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    Summary
    EudraCT Number:2019-001565-33
    Sponsor's Protocol Code Number:RA101495-02.302
    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:2021-01-07
    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 number2019-001565-33
    A.3Full title of the trial
    A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects with Generalized Myasthenia Gravis
    Studio di estensione di fase 3, multicentrico, in aperto su zilucoplan in soggetti affetti da miastenia grave generalizzata
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Open-Label Extension Study of Zilucoplan in Subjects with Generalized Myasthenia Gravis
    Studio di estensione in aperto su zilucoplan in soggetti affetti da miastenia grave generalizzata
    A.3.2Name or abbreviated title of the trial where available
    RAISE-XT
    RAISE-XT
    A.4.1Sponsor's protocol code numberRA101495-02.302
    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 SponsorRa 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 supportRa Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRa Pharmaceuticals, Inc
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address87 Cambridge Park Drive
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA, 02140
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16174014060
    B.5.5Fax number000000
    B.5.6E-mailtrials@rapharma.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameZilucoplan
    D.3.2Product code [na]
    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.3.8INN - Proposed INNzilucoplan
    D.3.9.1CAS number 1841136-73-9
    D.3.9.2Current sponsor codeRA101495
    D.3.9.4EV Substance CodeSUB194709
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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
    Generalized Myasthenia Gravis
    miastenia grave generalizzata
    E.1.1.1Medical condition in easily understood language
    Myasthenia Gravis
    miastenia grave
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10028415
    E.1.2Term Myasthenia
    E.1.2System Organ Class 100000004859
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    •To provide access to zilucoplan for subjects with gMG who have completed a qualifying Ra Pharmaceuticals sponsored zilucoplan study and who wish to continue receiving zilucoplan
    •To evaluate the long-term efficacy of zilucoplan in subjects with gMG who have completed a qualifying Ra Pharmaceuticals sponsored zilucoplan clinical study
    •To evaluate the long-term safety and tolerability of zilucoplan in subjects with gMG who have completed a qualifying Ra Pharmaceuticals sponsored zilucoplan clinical study
    • Fornire l’accesso a zilucoplan a soggetti affetti da gMG che hanno completato uno studio qualificante su zilucoplan sponsorizzato da Ra Pharmaceuticals e che desiderano continuare a ricevere zilucoplan
    • Valutare l’efficacia a lungo termine di zilucoplan in soggetti affetti da gMG che hanno completato uno studio clinico qualificante su zilucoplan sponsorizzato da Ra Pharmaceuticals
    • Valutare la sicurezza e la tollerabilità a lungo termine di zilucoplan in soggetti affetti da gMG che hanno completato uno studio clinico qualificante su zilucoplan sponsorizzato da Ra Pharmaceuticals
    E.2.2Secondary objectives of the trial
    na
    na
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    To be eligible for this study, subjects must meet ALL of the following inclusion criteria:
    1.Completion of a qualifying Ra Pharmaceuticals sponsored zilucoplan study
    2.Able to provide informed consent, including signing and dating the informed consent form (ICF)
    3.Vaccination with a quadrivalent meningococcal vaccine and, where available, meningococcal serotype B vaccine. A booster vaccination should also be administered as clinically indicated, according to the local SOC, for subjects who have been previously vaccinated against Neisseria meningitidis
    4.Female subjects of childbearing potential must have a negative urine pregnancy test within 24 hours prior to the first dose of study drug
    5.Sexually active female subjects of childbearing potential (i.e., women who are not postmenopausal or who have not had a hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) and all male subjects (who have not been surgically sterilized by vasectomy) must agree to use effective contraception during the study. Postmenopausal women are, for the purposes of this protocol, defined as women who have gone 12 consecutive months without menstruation
    Per essere idonei a partecipare a questo studio, i soggetti devono soddisfare TUTTI i criteri di inclusione elencati di seguito:
    1. Completamento di uno studio qualificante su zilucoplan sponsorizzato da Ra Pharmaceuticals
    2. Capacità di fornire il consenso informato, inclusa la capacità di firmare e datare il modulo di consenso informato (ICF)
