dispuesto y ser capaz de autoadministrarse pegcetacoplán o tener un cuidador
    E.4Principal exclusion criteria
    1. Previous exposure to pegcetacoplan.
    2. Evidence of improving renal disease in the 8 weeks prior to screening or during the screening period according to available data; improving renal disease is defined as >30% increase in eGFR or >50% decrease in proteinuria.
    3. From a renal transplant subject, evidence of rejection that requires treatment in the baseline renal biopsy collected during screening.
    4. C3G/IC-MPGN secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, a systemic autoimmune disease such as systemic lupus erythematosus, chronic antibody-mediated rejection, or a medication), in the opinion of the investigator.
    5. Current or prior diagnosis of HIV, hepatitis B, or hepatitis C infection or positive serology during screening that is indicative of infection with any of these viruses.
    6. Weight more than 100 kg at screening.
    7. Hypersensitivity to pegcetacoplan or to any of the excipients.
    8. History of meningococcal disease.
    9. Malignancy, except for the following:
    a. Cured basal or squamous cell skin cancer
    b. Curatively treated in situ disease
    c. Malignancy-free and off treatment for ≥5 years
    10. An absolute neutrophil count less than 1000 cells/mm3 at screening.
    11. Significant other renal disease that would, in the opinion of the investigator, confound interpretation of study results.
    12. Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or 5 half-lives from the last dose of investigational agent (whichever is longer) prior to screening period.
    13. Use of rituximab, belimumab, or any approved or investigational anticomplement therapy other than pegcetacoplan within 5 half-lives of that product prior to the screening period.
    14. Female subjects who are pregnant or who are currently breastfeeding and are unwilling to discontinue for the duration of the study and for at least 90 days after the final dose of study drug.
    15. Inability to cooperate or any condition that, in the opinion of the investigator, creates an undue risk for the subject by participating in the study or is likely to confound interpretation of the study results.
    16. Evidence of ongoing drug or alcohol abuse or dependence, in the opinion of the investigator.
    17. Presence or suspicion of severe recurrent or chronic infections that, in the opinion of the investigator, may place the subject at unacceptable risk by study participation.
    18. Known or suspected hereditary fructose intolerance.
    1. Exposición previa a pegcetacoplán.
    2. Pruebas de mejoría de la enfermedad renal en las 8 semanas previas a la selección o durante el período de selección según los datos disponibles; la mejoría de la enfermedad renal se define como un aumento >30 % de la TFGe o una disminución >50 % de la proteinuria.
    3. De un sujeto con trasplante renal, pruebas de rechazo que requiera tratamiento en la biopsia renal inicial recolectada durante la selección.
    4. GC3/GNMP-CI secundaria a otra afección (por ejemplo, infección, tumor maligno, gammapatía monoclonal, una enfermedad autoinmune sistémica, como el lupus eritematoso sistémico, rechazo crónico mediado por anticuerpos o un medicamento), según la opinión del investigador.
    5. Diagnóstico actual o previo de infección por virus de inmunodeficiencia humana (VIH), hepatitis B o hepatitis C o serología positiva durante la selección que sea indicativa de infección por cualquiera de estos virus.
    6. Peso superior a 100 kg en el momento de la selección.
    7. Hipersensibilidad al pegcetacoplán o a alguno de los excipientes.
    8. Antecedentes de enfermedad meningocócica.
    9. Tumor maligno, excepto por lo siguiente:
    a. cáncer de piel de células basales o escamosas curado;
    b. enfermedad in situ tratada con intención de curar;
    c. sin tumor maligno y sin tratamiento durante ≥5 años.
    10. Un recuento absoluto de neutrófilos <1000 células/mm3 en la selección.
    11. Otra enfermedad renal importante que confundiría la interpretación de los resultados del estudio, según la opinión del investigador.
    12. Participación en cualquier otro ensayo de medicamentos en investigación o exposición a otro agente, dispositivo o procedimiento en investigación dentro de los 30 días o 5 semividas desde la última dosis del agente en investigación (el que sea más prolongado) antes del período de selección.
    13. Uso de rituximab, belimumab o cualquier tratamiento anticomplemento aprobado o en investigación, que no sea pegcetacoplán, dentro de las 5 semividas de ese producto antes del período de selección.
