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Summary
EudraCT Number:2022-002833-33
Sponsor's Protocol Code Number:APL2-C3G-314
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:2022-12-20
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
A. Protocol Information
A.1Member State ConcernedSpain - AEMPS
A.2EudraCT number2022-002833-33
A.3Full title of the trial
An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Participants With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
Estudio de extensión abierto, no aleatorizado y multicéntrico para evaluar la seguridad y la eficacia a largo plazo de pegcetacoplán en participantes con glomerulopatía C3 o glomerulonefritis membranoproliferativa por inmunocomplejos
A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
A study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
Un estudio para evaluar la seguridad y la eficacia a largo plazo de pegcetacoplán en participantes con glomerulopatía C3 o glomerulonefritis membranoproliferativa por inmunocomplejos
A.3.2Name or abbreviated title of the trial where available
VALE
VALE
A.4.1Sponsor's protocol code numberAPL2-C3G-314
A.7Trial is part of a Paediatric Investigation Plan Yes
A.8EMA Decision number of Paediatric Investigation PlanP/284/2022
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 Yes
D.2.1.1.1Trade name Aspaveli
D.2.1.1.2Name of the Marketing Authorisation holderSwedish Orphan Biovitrum AB ((publ)
D.2.1.2Country which granted the Marketing AuthorisationSweden
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.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
C3 glomerulopathy (C3G) or immune complex membranoproliferative glomerulonephritis (IC-MPGN)
Glomerulopatía por C3 (GC3) o glomerulonefritis membranoproliferativa por inmunocomplejos (GNMP-IC)
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 rinón
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 establish the long-term safety and efficacy of pegcetacoplan in participants with C3G or IC-MPGN
El objetivo primario de este estudio es determinar la seguridad y la eficacia a largo plazo del pegcetacoplán en participantes con GC3 o GNMP-IC
E.2.2Secondary objectives of the trial
An additional exploratory objective is to characterize the long-term effects of treatment with pegcetacoplan in participants with C3G or IC-MPGN, including PK, pharmacodynamics (PD), and immunogenic response.
Un objetivo exploratorio adicional es caracterizar los efectos a largo plazo del tratamiento con pegcetacoplán en participantes con GC3 o GNMP-IC, incluyendo FC, farmacodinámica (FD) y la respuesta inmunogénica.
E.2.3Trial contains a sub-study No
E.3Principal inclusion criteria
1.completed participation in Study APL2-C3G -310 through the week 52 visit requirements
2.experienced clinical benefit from pegcetacoplan while participating in the previous trial, in the opinion of the investigator
3.must remain on a stable regimen for C3G or IC -MPGN treatment according to the requirements of Study APL2-C3G -310
4.received vaccinations against S pneumoniae, N meningitidis (types A, C, W, Y, and B), and H influenzae (type B) according to the requirements of Study APL2-C3G -310 and agree to receive any additional vaccinations recommended according to ACIP recommendations for adults or children with complement deficiencies and/or immunocompromising conditions or other similar local applicable guidelines
5.Female participants of childbearing potential, defined as any woman who has experienced menarche and who is not permanently sterile or postmenopausal, must have a negative urine pregnancy test at v isit 1 and must agree to use protocol-defined methods of contraception for the duration of the study through at least 90 days after receiving the last dose of pegcetacoplan.
6.Male participants must agree to use protocol-defined methods of contraception and agree to refrain from donating semen for the duration of the study through at least 90 days after receiving the last dose of pegcetacoplan.
7.Participants 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 participants under the legal age of consent must be willing and able to provide informed consent; where appropriate, participants under the legal age of consent must also give their assent to participation in the study.
8.willing and able to self-administer pegcetacoplan or have an identified caregiver who can perform the administration
1. Haber completado la participación en el estudio APL2-C3G-310, hasta satisfacer los requisitos de la visita de la semana 52.
2. Haber experimentado beneficio clínico con pegcetacoplán mientras se participaba en el ensayo anterior, en opinión del investigador.
3. Permanecer necesariamente en una pauta posológica estable para el tratamiento de la GC3 o la GNMP-IC, de acuerdo con los requisitos del estudio APL2-C3G-310.
4. Haberse puesto vacunas contra S. pneumoniae, N. meningitidis (tipos A, C, W, Y B) y H. influenzae (tipo B), de acuerdo con los requisitos del estudio APL2-C3G-310, y acceder a ponerse cualquier otra vacuna que venga aconsejada en las recomendaciones del Comité Asesor sobre Prácticas de Vacunación en el caso de adultos o niños afectados de déficit del complemento o trastornos inmunodepresores, o en otras directrices nacionales o regionales similares que sean de aplicación.
5. En el caso de las participantes que tengan capacidad de concebir, definidas como cualquier mujer que haya tenido la menarquia y que no sea posmenopáusica o estéril de forma permanente, presentar obligatoriamente un resultado negativo en la prueba de embarazo en orina de la visita 1 y acceder a usar los métodos anticonceptivos indicados en el protocolo durante todo el estudio y hasta pasados al menos 90 días de la última dosis de pegcetacoplán.
6. En el caso de los participantes masculinos, acceder a usar los métodos anticonceptivos indicados en el protocolo y abstenerse de donar semen durante el transcurso del estudio y hasta pasados al menos 90 días de la última dosis de pegcetacoplán.
7. En el caso de los participantes que hayan cumplido la edad legal mínima para prestar consentimiento, con arreglo a la normativa nacional, tener la voluntad y la capacidad de proporcionar consentimiento informado. El representante legal autorizado de los participantes que no alcancen dicha edad debe tener la voluntad y la capacidad de prestar dicho consentimiento; cuando proceda, tales participantes menores también deben consentir en participar en el estudio.
