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    Summary
    EudraCT Number:2019-003352-37
    Sponsor's Protocol Code Number:ALXN1210-NMO-307
    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:2021-06-17
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2019-003352-37
    A.3Full title of the trial
    A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD)
    Studio di fase III, multicentrico, controllato con placebo esterno e in aperto volto a valutare l'efficacia e la sicurezza di ravulizumab in pazienti adulti affetti da disturbo dello spettro della neuromielite ottica (NMOSD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 Efficacy and Safety Study of Ravulizumab in Adult Patients with NMOSD
    Uno studio di fase 3 sull'efficacia e la sicurezza di Ravulizumab in pazienti adulti con NMOSD
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code numberALXN1210-NMO-307
    A.5.4Other Identifiers
    Name:INDNumber:144,187
    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 Address103-105 Rue Anatole France
    B.5.3.2Town/ cityLevallois-Perret
    B.5.3.3Post code92300
    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 Yes
    D.2.1.1.1Trade name Ultromiris
    D.2.1.1.2Name of the Marketing Authorisation holderAlexion Europe SAS
    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.3.1Product 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.3.9.1CAS number 1803171-55-2
    D.3.9.2Current sponsor codeALXN1210
    D.3.9.4EV Substance CodeSUB192773
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number9
    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
    Neuromyelitis Optica Spectrum Disorder
    Disturbo dello spettro della neuromielite ottica
    E.1.1.1Medical condition in easily understood language
    NMOSD
    NMOSD
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10077875
    E.1.2Term Neuromyelitis optica spectrum disorder
    E.1.2System Organ Class 10029205 - Nervous system disorders
    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 effect of ravulizumab on adjudicated On-Trial Relapses in adult patients with NMOSD
    Valutare l'effetto di ravulizumab sulle recidive confermate durante studio in pazienti adulti affetti da NMOSD
    E.2.2Secondary objectives of the trial
    - To evaluate the safety of ravulizumab in adult patients with NMOSD
    - To evaluate the effect of ravulizumab on adjudicated annualized response rate (ARR) in adult patients with NMOSD
    - To characterize the PK of ravulizumab in adult patients with NMOSD
    - To characterize the pharmacodynamics (PD) of ravulizumab in adult patients with NMOSD
    - Valutare la sicurezza di ravulizumab in pazienti adulti affetti da NMOSD
    - Valutare l'effetto di ravulizumab sul tasso annualizzato di recidiva (Adjudicated Annualized Rate, ARR) confermata in pazienti adulti affetti da NMOSD
    - Caratterizzare la farmacocinetica (Pharmacokinetics, PK) di ravulizumab in pazienti adulti affetti da NMOSD
    - Caratterizzare la farmacodinamica (Pharmacodynamics, PD) di ravulizumab in pazienti adulti affetti da NMOSD
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient must be 18 years of age or older, at the time of signing the
    informed consent. Anti-AQP4 Ab-positive and a diagnosis of NMOSD as
    defined by the 2015 international consensus diagnostic criteria
    (Wingerchuk, 2015).
    2. At least 1 attack or relapse in the last 12 months prior to the
    Screening Period NOTE: Patients with a single life-time attack will be
    considered to satisfy inclusion criterion #3 if the attack occurred in the
    last 12 months.
    3. Expanded Disability Status Scale (EDSS) score = 7
    4. Patients who enter the trial receiving supportive IST (eg,
    corticosteroids, azathioprine [AZA], mycophenolate mofetil [MMF],
    methotrexate [MTX], and tacrolimus [TAC]) for the prevention of
    relapse, either in combination or monotherapy, must be on a stable
    dosing regimen of adequate duration prior to Screening with no plan to
    change the dose during the study period as follows:
    a. If patients who enter the study are receiving AZA, they must have
    been on AZA for = 6 months and have been on a stable dose for = 2
    months prior to Screening.
    b. If patients who enter the study are receiving other ISTs (eg, MMF,
    MTX, or TAC), they must have been on the IST for = 3 months and have
    been on a stable dose for = 4 weeks prior to Screening.
    c. If patients who enter the study are receiving oral corticosteroids, they
    must have been on a stable dose for = 4 weeks prior to Screening.
    d. If a patient enters the trial receiving oral corticosteroid(s) with or
    without other IST(s), the daily corticosteroid dose must be no more than
    prednisone 20 mg/day (or equivalent) prior to Screening.
