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    Summary
    EudraCT Number:2017-004777-14
    Sponsor's Protocol Code Number:MSK-003
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2021-01-21
    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 number2017-004777-14
    A.3Full title of the trial
    Long term safety study of amifampridine phosphate in patients with MuSK antibody positive and AChR antibody positive myasthenia gravis patients
    Studio sulla sicurezza a lungo termine dell’amifampridina fosfato nei pazienti affetti da miastenia gravis positivi per anticorpi anti MuSK e anti AChR
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical study evaluating the long term safety of amifampridine phosphate in patients with MuSK antibody and AChR antibody positive myasthenia gravis
    Studio clinico che valuta la sicurezza a lungo termine del farmaco amifampridina fosfato in pazienti con miastenia gravis positivi per anticorpi anti MuSK e anti AChR
    A.3.2Name or abbreviated title of the trial where available
    MSK-003
    MSK-003
    A.4.1Sponsor's protocol code numberMSK-003
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    MuSK antibody and AChR antibody positive myasthenia gravis
    Miastenia gravis positiva agli anticorpi anti-MuSK e anti-AChR
    E.1.1.1Medical condition in easily understood language
    MuSK antibody and AChR antibody positive myasthenia gravis
    Miastenia gravis positiva per gli anticorpi anti-MuSK e anti-AChR
    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 evaluate the long-term safety and tolerability of amifampridine in patients with MuSK antibody positive myasthenia gravis and AChR antibody positive myasthenia gravis
    Valutare la sicurezza e la tollerabilità a lungo termine dell’amifampridina fosfato nei pazienti con miastenia gravis (MG) positivi per anticorpi MuSK e nei pazienti con miastenia gravis positivi per anticorpi AChR
    E.2.2Secondary objectives of the trial
    To assess the effect of amifampridine phosphate on Myasthenia Gravis Activities of Daily Living Score (MG-ADL).
    Valutare l’efficacia clinica dell’amifampridina fosfato nei pazienti con miastenia gravis (MG) nel corso del tempo, sulla base delle variazioni del punteggio della Scala della miastenia gravis per le attività della vita quotidiana (MG-ADL)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Participated in the MSK-002 study
    - 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
    - Female patients of childbearing potential must have a negative pregnancy test (urine human chorionic gonadotropin [HCG] at the end of MSK-002 study); 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
    - Avere partecipato allo studio MSK-002
    - Essere 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
    - Le pazienti di sesso femminile in età fertile devono presentare un test di gravidanza negativo (gonadotropina corionica umana [HCG] misurata nelle urine al termine dello studio MSK-002) e devono seguire un regime anticoncezionale efficace e affidabile durante lo studio e per un massimo di 30 giorni dopo l’interruzione del trattamento
    - Avere la capacità di partecipare allo studio, in base allo stato di salute generale del paziente e alla prognosi della malattia, secondo il caso, a giudizio dello sperimentatore, ed essere in grado di attenersi a tutti i requisiti del protocollo, compresa la compilazione dei questionari dello studio
    E.4Principal exclusion criteria
    - Epilepsy and currently on medication
    - Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator
    - Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study
    - Intolerable amifampridine-related side effects
    - Treatment with an investigational drug (other than amifampridine) or device 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
    - Epilessia e attualmente in terapia
    - Anomalie clinicamente significative all’ECG a 12 derivazioni, a giudizio dello sperimentatore
    - Allattamento o gravidanza allo screening oppure pianificazione di una gravidanza in qualsiasi momento durante lo studio
    - Effetti collaterali correlati all’amifampridina intollerabili
    - Trattamento con un farmaco (diverso dall’amifampridina) o un dispositivo sperimentale durante la partecipazione allo studio
    - Qualsiasi patologia che, a giudizio dello sperimentatore, potrebbe interferire con la partecipazione del paziente allo studio, comportare un rischio aggiuntivo per il paziente o confondere la valutazione del paziente
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of the study is the safety and the tolerability of amifampridine. Safety 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 findings, and concomitant medications will also be evaluated
    L’endpoint primario dello studio è la sicurezza del farmaco che sarà valutata tramite l’incidenza degli eventi avversi emergenti dal trattamento (TEAE), compresi gli eventi avversi seri (SAE). Inoltre, verranno valutati i parametri vitali, gli ECG a 12 derivazioni, i test clinici di laboratorio, i risultati dell’esame obiettivo e i farmaci concomitanti
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary endpoint will be assessed at months 3, 6, 9, 12, 15 and 21 from the start of treatment with amifampridine
    L’endpoint primario dello studio sarà valutato ai mesi 3, 6, 9, 12, 15 e 21 dall’inizio del trattamento con amifampridina
    E.5.2Secondary end point(s)
    To assess the clinical efficacy of amifampridine phosphate over time in patients with Myasthenia Gravis MG based on change in Myasthenia Gravis Activities of Daily Living Score (MG-ADL)
    Valutare l’efficacia clinica dell’amifampridina fosfato nei pazienti con miastenia gravis (MG) nel corso del tempo, sulla base delle variazioni del punteggio della Scala della miastenia gravis per le attività della vita quotidiana (MG-ADL)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Evaluation of MG-ADL will be made at months 3, 6, 9, 12, 15, and 21 from the start of treatment
    La valutazione della scala MG-ADL sarà effettuata ai mesi 3, 6, 9, 12, 15 e 21 dall’inizio del trattamento con amifampridina
    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 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 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 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 or until amifampridine is approved by regulatory agencies for the treatment of myastenia gravis or the development program of amifampridine is discontinued for this indication, whichever occurs first
    LVLS o fino a quando l'amifampridina viene approvata dalle agenzie regolatorie per il trattamento della miastenia gravis o lo sviluppo clinico di amifampridina viene interrotto per l' indicazione in studio, a seconda di quale evento si verifichi per primo
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    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: 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)
    Patients will be monitored and treated according to local clinical practice
    I pazienti saranno seguiti e trattati in accordo alla pratica clinica locale
    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 Decision2018-06-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-04-11
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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