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    Summary
    EudraCT Number:2016-002025-11
    Sponsor's Protocol Code Number:ALXN1210-PNH-301
    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:2016-11-04
    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 number2016-002025-11
    A.3Full title of the trial
    A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Estudio en fase III, aleatorizado, abierto y controlado con principio activo de ALXN1210 frente a eculizumab en pacientes adultos con hemoglobinuria paroxística nocturna (HPN) sin tratamiento previo con inhibidores del complemento
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.
    Estudio de ALXN1210 frente a eculizumab en pacientes adultos con HPN sin tratamiento previo con inhibidores del complemento
    A.4.1Sponsor's protocol code numberALXN1210-PNH-301
    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 Incorporated
    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 Incorporated
    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+3493 272 3019
    B.5.5Fax number+4144 457 40 81
    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.3.2Product 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 nameFc- and CDR-modified humanised monoclonal antibody against C5
    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.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 Yes
    D.2.1.1.1Trade name Soliris
    D.2.1.1.2Name of the Marketing Authorisation holderAlexion Europe SAS
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/03/166
    D.3 Description of the IMP
    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 INNECULIZUMAB
    D.3.9.1CAS number 219685-50-4
    D.3.9.4EV Substance CodeSUB25187
    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 (HPN)
    E.1.1.1Medical condition in easily understood language
    Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Hemoglobinuria paroxística nocturna (HPN)
    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 19.0
    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
    The purpose of this study is to assess ALXN1210 compared to eculizumab in adult patients with PNH who have never been treated with a complement inhibitor.
    El objetivo de este estudio es evaluar ALXN1210 en comparación con el eculizumab en pacientes adultos con HPN que nunca han sido tratados con un inhibidor del complemento.
    E.2.2Secondary objectives of the trial
    - safety and tolerability of ALXN1210
    - efficacy
    - PK/PD and immunogenicity
    - long-term safety and efficacy
    - Seguridad y tolerabilidad de ALXN1210
    -Eficacia
    -FC/FD e inmunogenética
    -Seguridad y eficacia a largo plazo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female ≥ 18 years of age
    2. PNH diagnosis confirmed by documented by high-sensitivity flow cytometry
    3. Presence of 1 or more of the following PNH-related signs or symptoms within 3 months of Screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia (hemoglobin <10 g/dL), history of a major adverse vascular event (including thrombosis), dysphagia, or erectile dysfunction; or history of pRBC transfusion due to PNH.
    4. LDH level ≥ 1.5 × ULN at screening.
    5. All patients must be vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug
    6. Female patients of childbearing potential and male patients with female partners of childbearing potential must follow protocol-specified guidance for avoiding pregnancy while on treatment and for 8 months after last dose of study drug.
    7. Patients must be willing and able to give written informed consent and to comply with all study visits and procedures
    1. Varones o mujeres de 18 años de edad o mayores
    2. Diagnóstico documentado de HPN confirmado mediante citometría de flujo de alta sensibilidad
    3. Presencia de 1 o más de los siguientes signos o síntomas relacionados con la HPN en los 3 meses anteriores a la selección: fatiga, hemoglobinuria, dolor abdominal, dificultad para respirar (disnea), anemia (hemoglobina < 10 g/dl), antecedentes de un acontecimiento adverso vascular importante (incluida trombosis), disfagia o disfunción eréctil o antecedentes de transfusión de CGR como consecuencia de HPN.
    4. Nivel de LDH ≥ 1,5 veces el LSN en la selección.
    5. Se debe vacunar a todos los pacientes contra la meningitis meningocócica durante los 3 años antes de, o en el momento de, iniciar el fármaco del estudio.
    6. Las pacientes en edad fértil y los pacientes con parejas en edad fértil deben seguir las recomendaciones especificadas en el protocolo para evitar el embarazo durante el tratamiento y durante 8 meses después de la última dosis del fármaco del estudio.
    7. Los pacientes deben estar dispuestos y ser capaces de dar su consentimiento informado por escrito y cumplir con todas las visitas y procedimientos del estudio
    E.4Principal exclusion criteria
    1. Current or previous treatment with a complement inhibitor.
    2. History of bone marrow transplantation.
    3. Body weight < 40 kilograms.
    4. Females who plan to become pregnant or are currently pregnant or breastfeeding. Females who have a positive pregnancy test result at Screening or on Day 1.
    5. Participation in another interventional clinical study or use of any experimental therapy within 30 days before initiation of study drug on Day 1 in this study or within 5 half-lives of that investigational product, whichever is greater.
    6. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the Investigator or Sponsor, precludes the patient’s participation in an investigational clinical trial.
    7. Unstable medical conditions (eg, myocardial ischemia, active gastrointestinal bleed, severe congestive heart failure, anticipated need for major surgery within 6 months of randomization, coexisting chronic anemia unrelated to PNH)
    1. Tratamiento actual o previo con un inhibidor del complemento.
    2. Antecedentes de trasplante de médula ósea.
    3. Peso corporal < 40 kg.
    4. Mujeres que piensen quedarse embarazadas o que estén embarazadas o en periodo de lactancia en ese momento. Mujeres que tienen un resultado positivo en la prueba de embarazo en la selección o el día 1.
    5. Participación en otro estudio terapéutico intervencionista o uso de cualquier tratamiento experimental en los 30 días previos al inicio del fármaco del estudio el día 1 en este estudio, o durante 5 semividas del producto en fase de investigación, lo que sea mayor
    6. Antecedentes de enfermedad cardíaca, pulmonar, renal, endocrina o hepática importante o en curso que, en opinión del investigador o del promotor, excluye la participación del paciente en un ensayo clínico en fase de investigación.
    7. Trastornos médicos inestables (p. ej., isquemia miocárdica, hemorragia gastrointestinal activa, insuficiencia cardíaca congestiva grave, necesidad prevista de cirugía mayor en el plazo de 6 meses con respecto a la aleatorización, coexistencia de anemia crónica no relacionada con HPN)
    E.5 End points
    E.5.1Primary end point(s)
    - Percentage of patients who achieve transfusion avoidance (TA)
    - Normalization of lactate dehydrogenase (LDH) levels
    - Porcentaje de pacientes que consiguen evitar la transfusión (ET)
    - Normalización de los niveles de la lactato deshidrogenasa (LDH)
    E.5.1.1Timepoint(s) of evaluation of this end point
    26 weeks
    26 semanas
    E.5.2Secondary end point(s)
    - Percentage change from baseline in lactate dehydrogenase ( LDH) levels
    - Change from baseline in quality of life as assessed by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue
    - Percentage of patients with breakthrough hemolysis
    - Percentage of patients with stabilized hemoglobin
    - Cambio porcentual de los niveles de LDH desde el inicio
    - Cambio en la calidad de vida, evaluada a través de la escala de fatiga-evaluación funcional del tratamiento de enfermedades crónicas (Functional Assessment of Chronic Illness Therapy, FACIT)
    - Proporción de pacientes con hemólisis intercurrente,
    - Proporción de pacientes con hemoglobina estabilizada
    E.5.2.1Timepoint(s) of evaluation of this end point
    26 weeks
    26 semanas
    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 No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA66
    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
    Korea, Republic of
    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’s last visit in the Extension Period.
    El fin del estudio es definido con la fecha de la última visita del último paciente en el periodo de extensión.
    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: 192
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 22
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 214
    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)
    Patients will return to the care of his/her physician.
    Los pacientes volverán al cuidado de su médico.
    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 Decision2017-01-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-11-30
    P. End of Trial
    P.End of Trial StatusOngoing
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