    5. Vaccinated against N. meningitidis within 3 years prior to, or at the
    time of, initiating ravulizumab. Patients who initiate study drug
    treatment less than 2 weeks after receiving a meningococcal vaccine
    must receive appropriate prophylactic antibiotics until 2 weeks after the
    vaccination.
    1. I pazienti devono avere 18 anni o più al momento in cui firmano il consenso informato. Devono essere positivi per Anti-AQP4 Ab e la diagnosi di NMOSD deve essere formulata secondo "international consensus diagnostic criteria" del 2015
    2. Almeno un evento o 1 recidiva negli ultimi 12 mesi prima dello screening. NOTA: i pazienti con un solo evento nel corso della loro vita soddisfano il criterio n.3 se l'evento si è verificato negli ultimi 12 mesi.
    3. Punteggio Expanded Disability Status Scale (EDSS) = 7
    4. I pazienti che entrano nello studio con terapia immunosoppressiva di supporto (eg, corticosteroids, azathioprine [AZA], mycophenolate mofetil [MMF], methotrexate [MTX], and tacrolimus [TAC]) per la prevenzione delle recidive, sia in combinazione che in monoterapia, devo essere in dosaggio stabile di durata adeguata prima dello screening senza alcun piano di modificare la dose durante lo studio, secondo lo schema seguente:
    a. Se un paziente che entra nello studio sta assumendo AZA, deve essere in trattamento con AZA da = 6 mesi e deve essere in dosaggio stabile per = 2 mesi prima dello screening.
    b. Se un paziente che entra nello studio sta ricevendo altri IST (eg, MMF, MTX, o TAC), deve essere in trattamento per almeno = 3 mesi e devono essere in dosaggio stabile per = 4 settimane prima dello screening.
    c. Se un paziente che entra nello studio sta assumendo corticosteroidi per via orale, con o senza IST, la dose giornaliera di corticosteroide non deve essere superiore a prednisone 20 mg/day (o equivalente) prima dello screening.
    5. Vaccinato contro N. Meningitidis entro 3 anni prima, o al momento, di iniziare la terapia con Ravulizumab. I pazienti che iniziano il trattamento con il farmaco in studio meno di due settimane dopo aver ricevuto il vaccino devono ricevere anche una profilassi antibiotica adeguata fino a 2 settimane dopo la vaccinazione.
    E.4Principal exclusion criteria
    1. History of N. meningitidis infection.
    2. Human immunodeficiency virus (HIV) infection (evidenced by HIV-1
    or HIV-2 antibody titer)
    3. History of unexplained infections
    4. Active systemic bacterial, viral, or fungal infection within 14 days
    prior to study drug administration on Day 1
    5. Previously or currently treated with a complement inhibitor.
    6. Use of rituximab within 3 months prior to Screening
    7. Use of mitoxantrone within 3 months prior to Screening
    8. Use of Intravenous Immunoglobulin (IVIg) within 3 weeks prior to
    Screening
    9. Participation in any other investigational drug study or exposure to an
    investigational drug or device within 30 days of Screening or 5 half-lives
    of the study drug, whichever is greater
    10. Pregnant, breastfeeding, or intending to conceive during the course
    of the study
    1. Anamnesi di infezione da N. Meningitidis
    2. Infezione da HIV (anticorpi HIV-1 o HIV-2)
    3. Anamnesi di infezioni inspiegabili
    4. Infezioni attive da batteri, virus e funghi entro 14 giorni prima della somministrazione del farmaco dello studio al giorno 1