    3. Vaccinazione con un vaccino anti-meningococco quadrivalente e, ove disponibile, un vaccino contro il sierotipo B del meningococco. Inoltre, i soggetti precedentemente vaccinati contro Neisseria meningitidis devono ricevere una vaccinazione di richiamo come clinicamente indicato secondo la SOC locale
    4. I soggetti di sesso femminile potenzialmente fertili devono presentare un test di gravidanza sulle urine negativo entro 24 ore prima della prima dose di farmaco dello studio
    5. Se sessualmente attivi, i soggetti di sesso femminile potenzialmente fertili (ovvero, donne non in età post-menopausale o non sottoposte a isterectomia, ooforectomia bilaterale o legatura bilaterale delle tube) e tutti i soggetti di sesso maschile (non sottoposti a sterilizzazione chirurgica mediante vasectomia) devono acconsentire a utilizzare un metodo contraccettivo efficace durante lo studio. Per gli scopi del presente protocollo, si definiscono in età post-menopausale le donne con anamnesi di amenorrea per 12 mesi consecutivi
    E.4Principal exclusion criteria
    Subjects who meet ANY of the following exclusion criteria must be excluded from the study:
    1.Pregnant, planning to become pregnant, or nursing female subjects
    2.With the exception of a prior zilucoplan trial, participation in another concurrent clinical trial involving an experimental therapeutic intervention (participation in observational studies and/or registry studies is permitted)
    3.Unable or unwilling to comply with the requirements of the study
    4.Did not start any disallowed medication per the exclusion criteria from the qualifying zilucoplan study or alter the dose of any other concomitant medication, unless medically indicated
    5.Any new or worsening medical condition (since entry into the qualifying zilucoplan study) or any other reason that, in the opinion of the investigator or Sponsor, would disqualify the subject from participation in this study
    I soggetti che soddisfano UNO QUALSIASI dei seguenti criteri di esclusione devono essere esclusi dallo studio:
    1. Soggetti di sesso femminile in gravidanza, che intendono rimanere incinte o in allattamento
    2. Con l’eccezione di una precedente sperimentazione di zilucoplan, partecipazione a un’altra sperimentazione clinica concomitante che preveda un intervento terapeutico sperimentale (è ammessa la partecipazione a studi osservazionali e/o a studi di registro)
    3. Incapacità o riluttanza ad attenersi ai requisiti dello studio
    4. Soggetti che non hanno iniziato ad assumere alcun farmaco proibito secondo i criteri di esclusione dello studio qualificante su zilucoplan o che modificano la dose di qualsiasi altro farmaco concomitante, salvo se clinicamente indicato
    5. Qualsiasi condizione medica di nuova insorgenza o in fase di peggioramento (dal momento dell’ingresso nello studio qualificante di zilucoplan) o qualsiasi altro motivo che, a giudizio dello sperimentatore o dello sponsor, renderebbe il soggetto non idoneo alla partecipazione a questo studio
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy endpoints:
    •MG-ADL Score
    •QMG Score
    •MG-QOL15r
    •MGC Score
    Safety endpoints
    Valutazioni dell’efficacia:
    • Punteggio del Questionario sulle attività quotidiane nella miastenia grave (Miastenia Gravis-Activities of Daily Living, MG-ADL)
    • Punteggio della Scala di valutazione quantitativa della miastenia grave (Quantitative Myasthenia Gravis, QMG)
    • Punteggio del questionario rivisto sulla qualità della vita nella miastenia grave (Myasthenia Gravis-Quality of Life Revised, MG-QOL15r)
    • Punteggio della Scala composita per la valutazione della miastenia grave (Myasthenia Gravis Composite, MGC)
    Valutazioni della sicurezza
    E.5.1.1Timepoint(s) of evaluation of this end point
    during each visit
    durante ogni visita
    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 No
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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
    In aperto
    Open
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    Canada
    Japan
    United States
    France
    Germany
    Italy
    Norway
    Spain
    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
    LSLV
    LSLV
    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 months6
    E.8.9.1In the Member State concerned days19
    E.8.9.2In all countries concerned by the trial years3
    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: 150
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 50
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 28
    F.4.2.2In the whole clinical trial 200
    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)
    The patient will receive the standard of care for the disease
    Il paziente riceverà la cura disponibile per la patologia
    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 Decision2020-01-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-10-16
    P. End of Trial
    P.End of Trial StatusOngoing
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