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    Summary
    EudraCT Number:2017-002370-39
    Sponsor's Protocol Code Number:ALXN1210-PNH-303
    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:2019-01-18
    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 number2017-002370-39
    A.3Full title of the trial
    A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,
    Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab
    Estudio de fase III aleatorizado, con grupos paralelos, multicéntrico, farmacocinético abierto, de no inferioridad que compara ravulizumab administrado por vía subcutánea con ravulizumab administrado por vía intravenosa en pacientes adultos con hemoglobinuria paroxística nocturna tratada actualmente con eculizumab
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab
    Este estudio compara la farmacocinética (FC) de ravulizumab subcutáneo (SC) administrado a través de un sistema de administración corporal (SAC) con ravulizumab intravenoso (IV) en pacientes con hemoglobinuria paroxística nocturna (PNH) actualmente tratada con eculizumab
    A.4.1Sponsor's protocol code numberALXN1210-PNH-303
    A.5.4Other Identifiers
    Name:IND NumberNumber:128367
    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 Pharma Spain
    B.5.2Functional name of contact pointRosa Enrique
    B.5.3 Address:
    B.5.3.1Street AddressPº de Gracia,85
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08008
    B.5.3.4CountrySpain
    B.5.4Telephone number+34932723019
    B.5.5Fax number+41444574081
    B.5.6E-mailclinicaltrials.eu@alexion.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/3/16/1661
    D.3 Description of the IMP
    D.3.1Product nameRavulizumab
    D.3.2Product code ALXN1210
    D.3.4Pharmaceutical form Solution for 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 INNFc- and CDR-modified humanised monoclonal antibody against C5
    D.3.9.2Current sponsor codeALXN1210
    D.3.9.3Other descriptive nameRavulizumab
    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 number70
    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 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 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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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/3/16/1661
    D.3 Description of the IMP
    D.3.1Product nameRavulizumab
    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 INNFc- and CDR-modified humanised monoclonal antibody against C5
    D.3.9.2Current sponsor codeALXN1210
    D.3.9.3Other descriptive nameRavulizumab
    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) No
    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
    Hemoglobinuria paroxística nocturna
    E.1.1.1Medical condition in easily understood language
    Paroxysmal Nocturnal Hemoglobinuria
    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 20.1
    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
    To evaluate PK noninferiority of ravulizumab SC versus ravulizumab IV in adult patients with PNH
    Evaluar la FC de no inferioridad de ravulizumab s.c. frente a ravulizumab i.v. en pacientes con HPN
    E.2.2Secondary objectives of the trial
    - To characterize PK of ravulizumab SC
    - To characterize PD of ravulizumab SC
    - To characterize immunogenicity of ravulizumab SC
    - To evaluate HRQoL and treatment satisfaction on ravulizumab SC
    - To evaluate safety of ravulizumab SC and ravulizumab OBDS
    - To evaluate efficacy of ravulizumab SC
    - To assess performance of ravulizumab OBDS
    - Caracterizar la FC de ravulizumab s.c.
    - Caracterizar la FD de ravulizumab s.c.
    - Caracterizar la inmunogenia de ravulizumab s.c.
    - Evaluar la CdVRS y la satisfacción con el tratamiento con ravulizumab s.c.
    - Evaluar la seguridad de ravulizumab s.c. y el SAC de ravulizumab
    - Evaluación de la eficacia de ravulizumab s.c.
    - Evaluación del rendimiento del SAC de ravulizumab
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Age
    1. Patients must be at least 18 years of age at the time of signing the informed consent.

    Patient and Disease Characteristics
    2. Treated with eculizumab according to the labeled dosing recommendation for PNH (900 mg every 14 days ± 2 days) for at least 6 months prior to study entry with no missed doses within 2 months prior to study entry and no more than 2 doses outside of the visit window.
    3. Lactate dehydrogenase levels ≤ 1.5 × ULN (upper limit of normal), according to central laboratory, at Screening. Sample must be obtained within 24 hours of or immediately prior to a scheduled eculizumab dose administration (ie, at trough eculizumab level).
    4. Documented diagnosis of PNH confirmed by high-sensitivity flow cytometry evaluation (Borowitz, Craig et al. 2010).
    5. Vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug to reduce the risk of meningococcal infection (N meningitidis).
    Edad
    1.Los pacientes deben tener, al menos, 18 años en el momento de firmar el consentimiento informado.

