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    Summary
    EudraCT Number:2016-002025-11
    Sponsor's Protocol Code Number:ALXN1210-PNH-301
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-06-17
    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 ConcernedItaly - Italian Medicines Agency
    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-Na¿ve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
    STUDIO DI FASE 3, RANDOMIZZATO, IN APERTO, CON CONTROLLO ATTIVO DI ALXN1210 RISPETTO A ECULIZUMAB IN PAZIENTI ADULTI AFFETTI DA EMOGLOBINURIA PAROSSISTICA NOTTURNA (EPN) NA¿VE AL TRATTAMENTO CON INIBITORI 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.
    Studio su ALXN1210 rispetto a eculizumab in pazienti con EPN che non sono mai stati trattati con inibitori del complemento.
    A.3.2Name or abbreviated title of the trial where available
    Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a compleme
    Studio su ALXN1210 rispetto a eculizumab in pazienti con EPN che non sono mai stati trattati con ini
    A.4.1Sponsor's protocol code numberALXN1210-PNH-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02946463
    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 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 number0033147100606
    B.5.5Fax number0033147100611
    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 nameALXN1210
    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.3.9.2Current 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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 AuthorisationItaly
    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.3.1Product nameALXN1210
    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 INNECULIZUMAB
    D.3.9.1CAS number 219685-50-4
    D.3.9.2Current sponsor codeND
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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)
    EMOGLOBINURIA PAROSSISTICA NOTTURNA (EPN)
    E.1.1.1Medical condition in easily understood language
    Paroxysmal Nocturnal Hemoglobinuria (PNH)
    EMOGLOBINURIA PAROSSISTICA NOTTURNA (EPN)
    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 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.
    L¿obiettivo primario di questo studio ¿ valutare la ALXN1210 rispetto a eculizumab in pazienti adulti affetti da EPN che non sono mai stati sottoposti a trattamento con inibitori del complemento.
    E.2.2Secondary objectives of the trial
    - safety and tolerability of ALXN1210
    - efficacy
    - PK/PD and immunogenicity
    - long-term safety and efficacy
    - To evaluate the safety and efficacy in patients who switch from eculizumab to ALXN1210 in the Extension Period
    - sicurezza e tollerabilit¿ di ALXN1210
    - efficacia
    - PK/PD e immunogenicit¿
    - sicurezza ed efficacia a lungo termine
    - valutare la sicurezza e l'efficacia nei pazienti che passano da eculizumab a ALXN1210 nel periodo di estensione
    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. Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment.
    6. Female patients of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ALXN1210.
    7. Willing and able to give written informed consent and comply with study visit schedule.
    1. Soggetto di sesso maschile o femminile di almeno 18 anni di età al momento del consenso.
    2. Diagnosi documentata di EPN confermata mediante citometria a flusso ad alta sensibilità
    3. La presenza di 1 o più dei seguenti segni o sintomi correlati all’EPN entro 3 mesi dallo screening: affaticamento, emoglobinuria, dolore addominale, mancanza di respiro (dispnea), anemia (emoglobina <10 g/dl), anamnesi di un evento avverso vascolare maggiore (tra cui trombosi), disfagia o disfunzione erettile; oppure anamnesi di trasfusione di pRBC dovuta a EPN.
    4. Livello di LDH =1,5 volte l’ULN allo screening.
    5. Documentata vaccinazione contro le infezioni da meningococco entro i 3 anni precedenti o nel momento in cui si inizia l’assunzione del farmaco dello studio
    6. Le pazienti in età fertile devono seguire le linee guida previste dal protocollo per evitare una gravidanza durante il trattamento e negli 8 mesi successivi all’ultima dose del farmaco dello studio.
    7. I pazienti devono essere disposti a e in grado di fornire un consenso informato scritto e di rispettare tutte le visite e le procedure dello studio


    E.4Principal exclusion criteria
    1. Treatment with a complement inhibitor at any time.
    2. History of bone marrow transplantation.
    3. Body weight < 40 kilograms.
    4. Females who are pregnant, breastfeeding or who have a positive pregnancy test at screening or 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, would
    preclude participation.
    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. Trattamento attuale o precedente con un inibitore del complemento.
    2. Anamnesi di trapianto di midollo osseo.
    3. Peso corporeo <40 kg allo screening.
    4. Soggetti di sesso femminile in gravidanza, in allattamento o che prevedano di iniziare una gravidanza.
    5. Partecipazione a un altro studio di trattamento interventistico o uso di qualsiasi terapia sperimentale nei 30 giorni precedenti all’inizio della somministrazione del farmaco dello studio il Giorno 1 di questo studio o entro 5 emivite di quel prodotto sperimentale, a seconda di quale sia maggiore.
    6. Anamnesi o presenza di malattia cardiaca, polmonare, renale, endocrina o epatica maggiore (ad esempio epatite attiva) che, a giudizio dello Sperimentatore o dello Sponsor, precluda la partecipazione del paziente a una sperimentazione clinica.
    7. Condizioni mediche instabili (ad es. ischemia miocardica, emorragia gastrointestinale attiva, grave insufficienza cardiaca congestizia, necessità precoce di un intervento chirurgico maggiore entro 6 mesi dalla randomizzazione, anemia cronica concomitante non correlata all’EPN)
    E.5 End points
    E.5.1Primary end point(s)
    - Percentage of patients who achieve transfusion avoidance (TA)
    - Normalization of lactate dehydrogenase (LDH) levels
    - Percentuale di pazienti che non ricevono alcuna trasfusione e non necessitano di trasfusione
    - Normalizzazione dei livelli di LDH
    E.5.1.1Timepoint(s) of evaluation of this end point
    26 weeks
    26 settimane
    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
    - Variazione percentuale nell¿LDH dal basale
    - Cambiamento nella qualit¿ della vita (QoL) valutato mediante la Scala di valutazione funzionale della terapia per malattie croniche (FACIT)-Affaticamento
    - Percentuale di pazienti con emolisi episodica
    - Percentuale di pazienti con emoglobina stabilizzata
    E.5.2.1Timepoint(s) of evaluation of this end point
    26 weeks
    26 settimane
    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 concerned12
    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 Information not present in EudraCT
    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.
    La fine Studio ¿ definita come la data dell'ultima visita dell'ultimo paziente nel periodo di estensione
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    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 years6
    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 state25
    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.
    Il paziente torna alle cure del medico curante
    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 Decision2017-02-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-01-11
    P. End of Trial
    P.End of Trial StatusCompleted
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