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    Summary
    EudraCT Number:2017-004018-25
    Sponsor's Protocol Code Number:MSK-002
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-01-19
    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 number2017-004018-25
    A.3Full title of the trial
    A Randomized, Placebo-Controlled, Parallel Group Study to Evaluate the Effect of Amifampridine Phosphate in Patients with MuSK Antibody Positive Myasthenia Gravis, and a Sample of AChR Antibody Positive Myasthenia Gravis Patients
    Studio randomizzato, controllato verso placebo, a gruppi paralleli per valutare l’effetto di amifampridina fosfato in pazienti con miastenia gravis positiva agli anticorpi anti-MuSK e in un campione di pazienti con miastenia gravis positiva agli anticorpi anti-AChR
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical study evaluating the effect of Amifampridine phosphate in patients with MuSK antibody positive myasthenia gravis, and a sample of AchR antibody positive myasthenia gravis patients
    Studio clinico che valuta l’effetto del farmaco amifampridina fosfato in pazienti con miastenia gravis positiva agli anticorpi anti-MuSK e in un campione di pazienti con miastenia gravis positiva agli anticorpi anti-AChR
    A.3.2Name or abbreviated title of the trial where available
    MSK-002
    MSK-002
    A.4.1Sponsor's protocol code numberMSK-002
    A.5.4Other Identifiers
    Name:INDNumber:106263
    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 SponsorCATALYST 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 supportCatalyst Pharmaceuticals inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCatalyst Pharmaceuticals Inc.
    B.5.2Functional name of contact pointGary Ingenito
    B.5.3 Address:
    B.5.3.1Street Address355 Alhambra Circle, Suite 1250
    B.5.3.2Town/ cityCoral Gables
    B.5.3.3Post codeFL 33134
    B.5.3.4CountryUnited States
    B.5.4Telephone number3054203200
    B.5.5Fax number3054203200
    B.5.6E-mailgingenito@catalystpharma.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 FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE
    D.2.1.1.2Name of the Marketing Authorisation holderBIOMARIN EUROPE LTD
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameAmifampridina fosfato
    D.3.2Product code [Amifampridina fosfato]
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAMIFAMPRIDINA
    D.3.9.1CAS number 446254-47-3
    D.3.9.2Current sponsor codeDAPP or 3,4-DAP Phosphate
    D.3.9.4EV Substance CodeSUB28846
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 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 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 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 placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    MuSK antibody positive myasthenia gravis
    Miastenia gravis positiva agli anticorpi anti-MuSK
    E.1.1.1Medical condition in easily understood language
    MuSK antibody positive myasthenia gravis
    Miastenia gravis positiva agli anticorpi anti-MuSK
    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 21.1
    E.1.2Level PT
    E.1.2Classification code 10028417
    E.1.2Term Myasthenia gravis
    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 characterize the overall safety and tolerability of amifampridine phosphate compared with placebo in patients with MuSK antibody positive myasthenia gravis.
    To assess the clinical efficacy of amifampridine phosphate compared with placebo in patients with MuSK antibody positive myasthenia gravis based on change in Myasthenia Gravis Activities of Daily Living Score (MG-ADL)
    Valutare la sicurezza e la tollerabilità complessiva di amifampridina fosfato rispetto al placebo in pazienti con miastenia gravis positiva agli anticorpi anti-MuSK.
    Valutare l’efficacia clinica di amifampridina fosfato rispetto al placebo in pazienti affetti da miastenia gravis positiva agli anticorpi anti-MuSK sulla base delle variazioni del punteggio del questionario Miastenia gravis - Attività quotidiane specifiche (Myasthenia Gravis Activities of Daily Living Score, MG-ADL)
    E.2.2Secondary objectives of the trial
    To assess the clinical efficacy of amifampridine phosphate compared with placebo using the Quantitative Myasthenia Gravis (QMG) score.
    To assess the safety and efficacy of amifampridine phosphate compared with placebo in a sample of patients with AChR antibody positive myasthenia gravis
    Valutare l’efficacia clinica di amifampridina fosfato rispetto al placebo utilizzando il punteggio quantitativo del Quantitative Myasthenia Gravis, QMG test.
