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    Summary
    EudraCT Number:2015-002674-20
    Sponsor's Protocol Code Number:ALXN1210-PNH-201
    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:2015-10-05
    Trial results View 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 number2015-002674-20
    A.3Full title of the trial
    A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria
    ESTUDIO EN FASE II ABIERTO, CON DOSIS MÚLTIPLE ASCENDENTE PARA EVALUAR LA EFICACIA, SEGURIDAD,
    TOLERABILIDAD, INMUNOGENIA, FARMACOCINÉTICA Y FARMACODINÁMICA DE ALXN1210 ADMINISTRADO POR VÍA
    INTRAVENOSA A PACIENTES CON HEMOGLOBINURIA PAROXÍSTICA NOCTURNA
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria
    ESTUDIO EN FASE II ABIERTO, CON DOSIS MÚLTIPLE ASCENDENTE PARA EVALUAR LA EFICACIA, SEGURIDAD,
    TOLERABILIDAD, INMUNOGENIA, FARMACOCINÉTICA Y FARMACODINÁMICA DE ALXN1210 ADMINISTRADO POR VÍA
    INTRAVENOSA A PACIENTES CON HEMOGLOBINURIA PAROXÍSTICA NOCTURNA
    A.4.1Sponsor's protocol code numberALXN1210-PNH-201
    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 SponsorAlexion 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 supportALEXION PHARMACEUTICALS INC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationALEXION EUROPE SAS
    B.5.2Functional name of contact pointEuropean Clinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address1-15 avenue Edouard Belin
    B.5.3.2Town/ cityRueil-Malmaison
    B.5.3.3Post code92500
    B.5.3.4CountryFrance
    B.5.4Telephone number+33147100606
    B.5.5Fax number+33147100611
    B.5.6E-mailclinicaltrials.eu@alxn.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.3.2Product code ALXN1210
    D.3.4Pharmaceutical form Concentrate for solution for 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 INNALXN1210
    D.3.9.2Current sponsor codeALXN1210
    D.3.9.4EV Substance CodeSUB172162
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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) Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Paroxysmal Nocturnal Hemoglobinuria (PNH)
    HEMOGLOBINURIA PAROXÍSTICA NOCTURNA
    E.1.1.1Medical condition in easily understood language
    Paroxysmal Nocturnal Hemoglobinuria (PNH)
    HEMOGLOBINURIA PAROXÍSTICA NOCTURNA
    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 18.0
    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
    To evaluate the efficacy, safety, and tolerability of multiple doses of ALXN1210 administered intravenously (IV) to complement inhibitor treatment-naïve patients with PNH
    Evaluar la eficacia, seguridad y tolerabilidad de múltiples dosis de ALXN1210 administrado por vía intravenosa (IV) a pacientes con HPN sin tratamiento previo con inhibidor del complemento.
    E.2.2Secondary objectives of the trial
    The secondary objectives of the trial are:
    - To characterize the pharmacokinetic (PK) and pharmacodynamic (PD) effects of multiple doses of ALXN1210 administered IV to complement inhibitor treatment-naïve patients with PNH
    - To investigate the immunogenicity of ALXN1210 administered IV to complement inhibitor treatment-naïve patients with PNH
    -Caracterizar los efectos farmacocinéticos (FC) y farmacodinámicos (FD) de múltiples dosis de ALXN1210
    administrado por vía IV a pacientes con HPN sin tratamiento previo con inhibidor del complemento.
    -Investigar la inmunogenia de ALXN1210 administrado por vía IV a pacientes con HPN sin tratamiento previo con
    inhibidor del complemento.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female patients ? 18 years of age
    2. PNH diagnosis confirmed by documented high-sensitivity flow cytometry (red blood cells [RBCs] and/or granulocytes)
    3. Mean lactate dehydrogenase (LDH) ?3 × upper limit of normal, based on 2 measurements from separate blood samples collected at least 1 day apart during screening
    4. Willing and able to give written informed consent and comply with the study visit schedule
    5. Documented meningococcal vaccination not more than 3 years prior to dosing
    6. Female patients who consider themselves postmenopausal must provide evidence at screening of menopause status, based on a combination of amenorrhea for at least 1 year and increased serum follicle-stimulating hormone level (> 30 IU/L) on at least 2 occasions (eg, in the absence of hormone replacement therapy, dietary phytoestrogens) or estradiol concentration < 10 pg/mL.
    7. Female patients of childbearing potential must use highly effective contraception as defined below, starting at screening and continuing until at least 6 months after the last dose of ALXN1210.
    8. Male patients with a female spouse/partner of childbearing potential or a pregnant or breastfeeding spouse or partner must agree to use barrier contraception (male condom) during the Treatment Period and for at least 6 months after the last dose of ALXN1210. Barrier contraception is required even with documented medical assessment of surgical success of a vasectomy. Female spouses/partners of male patients who are of childbearing potential must use highly effective contraception or acceptable contraception, as defined below, starting at screening and continuing until at least 6 months after the last dose of ALXN1210. Male patients must not donate sperm during the
    Screening and Treatment Periods and for at least 6 months after the last dose of ALXN1210.
    1. Pacientes varones o mujeres ? 18 años de edad.
    2. Diagnóstico confirmado de HPN mediante documentación por citometría de flujo de alta sensibilidad (eritrocitos
    o granulocitos).
    3. Lactato deshidrogenasa (LDH) media ? 3 veces el límite superior de la normalidad, de acuerdo con 2 medidas
    procedentes de muestras de sangre por separado recogidas al menos con 1 día de diferencia durante la selección.
    4. Sujeto capaz y con voluntad de otorgar el consentimiento informado por escrito y cumplir el calendario de visitas
    del estudio.
    5. Vacuna meningocócica documentada no más de 3 años antes de la administración.
    6. Las pacientes que se consideren posmenopáusicas deben proporcionar pruebas de su estado de menopausia en la
    selección, de acuerdo con una combinación de amenorrea durante al menos 1 año y un aumento en la
    concentración sérica de la folitropina (> 30 UI/l) en al menos 2 ocasiones (por ejemplo, en ausencia de
    tratamiento de restitución hormonal, fitoestrógenos de origen alimentario) o concentración de estradiol <10 pg/ml.
    7. Las pacientes en edad fértil deben utilizar un método anticonceptivo de alta eficacia tal y como se define a
    continuación, empezando en la selección y hasta al menos 6 meses tras la última dosis de ALXN1210.
    8. Los pacientes varones con una pareja en edad fértil, embarazada o en periodo de lactancia deben aceptar el uso de
    anticonceptivos de barrera (preservativo masculino) durante el periodo de tratamiento y durante al menos 6 meses
    después de la última dosis de ALXN1210. Es necesario el uso del anticonceptivo de barrera incluso con la
    evaluación médica documentada de vasectomía realizada de forma satisfactoria. Las parejas de los pacientes
    varones que estén en edad fértil deben usar un método anticonceptivo de alta eficacia (según se define en el
    criterio de inclusión número 7) o un método anticonceptivo aceptable, según se define a continuación, empezando
    en la selección y hasta 6 meses después de la última dosis de ALXN1210. Los pacientes varones no deben donar
    semen durante los periodos de selección y tratamiento ni durante al menos 6 meses después de la última dosis de
    ALXN1210.
    E.4Principal exclusion criteria
    1. Treatment with a complement inhibitor at any time
    2. Platelet count < 30,000/mm3 (30 × 109 /L) at screening
    3. Absolute neutrophil count < 500/microLiter (0.5 × 109 /L) at screening
    4. History of bone marrow transplantation
    5. History of Neisseria meningitidis infection; history of unexplained, recurrent infection; or infection requiring treatment with systemic antibiotics within the last 90 days prior to dosing on Day 1
    6. Female patients who are planning to become pregnant, or are pregnant or breastfeeding
    1. Tratamiento con un inhibidor del complemento en cualquier momento.
    2. Recuento de plaquetas < 30 000/mm3 (30 × 109/l) en la selección.
    3. Recuento absoluto de neutrófilos < 500/microlitro (0,5 × 109/l) en la selección.
    4. Antecedentes de trasplante de médula ósea.
    5. Antecedentes de infección por Neisseria meningitidis; antecedentes de infección recidivante y de origen
    inexplicado; o infección que requiera tratamiento con antibióticos sistémicos en los últimos 90 días previos a la
    dosis del día 1.
    6. Pacientes de sexo femenino que estén pensando quedarse embarazadas o que estén embarazadas o en periodo de
    lactancia.
    E.5 End points
    E.5.1Primary end point(s)
    Change in LDH levels from baseline to Day 253
    cambios en los niveles de LDH desde el inicio al dia 253
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 1, 7, 15, 22, 29, 43, 57, 85, 113, 127, 141, 169, 197, 211, 225, 253
    dia 1, 7, 15, 22, 29, 43, 57, 85, 113, 127, 141, 169, 197, 211, 225, 253
    E.5.2Secondary end point(s)
    Efficacy endpoints:
    - Changes in hemolysis-related hematologic parameters
    - Changes in clinical manifestations

