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    Summary
    EudraCT Number:2019-001564-30
    Sponsor's Protocol Code Number:RA101495-02.301(RAISE)
    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:2019-11-11
    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 number2019-001564-30
    A.3Full title of the trial
    A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects with Generalized Myasthenia Gravis
    Estudio de fase 3, multicéntrico, aleatorizado, doble ciego, controlado con placebo, para confirmar la seguridad, tolerabilidad y eficacia 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
    A Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Patients with Generalized Myasthenia Gravis
    Estudio para confirmar la seguridad, tolerabilidad y eficacia de zilucoplan en pacientes con miastenia gravis generalizada
    A.3.2Name or abbreviated title of the trial where available
    RAISE
    A.4.1Sponsor's protocol code numberRA101495-02.301(RAISE)
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection in pre-filled syringe
    D.8.4Route of administration of the placeboSubcutaneous use
    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 confirm the efficacy of zilucoplan in subjects with gMG
    •To confirm the safety and tolerability of zilucoplan in subjects with gMG
    - Confirmar la eficacia de zilucoplan en pacientes con MGg
    - Confirmar la seguridad y tolerabilidad de zilucoplan en pacientes con MGg
    E.2.2Secondary objectives of the trial
    Not Applicable
    No aplica
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    To be eligible for this study, subjects must meet ALL the following inclusion criteria:

    1.Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IV] at Screening
    2.Positive serology for acetylcholine receptor (AChR) binding autoantibodies
    3.MG-ADL Score of ≥6 at Screening and Baseline
    4.QMG Score of ≥12 at Screening and Baseline (off acetylcholinesterase inhibitor therapy for at least 10 hours)
    5.No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the 12-week treatment period
    6.No change in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the 12-week treatment period
    Para ser aptos para el estudio, los pacientes deberán cumplir TODOS los criterios de inclusión siguientes:

    1. Diagnóstico de MGg [clase II-IV según la Myasthenia Gravis Foundation of America (MGFA)] en la selección
    2. Pruebas serológicas positivas para autoanticuerpos de unión al receptor de la acetilcolina (AChR)
    3. Puntuación de AVD-MG ≥6 en la selección y el periodo inicial
    4. Puntuación QMG ≥12 en la selección y el periodo inicial (sin recibir tratamientos con inhibidores de la acetilcolinesterasa durante al menos 10 horas)
    5. Ningún cambio en la dosis de corticoesteroides durante al menos 30 días antes del inicio del estudio ni previsión de que ocurran cambios durante el periodo de tratamiento de 12 semanas
    6. Ningún cambio en el tratamiento inmunosupresor, incluida la dosis, durante al menos 30 días antes del periodo inicial ni previsión de que ocurran cambios durante el periodo de tratamiento de 12 semanas
    E.4Principal exclusion criteria
    Subjects who meet ANY of the following exclusion criteria must be excluded from the study:
    1.Thymectomy within 12 months prior to baseline or scheduled to occur during the 12-week study
    2.History of meningococcal disease
    3.Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Baseline
    Los pacientes que cumplan CUALQUIERA de los siguientes criterios de exclusión se excluirán del estudio:

    1. Timectomía en los 12 meses anteriores al inicio del estudio o programada para realizarse durante el estudio de 12 semanas
    2. Antecedentes de enfermedad meningocócica
    3. Infección sistémica actual o reciente en las 2 semanas anteriores al periodo inicial o infección que precisó antibióticos intravenosos (i.v.) en las 4 semanas anteriores al periodo inicial
    E.5 End points
    E.5.1Primary end point(s)
    •Change from Baseline (CFB) to Week 12 in the MG-ADL Score
    - Variación desde el periodo inicial hasta la semana 12 en la puntuación de AVD-MG
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 12
    Semana 12
    E.5.2Secondary end point(s)
    •CFB to Week 12 in the QMG Score
    •CFB to Week 12 in the MG Composite (MGC)
    •CFB to Week 12 in the MG-QOL15r Survey
    - Variación desde el periodo inicial hasta la semana 12 en la puntuación QMG
    - Variación desde el periodo inicial hasta la semana 12 en la puntuación compuesta de MG (MGC)
    - Variación desde el periodo inicial hasta la semana 12 en la encuesta de calidad de vida MG-QOL15r
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 12
    Semana 12
    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 Yes
    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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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) No
    E.8.2.2Placebo Yes
    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 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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days12
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months10
    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: 100
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 30
    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 130
    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)
    At the end of the Treatment Period, all subjects will have the option to receive zilucoplan in a separate Extension Study, provided they meet the Extension Study inclusion criteria
    Al final del periodo de tratamiento, todos los pacientes tendrán la opción de recibir zilucoplan en un estudio de extensión independiente, siempre que cumplan con los criterios de inclusión del estudio de extensión.
    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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