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    Summary
    EudraCT Number:2019-001565-33
    Sponsor's Protocol Code Number:RA101495-02.302
    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:2020-01-14
    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 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
    Estudio de extensión abierto, de fase 3, multicéntrico, de zilucoplan en pacientes con miastenia gravis generalizada
    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
    Estudio de extensión abierto de zilucoplan en pacientes con miastenia gravis generalizada
    A.3.2Name or abbreviated title of the trial where available
    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.5.6E-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.3.4Pharmaceutical form Solution for injection in pre-filled syringe
    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 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 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 gravis generalizada
    E.1.1.1Medical condition in easily understood language
    Myasthenia Gravis
    Miastenia gravis
    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
    E.2.2Secondary objectives of the trial
    Not applicable
    - Proporcionar acceso a zilucoplan a pacientes con MGg que hayan completado un estudio de cualificación de zilucoplan patrocinado por Ra Pharmaceuticals y que deseen continuar recibiendo zilucoplan
    - Evaluar la eficacia a largo plazo de zilucoplan en pacientes con MGg que hayan completado un estudio clínico de cualificación de zilucoplan patrocinado por Ra Pharmaceuticals
    - Evaluar la seguridad y tolerabilidad a largo plazo de zilucoplan en pacientes con MGg que hayan completado un estudio clínico de cualificación de zilucoplan patrocinado por Ra Pharmaceuticals
    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
    Para ser aptos para el estudio, los pacientes deberán cumplir TODOS los criterios de inclusión siguientes:
    1. Finalización de un estudio de cualificación de zilucoplan patrocinado por Ra Pharmaceuticals
    2. Capaz de proporcionar un consentimiento informado, incluida la firma y la fecha del formulario de consentimiento informado (FCI)
    3. Vacunación con una vacuna meningocócica tetravalente y, cuando esté disponible, la vacuna contra el meningococo del serotipo B. También deberá administrarse una vacuna de refuerzo si está indicado clínicamente, de acuerdo con el tratamiento habitual local, a los pacientes que hayan sido vacunados previamente contra la Neisseria meningitidis
    4. Las pacientes fértiles deberán obtener resultado negativo en una prueba de embarazo en orina en las 24 horas previas a la primera dosis del fármaco del estudio
    5. Las pacientes fértiles sexualmente activas (es decir, las mujeres que no sean posmenopáusicas o que no hayan tenido una histerectomía, ooforectomía bilateral o ligadura de trompas bilateral) y todos los pacientes varones (que no hayan sido esterilizados quirúrgicamente por vasectomía) deben comprometerse a utilizar un método anticonceptivo eficaz durante el estudio. A efectos de este protocolo, las mujeres posmenopáusicas son las mujeres que han pasado 12 meses consecutivos sin menstruación
    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
    Los pacientes que cumplan CUALQUIERA de los siguientes criterios de exclusión se excluirán del estudio:
    1. Mujeres embarazadas, que tengan previsto quedarse embarazadas o en periodo de lactancia
    2. Con la excepción del ensayo sobre zilucoplan anterior, la participación en otro ensayo clínico concurrente que involucre una intervención terapéutica experimental (se permite la participación en estudios observacionales y/o estudios de registro)
    3. Incapacidad o falta de disposición para cumplir con los requisitos del estudio
    4. No se inició ninguna medicación no permitida según los criterios de exclusión del estudio de cualificación de zilucoplan ni se alteró la dosis de ninguna otra medicación concomitante, a menos que esté médicamente indicado
    5. Cualquier afección médica nueva o que empeore (desde que ingresara en el estudio de cualificación de zilucoplan) o cualquier otra razón que, en opinión del investigador o el Promotor, descalifique al sujeto de participar en este estudio
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy endpoints:
    •MG-ADL Score
    •QMG Score
    •MG-QOL15r
    •MGC Score
    Safety endpoints
    Evaluaciones de la eficacia:
    - Puntuación de actividades de la vida diaria-miastenia grave (AVD-MG)
    - Puntuación cuantitativa de miastenia grave (QMG)
    - Puntuación de calidad de vida revisada-miastenia grave (MG-QOL15r)
    - Puntuación compuesta de la miastenia grave (MGC)
    Evaluaciones de seguridad
    E.5.1.1Timepoint(s) of evaluation of this end point
    during each visit
    Durante cada visita
    E.5.2Secondary end point(s)
    Not applicable
    No aplica
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    No aplica
    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 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 No
    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 concerned3
    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 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
    Canada
    France
    Germany
    Italy
    Japan
    Norway
    Spain
    United Kingdom
    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
    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 months9
    E.8.9.1In the Member State concerned days14
    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
    El paciente recibirá el tratamiento habitual para la indicación.
    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 Decision2020-05-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-11-13
    P. End of Trial
    P.End of Trial StatusOngoing
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