    Características del paciente y de la enfermedad
    2.Tratado con eculizumab de acuerdo con la recomendación posológica de la ficha técnica para la HPN (900 mg cada 14 días ± 2 días) durante, al menos, 6 meses antes de la incorporación al estudio y que no se haya saltado ninguna dosis en los 2 meses previos a la incorporación al estudio y no más de 2 dosis fuera del margen de la visita.
    3.Niveles de lactato deshidrogenasa ≤ 1,5 × LSN (límite superior de la normalidad), de acuerdo con el laboratorio central, en la selección. La muestra se debe obtener en las 24 horas previas o inmediatamente antes de una administración de dosis de eculizumab programada (es decir, con el nivel mínimo de eculizumab).
    4.Diagnóstico documentado de HPN confirmado mediante evaluación con citometría de flujo de alta sensibilidad (Borowitz, 2010).
    5.Vacunado contra infecciones meningocócicas en los 3 años previos al inicio del fármaco del estudio o en dicho momento, para reducir el riesgo de infección meningocócica (N. meningitidis).
    E.4Principal exclusion criteria
    Medical Conditions
    1. More than 1 LDH value > 2 × ULN within the 6 months prior to study entry.
    2. Major adverse vascular event (MAVE) in the 6 months prior to study entry.
    3. Platelet count < 30,000/mm3 (30 × 109/L) at Screening.
    4. Absolute neutrophil count < 500/μL (0.5 × 109/L) at Screening.
    5. History of bone marrow transplantation.
    6. History of N meningitidis infection.
    Afecciones médicas
    1.Más de 1 valor de LDH > 2 × LSN a lo largo de los 6 meses previos a la incorporación
    al estudio.
    2.Acontecimiento adverso vascular importante (AAVI) en los 6 meses previos a la incorporación al estudio.
    3.Recuento plaquetario ≤ 30 000/mm3 (30 × 109/l) en la selección.
    4.Recuento absoluto de neutrófilos < 500/µl (0,5 × 109/l) en la selección.
    5.Antecedentes de trasplante de médula ósea.
    6.Antecedentes de infección por N. meningitidis.
    E.5 End points
    E.5.1Primary end point(s)
    Day 71 serum ravulizumab Ctrough
    Cmín. sérica de ravulizumab del día 71
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 71
    Dia 71
    E.5.2Secondary end point(s)
    PK Endpoint: Ctrough over time

    PD Endpoint: Free serum C5 concentrations over time

    Immunogenicity

    HRQoL and Treatment Satisfaction Endpoints

    Safety Endpoints

    Efficacy Endpoints
    Criterio de valoración FC: Cmín. a lo largo del tiempo

    Criterio de valoración FD: Concentraciones séricas de C5 libre a lo largo del tiempo

    Criterio de valoración de inmunogenia

    Criterios de valoración de satisfacción con el tratamiento y CdVRS

    Criterios de valoración de la seguridad

    Criterios de valoración de la eficacia
    E.5.2.1Timepoint(s) of evaluation of this end point
    throughout the study
    A lo largo del estudio
    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 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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Pacientes en el grupo de ravulizumab IV cambiarán a 490 mg de ravulizumab SC en el período de extens
    Patients in the ravulizumab IV group will switch to 490 mg of ravulizumab SC in the extension period
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Ravulizumab IV
    Ravulizumab iv
    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 EEA46
    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
    Austria
    Belgium
    Brazil
    Canada
    Czech Republic
    Finland
    France
    Germany
    Italy
    Korea, Republic of
    Netherlands
    Russian Federation
    Spain
    Sweden
    Turkey
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    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 is defined as the date of the last patient visit or safety follow up, whichever occurs later.
    El final del estudio se define como la fecha de la última visita del paciente o del último seguimiento de seguridad, lo que ocurra más tarde.
    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 months11
    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 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: 95
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 state21
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 65
    F.4.2.2In the whole clinical trial 105
    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)
    Upon completion of the last study visit, patients will return to the care of their treating physician
    Al finalizar la última visita del estudio, los pacientes volverán al cuidado de su médico habitual
    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 Decision2019-02-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-01-31
    P. End of Trial
    P.End of Trial StatusOngoing
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