    Valutare la sicurezza e l’efficacia di amifampridina fosfato rispetto al placebo in un campione di pazienti con miastenia gravis positiva agli anticorpi anti AChR
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Willing and able to provide written informed consent after the nature of the study has been explained and before the start of any research-related procedures
    - Male or female =18 years of age
    - Positive serologic test for anti-MuSK antibodies or anti-AChR antibodies as confirmed at screening or by previous antibody test, with report available
    - Confirmatory electromyography (EMG) or EMG report
    - Myasthenia Gravis Foundation of America (MGFA) Class II to IV at screening
    - MG-ADL score of =6 at screening, with more than 50% of this score attributed to non-ocular items
    - Patients receiving steroids and/or pyridostigmine should not have any modification of drug regimen during the month before screening
    - Female patients of childbearing potential must have a negative pregnancy test (serum human chorionic gonadotropin [HCG] at screening); and must practice an effective, reliable contraceptive regimen during the study and for up to 30 days following discontinuation of treatment
    - Ability to participate in the study based on overall health of the patient and disease prognosis, as applicable, in the opinion of the Investigator; and able to comply with all requirements of the protocol, including completion of study questionnaires

    - Soggetti disposti e in grado di fornire il consenso informato scritto dopo la spiegazione della natura dello studio e prima dell’inizio di qualsiasi procedura correlata alla ricerca
    - Sesso maschile o femminile di età = 18 anni
    - Test sierologico positivo agli anticorpi anti-MuSK o agli anticorpi anti-AChR confermato allo screening o tramite test anticorpale precedente, con referto disponibile
    - Elettromiografia confermativa (EMG) o referto EMG.
    - Classe da II a IV della Myasthenia Gravis Foundation of America (MGFA) allo screening
    - Punteggio MG-ADL di = 6 allo screening, con più del 50% del punteggio attribuito alle voci non oculari
    - I pazienti che ricevono steroidi e/o piridostigmina non devono variare in alcun modo il regime farmacologico durante il mese precedente allo screening
    - Le pazienti di sesso femminile in età fertile devono presentare un test di gravidanza negativo (gonadotropina corionica umana [Human Chorionic Gonadotropin, HCG] nel siero allo screening); e devono praticare un regime anticoncezionale efficace e affidabile durante lo studio e per un massimo di 30 giorni dopo l’interruzione del trattamento
    - Capacità di partecipare allo studio basata sulla salute generale del paziente e sulla prognosi della malattia, a seconda dei casi, secondo l’opinione dello sperimentatore; e in grado di rispettare tutti i requisiti del protocollo, compresa la compilazione dei questionari dello studio
    E.4Principal exclusion criteria
    - Epilepsy and currently on medication
    - Concomitant use of medicinal products with a known potential to cause QTc prolongation
    - Patients with long QT syndromes
    - History of thymectomy within 12 months before screening
    - An electrocardiogram (ECG) within 6 months before starting treatment that shows clinically significant abnormalities, in the opinion of the Investigator
    - Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study
    - Patients receiving immunomodulatory treatment (e.g. plasma exchange [PE], therapeutic plasma exchange [TPE], intravenous immunoglobulin G [IVIG]) should not have any treatment in the previous 4 weeks prior to Randomization or at any time during the study
    - Use of rituximab or other similar biologic medications for immunomodulation within 6 months prior to screening
    - Treatment with an investigational drug (other than amifampridine), device, or biological agent within 60 days prior to screening or while participating in this study
    - Any medical condition that, in the opinion of the Investigator, might interfere with the patient’s participation in the study, poses an added risk for the patient, or confound the assessment of the patient
    - History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s)
    - Epilessia e attualmente sotto farmaci
    - Uso concomitante di prodotti medicinali con noto potenziale di causare prolungamento del QTc
    - Pazienti con sindrome del QT lungo
    - Anamnesi di timectomia entro 12 mesi prima dello screening
    - Un elettrocardiogramma (ECG) entro i 6 mesi precedenti all’inizio del trattamento che mostra anomalie clinicamente significative secondo l’opinione dello sperimentatore
    - In allattamento o in gravidanza allo screening o che pianificano una gravidanza in qualsiasi momento durante lo studio
    - I pazienti che ricevono un trattamento immunomodulante (ad es. plasmaferesi [plasma exchange, PE], plasmaferesi terapeutica [therapeutic plasma exchange, TPE], immunoglobuline G endovena [intravenous immunoglobulin G, IVIG]) non devono sottoporsi ad alcun trattamento nelle 4 settimane precedenti la randomizzazione o in qualsiasi momento durante lo studio
    - Uso di rituximab o di altri farmaci biologici simili per l’immunomodulazione entro i 6 mesi precedenti allo screening
    - Trattamento con un farmaco (diverso dall’amifampridina), un dispositivo o un agente biologico sperimentale entro i 60 giorni precedenti allo screening o durante la partecipazione a questo studio