    5. In trattamento oppure trattato in passato con un inibitore del complemento.
    6. Uso di rituximab nei 3 mesi precedenti priam dello screening.
    7. Uso di Mitroxantrone nei 3 mesi precedenti priam dello screening.
    8. Uso di immunoglobuline Endovena nei 3 mesi precedenti priam dello screening
    9. Partecipazione a qualsiasi altri studio con farmaco sperimentale o esposizione a qualsiasi farmaco o dispositivo sperimentale entro 30 giorni dallo screening o 5 emivite del farmaco, qualunque durata sia più elevata
    10. Incinta, in allattamento o che ha intenzione di concepire durante il corso dello studio.
    E.5 End points
    E.5.1Primary end point(s)
    Time to first adjudicated On-Trial Relapse and relapse risk reduction.
    Tempo alla prima recidiva confermata in studio e riduzione del rischio di recidiva.
    E.5.1.1Timepoint(s) of evaluation of this end point
    On-Trial Relapses will be monitored throughout the study. The Investigator or a qualified designee will review the signs and symptoms of a potential relapse with the patient in detail at each visit.
    La recivida verrà monitorata durante il corso dello studio. Lo sperimentatore o un collaboratore qualificato valuterà i segni e i sintomi di una potenziale recidiva con il paziente ad ogni visita.
    E.5.2Secondary end point(s)
    1. Adjudicated On Trial ARR
    2. Clinically important worsening in expanded disability status scale
    (EDSS)
    3. Change from baseline in EuroQoL-5D (EQ-5D)
    4. Clinically important change in Hauser ambulation index (HAI)
    5. Change in serum ravulizumab concentration over the study duration
    6. Change in serum free C5 concentration over the study duration
    7. Presence and titer of ADAs over the study duration
    1. ARR in studio stabilito.
    2. Peggioramento clinicamente significativo del risultato ottenuto alla scala dello stato di disabilità espansa (Expanded Disability Status Scale, EDSS).
    3. Variazione dal basale dei risultati ottenuti al questionario EuroQoL-5D (EQ-5D).
    4. Variazione clinicamente significativa dell'indice di deambulazione di Hauser (Hauser Ambulation Index, HAI).
    5. Variazione della concentrazione sierica di ravulizumab nell'arco dello studio.
    6. Variazione della concentrazione sierica di C5 libero nell'arco dello studio.
    7. Presenza e titolo degli anticorpi anti-farmaco (Anti-Drug Antibody, ADA) nell'arco dello studio.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Time of on-trial relapse
    2. Throughout the study
    3. Throughout the study
    4. Throughout the study
    5. Throughout the study
    6. Throughout the study
    7. Throughout the study
    1. Nel corso dello studio.
    2. Nel corso dello studio.
    3. Nel corso dello studio.
    4. Nel corso dello studio.
    5. Nel corso dello studio.
    6. Nel corso dello studio.
    7. Nel corso dello 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 Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity Assessments
    Valutazione di immunogenicità
    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 No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Placebo esterno
    External Placebo
    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
    Placebo esterno
    External Placebo
    E.8.2.4Number of treatment arms in the trial1
    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 EEA36
    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
    Canada
    Japan
    Korea, Republic of
    Russian Federation
    Turkey
    United States
    Austria
    Denmark
    France
    Germany
    Italy
    Netherlands
    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
    Date of the last visit of the last patient in the trial globally
    LPLV globale.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    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: 50
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 55
    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)
    Ravulizumab will not be provided to the patients after the last scheduled dosing. All patients will be followed for safety for an additional 8 weeks after the last dose of study drug or early discontinuation.
    Ravulizumab non sarà fornito ai pazienti dopo l'ultima dose programmata. Tutti i pazienti verranno seguiti per la sicurezza per 8 settimane dopo l'ultima dose del farmaco in studio o dopo il loro ritiro.
    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 Decision2020-03-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-02-19
    P. End of Trial
    P.End of Trial StatusOngoing
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