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    The EU Clinical Trials Register currently displays   43233   clinical trials with a EudraCT protocol, of which   7153   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2017-002370-39
    Sponsor's Protocol Code Number:ALXN1210-PNH-303
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-01-18
    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 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.
    Studio di fase 3, randomizzato, a gruppi paralleli, multicentrico, di farmacocinetica in aperto, di non inferiorità su ravulizumab somministrato per via sottocutanea comparato alla somministrazione per via endovenosa in pazienti adulti con emoglobinuria parossistica notturna trattati 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.
    Questo studio confronta la farmacocinetica (PK) di ravulizumab sottocutaneo (SC) somministrato tramite un sistema di rilascio nel corpo (OBDS) con quella di ravulizumab somministrato per via endovenosa (IV) n pazienti con emoglobinuria parossistica notturna (PNH) attualmente in trattamento con eculizumab.
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code numberALXN1210-PNH-303
    A.5.4Other Identifiers
    Name:INDNumber: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 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, 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 number0033147100615
    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.2.1.1.1Trade name NA
    D.2.1.1.2Name of the Marketing Authorisation holderNA
    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 injection
    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.3.9.2Current 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 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 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.2.1.1.1Trade name NA
    D.2.1.1.2Name of the Marketing Authorisation holderNA
    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.3.9.2Current 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 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) 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
    Emoglobinuria parossistica notturna
    E.1.1.1Medical condition in easily understood language
    Paroxysmal Nocturnal Hemoglobinuria
    Emoglobinuria parossistica notturna
    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
    To evaluate PK noninferiority of ravulizumab SC versus ravulizumab IV in adult patients with PNH.
    Valutare la non inferiorità della farmacocinetica di ravulizumab sc contro ravulizumab IV in pazienti adulti con PNH.
    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
    - Caratterizzare la farmacocinetica di ravulizumab SC
    - Caratterizzare la farmacodinamica di ravulizumab SC
    - Caratterizzare l'immunogenicità di ravulizumab SC
    - Valutare HRQoL e la soddisfazione al trattamento con ravulizumab SC
    - Valutare la sicurezza di ravulizumab SC e di ravulizumab OBDS
    - Valutare l'efficacia di ravulizumab SC
    - Valutare la performance di ravulizumab OBDS
    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).
    Età
    1. I pazienti devono avere minimo 18 anni al momento della firma del consenso informato.

    Pazienti e caratteristiche della malattia
    2. Trattati con eculizumab secondo il dosaggio previsto sull'etichetta per pazienti con PNH (900 mg ogni 14 giorni ± 2 giorni) per almeno 6 mesi prima dell'entrata nello studio e che non abbiano saltato alcuna dose entro i 2 mesi prima dell'inizio dello studio e che non abbiano ricevuto più di 2 dosial di fuori della finiestra delle visite.
    3. Livelli di lattatodeidrogenasi = 1.5 × ULN (upper limit of normal), secondo il laboratorio centrale, allo screening. I campioni devono essere ottenuti entro 24 ore oppure immediatamente prima di un trattamento programmato con eculizumab (i.e. con il livello pià basso di eculizumab)
    4. Diagnosi documentata di PNH confermata da citometria a flusso ad alta sensibilità (Borowitz, Craig et al. 2010).
    5. Vaccinati contro infezione meningococcica entro 3 anni dall'inizio dello studio, oppure all'inizio dello studio, per ridurre il rischio di infezione meningococcica (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.
    Condizione medica
    1. Più di 1 valore di LDH > 2 × ULN entro 6 mesi dall'ingresso nello studio.
    2. Evento vascolare avverso importante (MAVE) entro 6 mesi priam dell'ingresso nello studio.
    3. Conta delle piastrine < 30,000/mm3 (30 × 109/L) allo screening
    4. Conta assoluta dei neutrofili < 500/µL (0.5 × 109/L) allo screening
    5. Anamnesi di trapianto di midollo
    6. Anamnesi di infezione da N meningitidis
    E.5 End points
    E.5.1Primary end point(s)
    Day 71 serum ravulizumab Ctrough
    Cthrough di ravulizumab nel siero al giorno 71
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 71
    Giorno 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
    - Cthrough e PK nel tempo
    - PD: concentrazione diC5 nel siero nel tempo
    - Immunogenicità
    - HRQoL e soddisfazione al trattamento
    - Sicurezza
    - Efficacia
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the study
    Durante lo studio
    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
    I pazienti nel gruppo ravulizimab IV passeranno a ravulizumab SC 490 mg durante il periodo di estens
    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
    Stesso farmaco, forma farmaceutica differente.
    Same drug, different pharmaceutical form.
    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 concerned7
    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 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
    Australia
    Brazil
    Canada
    Korea, Republic of
    Russian Federation
    Turkey
    United States
    Austria
    Belgium
    Czechia
    Finland
    France
    Germany
    Italy
    Netherlands
    Spain
    Sweden
    United Kingdom
    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.
    La fine dello studio è definita come la data dell'ultima visita dell'ultimo paziente oppure del follow-up di sicurezza, a seconda di quale evento occorra prima.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months1
    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 state10
    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.
    Dopo il completamento dell'ultima visita dello studio, i pazienti torneranno in cura presso i rispettivi medici.
    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 Decision2019-02-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-02-18
    P. End of Trial
    P.End of Trial StatusOngoing
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