    - Qualsiasi condizione medica che, secondo l’opinione dello sperimentatore, potrebbe interferire con la partecipazione del paziente allo studio, rappresentare un rischio aumentato per il paziente o confondere la valutazione del paziente
    - Anamnesi di allergia farmacologica a qualsiasi sostanza contenente piridina o a qualsiasi eccipiente dell’amifampridina
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint of the study is the change in MG-ADL score from Day 0 (baseline) for MuSK-MG subjects treated with amifampridine and placebo
    L' endpoint primario di efficacia è la variazione del punteggio totale MG-ADL rispetto al basale nei pazienti con miastenia gravis positiva per gli anticorpi anti-Musk trattati con amifampridina e placebo
    E.5.1.1Timepoint(s) of evaluation of this end point
    About 38 days after the start of the IMP intake in the run-in phase
    Circa 38 giorni dopo l' inizio della assunzione del farmaco sperimentale nella fase di run-in
    E.5.2Secondary end point(s)
    The change in QMG score from Day 0 (baseline) for MuSK-MG subjects treated with amifampridine and placebo; The proportion of subjects with a change of 2, or more, in MG-ADL score for MuSK-MG subject treated with amifampridine and placebo; The proportion of subjects with a change of 3, or more, in QMG score for MuSK-MG subjects treated with amifampridine and placebo; Safety of IMP will be assessed by the incidence of treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs). Vital signs, 12-lead ECGs, clinical laboratory tests, physical examination, and concomitant medications will also be evaluated
    Variazione del punteggio totale QMG rispetto al basale nei pazienti con miastenia gravis positiva per gli anticorpi anti-MuSK trattati con amifampridina e placebo; Proporzione di pazienti con miastenia gravis positiva per gli anticorpi anti-MuSK trattati con amifampridina e placebo con almeno una variazione di 2 punti del punteggio MG-ADL rispetto al basale; Proporzione di pazienti con miastenia gravis positiva per gli anticorpi anti-MuSK trattati con amifampridina e placebo con almeno una variazione di 3 punti del punteggio QMG rispetto al basale; La sicurezza del farmaco sperimentale sarà valutata tramite l’incidenza degli eventi avversi emergenti dal trattamento (treatment-emergent adverse events, TEAE), compresi gli eventi avversi seri (serious adverse events, SAE). Saranno valutati anche i segni vitali, gli ECG a 12 derivazioni, i test clinici di laboratorio, l’esame obiettivo e i farmaci concomitanti
    E.5.2.1Timepoint(s) of evaluation of this end point
    About 38 days after the start of the IMP intake in the run-in phase; About 38 days after the start of the IMP intake in the run-in phase; About 38 days after the start of the IMP intake in the run-in phase; The study period during which all non-serious AEs will be reported begins after the first administration of study drug through the termination visit or at the early termination visit. After informed consent but prior to initiation of study treatment, only SAEs associated with any protocol-imposed interventions will be reported. The reporting period for SAEs begins after informed consent is obtained and continues through 4 weeks after the last visit
    Circa 38 giorni dopo l' inizio della assunzione del farmaco sperimentale nella fase di run-in; Circa 38 giorni dopo l' inizio della assunzione del farmaco sperimentale nella fase di run-in; Circa 38 giorni dopo l' inizio della assunzione del farmaco sperimentale nella fase di run-in; Gli eventi avversi non seri saranno registrati dalla prima dose di farmaco sperimentale fino alla visita di fine trattamento. Nel periodo compreso tra la sottoscrizione del consenso informato e l’inizio del trattamento sperimentale saranno registrati solo gli eventi avversi seri (SAE) correlati ad una procedura prevista dal protocollo. I SAE verranno registrati fino a 4 settimane dopo l’ultima 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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    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 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 Yes
    E.8.1.7.1Other trial design description
    Fase di run-in in aperto con amifampridina seguita da una fase randomizzata vs PL, in doppio cieco
    Open-label run-in with amifampridine followed by double-blind randomized (amifampridine vs PL) phase
    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 concerned5
    E.8.5The trial involves multiple Member States No
    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
    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 months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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: 63
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 7
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 70
    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)
    There will be an open label study that patients may enter if they demonstrate benefit from amifampridine phosphate
    Ci sarà una sperimentazione clinica in aperto alla quale potranno partecipare i soggetti che hanno dimostrato di avere beneficio dalla amifampridina fosfato
    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 Decision2018-02-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-12-13
    P. End of Trial
    P.End of Trial StatusCompleted
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