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    Summary
    EudraCT Number:2017-004600-22
    Sponsor's Protocol Code Number:SMA-001
    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:2021-04-07
    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 number2017-004600-22
    A.3Full title of the trial
    A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients with Spinal Muscular Atrophy (SMA) Type 3
    Studio clinico randomizzato, controllato con placebo, cross-over per valutare la sicurezza e l¿efficacia di amifampridina fosfato in pazienti deambulanti con Atrofia Muscolare Spinale (SMA) tipo 3.
    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 Ambulatory Patients with Spinal Muscular Atrophy (SMA) Type 3.
    Studio clinico che valuta l'effetto 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-001
    SMA-001
    A.4.1Sponsor's protocol code numberSMA-001
    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 Address335 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.3.9.3Other descriptive name3,4-Diaminopyridine 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
    Spinal Muscular Atrophy (SMA) Type 3
    Atrofia Muscolare Spinale (SMA) tipo 3
    E.1.1.1Medical condition in easily understood language
    Spinal Muscular Atrophy
    Atrofia Muscolare Spinale
    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 characterize the overall safety and tolerability of amifampridine phosphate compared with placebo in patients with SMA Type 3.
    To assess the clinical efficacy of amifampridine phosphate compared with placebo in ambulatory patients with SMA Type 3 using the Hammersmith Functional Motor Scale Expanded (HFMSE).
    Definire la sicurezza e la tollerabilit¿ complessive di amifampridina fosfato rispetto al placebo in pazienti deambulanti con SMA di Tipo 3.
    Valutare l¿efficacia clinica di amifampridina fosfato rispetto al placebo in pazienti deambulanti con SMA di Tipo 3 mediante la scala Hammersmith Functional Motor Scale Expanded (HFMSE).
    E.2.2Secondary objectives of the trial
    To assess the clinical efficacy of amifampridine phosphate compared with placebo using the six-minute walk test, timed tests, and individual quality of life (INQoL) and pediatric quality of life inventory (PEDSQoL) assessments.
    Valutare l¿efficacia clinica di amifampridina fosfato rispetto al placebo mediante le valutazioni del test del cammino dei 6 minuti (6MWT), del test a tempo (timed tests) e della qualit¿ individuale della vita (INQoL) e della qualit¿ della vita in soggetti pediatrici (PEDSQoL).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Inclusion criteria:
    1.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.
    2.Male or female between the ages of 6 and 50 years.
    3.Genetically confirmed diagnosis of SMA Type 3.
    4.Able to walk independently for at least 30 meters.
    5.Do not to take Nusinersen for the treatment of SMA (Nusinersen should be stopped at least 6 months before screening). Salbutamol is permitted only if the dose has been stable during the 6 months before screening.
    6.Able to swallow oral medication.
    7.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.
    8.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.
    Criteri di inclusione
    1.Essere disposti ed in grado di fornire il consenso informato scritto dopo che la tipologia dello studio sia stata spiegata e prima che sia iniziata qualsiasi procedura correlata con la ricerca.
    2.Maschi o femmine di età compresa tra 6 e 50 anni.
    3.Diagnosi di SMA di Tipo 3 confermata geneticamente.
    4. Essere in grado di camminare in modo indipendente per almeno 30 metri.
    5.Non assumere Nusinersen per il trattamento della SMA (l’assunzione di Nusinersen dovrà essere stata interrotta almeno 6 mesi prima della visita di Screening). L’assunzione di Salbutamolo è permessa solo se la dose di farmaco è rimasta stabile nel corso dei 6 mesi precedenti alla visita di screening.
    6.Essere in grado di assumere farmaci per via orale.
    7.Pazienti donne in età fertile devono avere un test di gravidanza negativo (gonadotropina corionica [HCG] nel siero umano allo Screening) e devono utilizzare un metodo contraccettivo efficace e affidabile durante lo studio e per i 30 giorni successivi alla fine del trattamento.