    Safety endpoints:
    - Change from baseline in the need for blood transfusions
    - Change from baseline in disease-associated biomarkers
    - Change from baseline in quality of life
    - Change from baseline in major adverse vascular events (MAVEs)

    Immunogenicity: Measurement of antidrug antibodies (ADA)

    Pharmacokinetic/Pharmacodynamic

    Safety
    Las variables de eficacia:
    - Los cambios en hemólisis relacionados con parámetros hematológicos
    - Cambios en las manifestaciones clínicas

    Criterios de valoración de seguridad:
    - Cambio del valor inicial en la necesidad de transfusiones de sangre
    - Cambio del valor inicial en biomarcadores asociados a la enfermedad
    - Cambio del valor inicial en la calidad de vida
    - Cambio del valor inicial en los principales eventos vasculares adversos (MAVES)

    Inmunogenicidad: Medición de anticuerpos antidrogas (ADA)

    Farmacocinético / farmacodinámico

    Seguridad
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 1, 7, 15, 22, 29, 43, 57, 85, 113, 127, 141, 169, 197, 211, 225, 253
    dia 1, 7, 15, 22, 29, 43, 57, 85, 113, 127, 141, 169, 197, 211, 225, 253
    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 Yes
    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
    immunogenicity
    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 No
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    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 trial3
    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
    Canada
    Finland
    France
    Germany
    Russian Federation
    Spain
    Sweden
    Taiwan
    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
    LVLS
    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 months1
    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: 15
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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 14
    F.4.2.2In the whole clinical trial 18
    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)
    return to the standard of care
    Volver al tratamiento estándar
    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 Decision2015-11-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-11-16
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-01-12
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