    14. Mujeres que están embarazadas o que están amamantando en la actualidad y no están dispuestas a interrumpir la lactancia durante el estudio y durante al menos 90 días después de la dosis final del medicamento del estudio.
    15. Incapacidad para cooperar o cualquier afección que, según la opinión del investigador, cree un riesgo indebido para el sujeto por participar en el estudio o que pueda confundir la interpretación de los resultados del estudio.
    16. Pruebas de abuso o dependencia continua de drogas o alcohol, según la opinión del investigador.
    17. Presencia o sospecha de infecciones crónicas o recurrentes severas que, según la opinión del investigador, pueden poner al sujeto en un riesgo inaceptable por participar en el estudio.
    18. Intolerancia hereditaria a la fructosa conocida o en sospecha.
    E.5 End points
    E.5.1Primary end point(s)
    The proportion of subjects with a reduction from baseline in uPCR of at least 50%. The primary and secondary endpoints will be evaluated at Week 26. The primary and secondary endpoints will also be evaluated at Week 52 as exploratory endpoints
    La proporción de sujetos con reducciones desde el valor inicial en uPCR de al menos el 50 % en la semana 26. Los criterios de valoración primarios y secundarios también se evaluarán en la semana 52 como criterios de valoración exploratorios.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Weeks 26 and 52
    Semana 26 y 52
    E.5.2Secondary end point(s)
    •The proportion of subjects with eGFR values that are stable or improved from baseline
    For subjects with evaluable renal biopsies, the change from baseline in the C3G histologic activity index score (Bomback et al. 2018)
    The proportion of subjects with evaluable renal biopsies showing decreases in C3c staining on renal biopsy from baseline
    The proportion of subjects achieving proteinuria <1 g/d
    Change from baseline in uPCR
    Change from baseline in eGFR
    For subjects with serum albumin levels below the LLN at baseline, the proportion of subjects with normalization of serum albumin levels
    For subjects with serum C3 levels below the LLN at baseline, the proportion of subjects with serum C3 levels above the LLN
    The change from baseline in the FACIT-Fatigue Scale score
    The change from baseline in the KDQOL score
    • La proporción de sujetos con valores de TFGe que sean estables o hayan mejorado desde el valor inicial valor inicial.
    Para los sujetos con biopsias renales evaluables, el cambio desde el inicio en la puntuación del índice de actividad histológica C3G (Bomback et al. 2018)
    La proporción de sujetos con biopsias renales evaluables que muestran disminuciones en la tinción C3c en la biopsia renal desde el inicio
    La proporción de sujetos que alcanzan proteinuria <1 g/d
    Cambio con respecto al inicio en la uPCR
    Cambio con respecto a la línea de base en la TFGe
    Para sujetos con niveles de albúmina sérica por debajo de la LLN al inicio del estudio, la proporción de sujetos con normalización de los niveles séricos de albúmina
    Para sujetos con niveles séricos de C3 por debajo de la LLN al inicio del estudio, la proporción de sujetos con niveles séricos de C3 por encima de la LLN
    El cambio desde el inicio en la puntuación de la Escala de Fatiga FACIT
    El cambio desde el inicio en la puntuación KDQOL
    E.5.2.1Timepoint(s) of evaluation of this end point
    Weeks 26 and 52
    Semana 26 y 52
    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 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 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 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 concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA44
    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
    Argentina
    Australia
    Austria
    Belgium
    Brazil
    Canada
    Czechia
    Finland
    France
    Germany
    Israel
    Italy
    Japan
    Korea, Republic of
    Netherlands
    Peru
    Poland
    Spain
    Switzerland
    Ukraine
    United Kingdom
    United States
    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
    The end of the study will be the last subject’s last visit or the last subject’s last scheduled visit/assessment as indicated in the schedule of activities or as requested by the sponsor.
    El final del estudio será la última visita del último sujeto o la última visita / evaluación programada del último sujeto como se indica en el calendario de actividades o según lo solicite el patrocinador.
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days22
    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: 8
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 91
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 43
    F.4.2.2In the whole clinical trial 100
    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 completion of the open-label period, subjects who would benefit from continuing to receive pegcetacoplan, as per investigator’s opinion, may roll over into a long-term extension study. Subjects who do not enter a long-term extension study will discontinue pegcetacoplan treatment and complete the 8-week follow-up period.
    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 Decision2022-02-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-02-14
    P. End of Trial
    P.End of Trial StatusOngoing
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