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    Summary
    EudraCT Number:2019-001106-23
    Sponsor's Protocol Code Number:APL2-307
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-01-14
    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 number2019-001106-23
    A.3Full title of the trial
    An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).
    ESTUDIO DE EXTENSIÓN ABIERTO, NO ALEATORIZADO, MULTICÉNTRICO PARA EVALUAR LA EFICACIA Y SEGURIDAD A LARGO PLAZO DE APL-2 EN EL TRATAMIENTO DE HEMOGLOBINURIA PAROXÍSTICA NOCTURNA (HPN)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).
    A.4.1Sponsor's protocol code numberAPL2-307
    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 organisationBiorasi GmbH
    B.5.2Functional name of contact pointEuropean Project Operations
    B.5.3 Address:
    B.5.3.1Street AddressNiermannsweg 11-15
    B.5.3.2Town/ cityErkrath
    B.5.3.3Post code40699
    B.5.3.4CountryGermany
    B.5.4Telephone number+492112503180
    B.5.5Fax number+4921125033233
    B.5.6E-mailhschmied@biorasi.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/3/17/1873
    D.3 Description of the IMP
    D.3.1Product nameAPL-2
    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.3.9.3Other descriptive nameAPL-2
    D.3.9.4EV Substance CodeSUB192794
    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
    Paroxysmal Nocturnal Hemoglobinuria
    hemoglobinuria paroxística nocturna
    E.1.1.1Medical condition in easily understood language
    PNH is a rare disorder causing red blood cells to break down too early
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10055629
    E.1.2Term Paroxysmal nocturnal hemoglobinuria
    E.1.2System Organ Class 100000004857
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objectives of this study are to:
    1. Establish the long-term safety of APL-2 in PNH subjects
    2. Establish the long-term efficacy of APL-2 in PNH subjects
    A continuación, se detallan los objetivos de este estudio:
    1. Establecer la seguridad a largo plazo de APL-2 en pacientes con HPN
    2. Establecer la eficacia a largo plazo de APL-2 en pacientes con HPN
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects with PNH who have participated in an APL-2 clinical trial.
    Subjects who received treatment with APL-2 must have experienced
    clinical benefit in the opinion of the Investigator.
    Note: Subjects with PNH who completed an APL-2 clinical trial without
    receiving APL-2 (or without receiving APL-2 for long enough to
    demonstrate clinical benefit) may be enrolled in this study if, in the
    opinion of the Investigator, the subject is expected to demonstrate
    clinical benefit upon the initiation or continuation of APL-2 therapy.
    2. Willing and able to give written informed consent.
    3. Women of child-bearing potential (WOCBP) must have a negative
    pregnancy test and must agree to continue to use an approved method
    of contraception for the duration of the study and 60 days after their last
    dose of study drug.
    4. Males must agree to continue to use an approved method of
    contraception and must agree to refrain from donating sperm for the
    duration of the study and 60 days after their last dose of study drug.
    1. Pacientes con HPN que participaron en un ensayo clínico de APL-2. Los pacientes que recibieron tratamiento con APL-2 deben haber obtenido beneficios clínicos según el Investigador.
    Nota: Los pacientes con HPN que completaron un ensayo clínico de APL-2 sin recibir APL-2 (o sin haber recibido APL-2 por tiempo suficiente para demostrar beneficios clínicos) pueden participar en este estudio si, según el Investigador, se espera que el paciente demuestre beneficios clínicos al iniciar o continuar el tratamiento con APL-2.
    2. Los pacientes que deseen y puedan brindar su consentimiento informado por escrito.
    3. Las mujeres en edad fértil (women of child-bearing potential, WOCBP) deben tener una prueba de embarazo negativa y deben aceptar continuar con el uso de un método anticonceptivo aprobado durante la vigencia del estudio y 60 días después de su última dosis del medicamento en estudio.
    4. Los hombres deben aceptar continuar con el uso de un método anticonceptivo aprobado y abstenerse de donar esperma durante la vigencia del estudio y 60 días después de su última dosis del medicamento en estudio.
    E.4Principal exclusion criteria
    1. Subjects who have withdrawn from an APL-2 clinical study
    2. Any condition that could increase the subject’s risk by participating in the study
    1. Los pacientes que se hayan retirado de un estudio clínico de APL-2.
    2. Toda enfermedad que podría aumentar el riesgo del paciente al participar en el estudio
    E.5 End points
    E.5.1Primary end point(s)
    Safety Endpoints
    • Incidence and severity of Treatment Emergent Adverse Events (TEAE)
    • Incidence of thromboembolic events
    • Laboratory parameters
    • ECG parameters

    Efficacy Endpoints
    • Lactate dehydrogenase (LDH) level
    • Reticulocyte count
    • Hemoglobin level
    • Red blood cell (RBC) transfusions
    • FACIT-fatigue scale score
    Criterios de valoración de seguridad:
    • Incidencia y gravedad de los eventos adversos derivados del tratamiento (treatment emergent adverse event, TEAE)
    • Incidencia de los eventos tromboembólicos
    • Parámetros de laboratorio
    • Parámetros del ECG

    Criterios de valoración de eficacia:
    • Nivel de hemoglobina
    • Nivel de lactato deshidrogenasa (lactate dehydrogenase, LDH)
    • Recuento de reticulocitos
    • Transfusiones de glóbulos rojos (red blood cell, RBC)
    • Puntuación de la escala FACIT de fatiga
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety Endpoints
    • Incidence and severity of Treatment Emergent Adverse Events (TEAE): after dosing begins, or worsen of severity, for up to 30 days after the last dose of APL-2
    • Incidence of thromboembolic events:
    after dosing begins for up to 30 days after the last dose of APL-2
    • Laboratory parameters: Please refer to Schedule Events in Protocol
    • ECG parameters: Please refer to Schedule Events in Protocol
    Efficacy Endpoints:
    Please Refer to Schedule Events in Protocol
    E.5.2Secondary end point(s)
    Pharmacokinetic Endpoint
    • APL-2 pharmacokinetic concentrations

    Pharmacodynamic Endpoints
    • Complement (e.g., CH50, AH50, and C3) levels
    • C3 deposition on RBC cells
    • Clonal distribution of PNH RBCs
    • Incidence of anti-APL-2 and anti-PEG antibodies
    Criterios de valoración de farmacocinética:
    • Concentraciones farmacocinéticas de APL-2
    Marcadores farmacodinámicos:
    • Niveles de complemento (p. ej., CH50, AH50 y C3)
    • Depósito de C3 en células RBC
    • Distribución clonal de RBC de HPN
    • Incidencia de anticuerpos anti-APL-2 y anti-PEG
    E.5.2.1Timepoint(s) of evaluation of this end point
    Pharmacokinetic Endpoints:
    Blood samples will be collected at the time points delineated in the Schedule of Events in Section 3 of Protocol

    Pharmacodynamic Endpoints: Blood samples will be collected at the time points delineated in the Schedule of Events in Section 3 of Protocol
    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 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 EEA30
    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
    Australia
    Belgium
    Bulgaria
    Canada
    France
    Germany
    Hong Kong
    Japan
    Korea, Republic of
    Malaysia
    Poland
    Russian Federation
    Serbia
    Spain
    Thailand
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    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: 66
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 16
    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 state11
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 48
    F.4.2.2In the whole clinical trial 119
    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. Expected normal treatment of the condition.
    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 Decision2020-01-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-01-15
    P. End of Trial
    P.End of Trial StatusCompleted
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