    8.Essere 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
    Exclusion criteria:
    1.Epilepsy and currently on medication for epilepsy.
    2.Concomitant use of medicinal products with a known potential to cause QTc prolongation.
    3.Patients with long QT syndromes.
    4.An electrocardiogram (ECG) within 6 months before starting treatment that shows clinically significant abnormalities, in the opinion of the Investigator.
    5.Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
    6.Treatment with an investigational drug (other than amifampridine), device, or biological agent within 6 months prior to Screening or while participating in this study.
    7.Surgery for scoliosis or joint contractures within the previous 6 months.
    8.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.
    9.History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s).
    10.Less than a 3-point improvement in HFSME from start of the Open label Run -in period to end of Run-in (Day 0).
    Criteri di esclusione:
    1.Epilessia e attualmente in terapia per l'epilessia.
    2.Uso concomitante di prodotti medicinali noti per poter potenzialmente causare un prolungamento del QTc.
    3.Pazienti con sindrome del QT lungo.
    4.Un elettrocardiogramma (ECG) eseguito entro i 6 mesi precedenti all’inizio del trattamento che mostri, secondo il parere dello Sperimentatore, anormalità clinicamente significative.
    5.In allattamento o in stato di gravidanza alla visita di Screening o che pianifichino una gravidanza nel corso del periodo di studio.
    6.Trattamento con un farmaco sperimentale (diverso da amifampridina), un dispositivo medico, o con agenti biologici entro i 6 mesi precedenti alla visita di Screening o nel corso del periodo di studio.
    7.Interventi chirurgici per scoliosi o contratture articolari entro i 6 mesi precedenti.
    8.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.
    9.Storia di allergia a farmaci a qualsiasi sostanza contenente piridina o a uno qualsiasi degli eccipienti di amifampridina.
    10.Un miglioramento inferiore a 3-punti del punteggio HFSME tra l’inizio della fase di Run-in in Aperto e la fine della fase di Run-in (Giorno 0).
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint of the study is the change in HFMSE score from Day 0 (baseline) for SMA subjects treated with amifampridine and placebo.
    L'endpoint primario di efficacia dello studio è la variazione dello score totale di HFMSE rispetto alla visita basale per i soggetti con SMA trattati con amifampridina e placebo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    About 60 days from the start date of the treatment with IMP
    Circa 60 giorni dalla data di inizio del trattamento con IMP
    E.5.2Secondary end point(s)
    The change in the six-minute walk test from Day 0(baseline) ; The evaluation of the timed tests ; The change in the quality of life assessments from Day 0 (baseline)

    ; 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 test del cammino dei 6 minuti rispetto alla visita basale; Valutazione degli items del test a tempo; Variazione della valutazione della qualit¿ della vita rispetto alla visita basale; La sicurezza del farmaco sperimentale verr¿ valutata in base all'incidenza degli eventi avversi verificatisi nel corso del trattamento (TEAEs), inclusi gli eventi avversi seri (SAEs). Saranno anche valutati: segni vitali +, ECG a 12 derivazioni, test clinici di laboratorio, esame obiettivo e trattamenti concomitanti.
    E.5.2.1Timepoint(s) of evaluation of this end point
    About 60 days from the start date of the treatment with IMP; About 60 days from the start date of the treatment with IMP; About 60 days from the start date of the treatment with IMP; 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 60 giorni dalla data di inizio del trattamento con IMP; Circa 60 giorni dalla data di inizio del trattamento con IMP
    ; Circa 60 giorni dalla data di inizio del trattamento con IMP; 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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 No
    E.8.1.6Cross over Yes
    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 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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months12
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months12
    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)
    There will be an open label study that patients may enter if they demonstrate benefit from amifampridine phosphate
    Ci sar¿ 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 Decision2018-04-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-03-07
    P. End of Trial
    P.End of Trial StatusCompleted
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