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    Summary
    EudraCT Number:2018-000160-28
    Sponsor's Protocol Code Number:SMA-002
    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:2021-05-24
    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 number2018-000160-28
    A.3Full title of the trial
    Long term safety study of amifampridine phosphate in ambulatory patients with Spinal Muscular Atrophy (SMA) type 3
    Long term safety study of amifampridine phosphate in ambulatory patients with Spinal Muscular Atrophy (SMA) type 3
    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 ambulatory patients with Spinal Muscular Atrophy (SMA) type 3
    Studio clinico che valuta la sicurezza a
    lungo termine del farmaco amifampridina fosfato in pazienti deambulanti con Atrofia Muscolare Spinale (SMA) tipo 3
    A.3.2Name or abbreviated title of the trial where available
    SMA-002
    SMA-002
    A.4.1Sponsor's protocol code numberSMA-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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Spinal Muscular Atrophy (SMA) Type 3
    Atrofia Muscolare Spinale (SMA) tipo 3
    E.1.1.1Medical condition in easily understood language
    Spinal Muscular Atrophy (SMA)
    Atrofia Muscolare Spinale (SMA)
    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 20.1
    E.1.2Level PT
    E.1.2Classification code 10041582
    E.1.2Term Spinal muscular atrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic 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 phosphate in patients with SMA Type 3
    Definire la sicurezza e la tollerabilità a lungo termine di amifampridina fosfato in pazienti con SMA di Tipo 3
    E.2.2Secondary objectives of the trial
    To assess the clinical efficacy of amifampridine phosphate over time in patients with SMA Type 3 based on changes in quality of life (QoL)
    Valutare, nel corso del tempo, l’efficacia clinica di amifampridina fosfato in pazienti con SMA di Tipo 3 in base ai cambiamenti rilevati mediante il questionario sulla qualità della vita (QoL)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Patients participated in the SMA-001 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 defined according to the Clinical Trial Facilitation Group - CTFG guidelines* must have a negative pregnancy test (urine human chorionic gonadotropin [HCG] at the end of SMA-001 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.

    * According to CTFG guidelines a woman is considered of childbearing potential, i.e. fertile,
    following menarche and until becoming post-menopausal unless permanently sterile.
    Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral
    oophorectomy. A postmenopausal state is defined as no menses for 12 months without an
    alternative medical cause.
    - Pazienti che hanno preso parte allo studio SMA-001
    - Disposti e in grado di fornire il consenso informato scritto dopo che la tipologia dello studio sia stata spiegata e prima di che sia iniziata qualsiasi procedura correlata con la ricerca
    - Pazienti donne in età fertile definita in accordo ai criteri della linea guida del Clinical Trial Facilitation Group- CTFG (si faccia riferimento al protocollo) devono avere un test di gravidanza negativo (gonadotropina corionica [HCG] umana nelle urine alla fine dello studio SMA-001) e devono utilizzare un metodo contraccettivo efficace e affidabile in accordo alle raccomandazioni della linea guida del CTFG (si faccia riferimento al protocollo) durante lo studio e per i 30 giorni successivi alla fine del trattamento
    - In grado di partecipare allo studio in base allo stato generale di salute del paziente e alla prognosi di malattia, come applicabile, in base al parere dello Sperimentatore; e in grado di soddisfare tutti i requisiti del protocollo, compreso il completamento dei questionari previsti dallo studio
    E.4Principal exclusion criteria
    - Epilepsy and currently on medication
    - Uncontrolled asthma
    - Concomitant use with sultopride
    - Concomitant use with medicinal products with a narrow therapeutic window
    - Concomitant use with medicinal products with a known to cause QTc prolongation
    - Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator
    - Subjects with congenital QT syndromes
    - 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
    - History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s)
    - Epilessia e attualmente in terapia per l'epilessia
    - Asma incontrollata
    - Uso concomitante con sultopride
    - Uso concomitante con medicinali a basso indice terapeutico
    - Uso concomitante con medicinali di cui sia noto il potenziale di causare prolungamento dell'intervallo QTc
    - Un elettrocardiogramma (ECG a 12 derivazioni) che, secondo il parere dello Sperimentatore, presenta anormalità clinicamente significative
    - Sindrome congenita del QT
    - In allattamento o in stato di gravidanza alla visita di Screening o che pianifichino una gravidanza nel corso del periodo di studio
    - Effetti collaterali intollerabili correlati al trattamento con amifampridina.
    - Trattamento con un farmaco sperimentale (diverso da amifampridina) o con un dispositivo medico nel corso del periodo di studio
    - Qualsiasi condizione medica che, secondo il parere dello Sperimentatore, potrebbe interferire con la partecipazione del paziente allo studio, che potrebbe comportare un ulteriore rischio per il paziente, o che potrebbe rendere poco chiara la valutazione del paziente
    - Storia di allergia ai farmaci con qualsiasi sostanza contenente piridina o a qualsiasi eccipiente di amifampridina
    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 è la valutazione della sicurezza dell'amifampridina fosfato che verrà valutata in base all’incidenza degli eventi avversi verificatisi nel corso del trattamento (TEAEs), inclusi gli eventi avversi seri (SAEs). Saranno anche valutati: i risultati ottenuti dalle valutazioni dei parametri vitali, degli ECG a 12 derivazioni, dei test clinici di laboratorio, dell’esame obiettivo e dei trattamenti concomitanti
    E.5.1.1Timepoint(s) of evaluation of this end point
    The study primary endpoint will be assessed at months 3, 6, 9, 12, 15, 21 and 27 from the start of treatment with amifampridine phosphate
    L’endpoint primario dello studio sarà valutato ai mesi 3, 6, 9, 12, 15, 21 e 27 dall’inizio del trattamento con amifampridina fosfato
    E.5.2Secondary end point(s)
    The secondary endpoint is the efficacy of amifampridine phosphate based on changes of the Quality of Life (QoL) scores over time. The Individualized Quality of Life for Neuromuscular disease (INQoL) or the Pediatric Quality of Life (PEDSQLTM) will be assessed for adult or pediatric patients, respectively
    L’endpoint secondario è l'efficacia dell'amifampridina fosfato e sarà valutato in base ai cambiamenti nel corso del tempo nei punteggi dei questionari sulla qualità della vita (QoL). Nei pazienti adulti sarà utilizzato il questionario Individualized Quality of Life for Neuromuscular disease (INQoL), mentre nei pazienti pediatrici sarà utilizzato il questionario Pediatric Quality of Life (PEDSQL)
    E.5.2.1Timepoint(s) of evaluation of this end point
    The Quality of Life (QoL) will be assessed at months 3, 6, 9, 12, 15, 21 and 27 from the start of treatment with amifampridine phosphate
    La valutazione della qualità della vita sarà effettuata ai mesi 3, 6, 9, 12, 15, 21 e 27 dall’inizio del trattamento con amifampridina fosfato
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    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 No
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    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 Spinal Muscular Atrophy (SMA) type 3 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 Atrofia Muscolare Spinale (SMA) di tipo 3 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 Yes
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Minors
    Minori
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 12
    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 Decision2018-11-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-13
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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