8. Tener voluntad y capacidad de autoadministrarse pegcetacoplán o contar con un cuidador identificado que pueda llevar a cabo la administración.
E.4Principal exclusion criteria
1.female participants who are or are planning to become 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
2.inability or unwillingness to cooperate with the requirements of the protocol
3.any condition that, in the opinion of the investigator, creates an undue risk for the participantby participating in the study or is likely to confound interpretation of the study results
4.evidence of ongoing drug or alcohol abuse or dependence, in the opinion of the investigator
1. En el caso de las voluntarias de sexo femenino, estar embarazada o tener previsto quedarse embarazada, o encontrarse en período de lactancia y no estar dispuesta a interrumpir el amamantamiento durante el estudio y durante al menos los 90 días posteriores a la última dosis del fármaco estudiado.
2. Mostrar incapacidad o falta de voluntad de cooperar para cumplir los requisitos del protocolo.
3. Presentar cualquier afección que, en opinión del investigador, genere un riesgo indebido para el voluntario a resultas de su participación en el estudio o que probablemente vaya a dificultar la interpretación de los resultados del estudio.
4. Presentar signos de drogadicción, drogodependencia, alcoholismo o dependencia del alcohol, en opinión del investigador.
E.5 End points
E.5.1Primary end point(s)
The primary efficacy endpoint is the proportion of participants with a reduction in urine protein-to-creatinine ratio (uPCR) of at least 50% from the pretreatment value over time.
El criterio de valoración principal de la eficacia es la proporción de participantes que presenten una reducción en el cociente proteína/creatinina en orina (CPCo) de al menos el 50 % con respecto al valor previo al tratamiento a lo largo del tiempo
E.5.1.1Timepoint(s) of evaluation of this end point
(uPCR) value over time.
CPCo a lo largo del tiempo.
E.5.2Secondary end point(s)
•the proportion of participants with estimated glomerular filtration rate (eGFR) values that are stable or improved from pretreatment values over time
•the proportion of participants achieving proteinuria <1 g/day over time
•change from pretreatment in uPCR values over time
•change from pretreatment in eGFR values over time
•for participants with pretreatment serum albumin levels below the lower limit of normal (LLN), the proportion of participants with normalization of serum albumin levels over time
•for participants with pretreatment serum C3 levels below the LLN, the proportion of participant s with normalization of serum C3 levels over time
•the proportion of participants with progression to a clinical composite outcome (at least one of the following: doubling of serum creatinine, progression to chronic kidney disease stage 5 or ESRD, renal transplantation, or death) over time
•la proporción de participantes que presenten valores estimados de la tasa de filtración glomerular (TFGe) estables o mejorados con respecto a los valores previos al tratamiento a lo largo del tiempo
•la proporción de participantes que logren proteinuria <1 g/día a lo largo del tiempo
•cambio con respecto a antes del tratamiento en los valores de la CPCo a lo largo del tiempo
•cambio con respecto a antes del tratamiento en los valores de la TFGe a lo largo del tiempo
•en el caso de los participantes que muestren antes del tratamiento una concentración de albúmina sérica situada por debajo del límite inferior de la normalidad (LIN), la proporción de participantes que presenten normalización de dicha concentración a lo largo del tiempo
•en el caso de los participantes que muestren antes del tratamiento una concentración de C3 en suero por debajo del LIN, la proporción de participantes que presenten normalización de dicha concentración a lo largo del tiempo
•la proporción de participantes que muestren progresión a un criterio de valoración clínico compuesto (al menos uno de los siguientes: duplicación de la creatinina sérica, progresión a nefropatía crónica en estadio 5 o nefropatía terminal, trasplante renal o muerte) a lo largo del tiempo
E.5.2.1Timepoint(s) of evaluation of this end point
(eGFR), uPCR, serum albumin level values over time
(TFGe), CPCo, valores del nivel de albúmina sérica a lo largo del tiempo
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 No
E.8.1.1Randomised No
E.8.1.2Open Yes
E.8.1.3Single blind No
E.8.1.4Double blind No
E.8.1.5Parallel group No
E.8.1.6Cross over No
E.8.1.7Other No
E.8.2 Comparator of controlled trial
E.8.2.1Other medicinal product(s) No
E.8.2.2Placebo No
E.8.2.3Other No
E.8.2.4Number of treatment arms in the trial1
E.8.3 The trial involves single site in the Member State concerned No
E.8.4 The trial involves multiple sites in the Member State concerned Yes
E.8.4.1Number of sites anticipated in Member State concerned2
E.8.5The trial involves multiple Member States Yes
E.8.5.1Number of sites anticipated in the 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
Brazil
Canada
Israel
Japan
Korea, Republic of
Peru
United States
Austria
France
Poland
Bulgaria
Netherlands
Spain
Switzerland
Czechia
Germany
Italy
Belgium
United Kingdom
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 when the last participant has completed their last visit as indicated in the schedule of activities (Table 1) or has withdrawn from the study as described in Section 7.3 of the protocol or as requested by the sponsor
El final del estudio será cuando el último paciente haya completado su última visita como se indica en el cronograma de actividades (Tabla 1) o se haya retirado del estudio como se describe en la Sección 7.3 del protocolo o según lo solicite el Promotor.
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 months6
E.8.9.1In the Member State concerned days
E.8.9.2In all countries concerned by the trial years4
E.8.9.2In all countries concerned by the trial months2
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: 10
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: 10
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 No
F.3.3.1Women of childbearing potential not using contraception No
F.3.3.2Women of child-bearing potential using contraception No
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 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)
None
Ninguno
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 Decision2023-02-22
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2023-02-21
P. End of Trial
P.End of Trial StatusOngoing

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