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    Summary
    EudraCT Number:2014-002228-28
    Sponsor's Protocol Code Number:STEMALS-II
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2014-06-12
    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 number2014-002228-28
    A.3Full title of the trial
    A double blind, placebo controlled, parallel groups, multicenter study on filgrastim in amyotrophic lateral sclerosis
    STUDIO MULTICENTRICO, A DOPPIO CIECO, CONTROLLATO CON PLACEBO, A GRUPPI PARALLELI SUL TRATTAMENTO CON FILGRASTIM NELLA SCLEROSI LATERALE AMIOTROFICA
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    STEMALS-II
    STEMALS-II
    A.3.2Name or abbreviated title of the trial where available
    STEMALS-II
    STEMALS-II
    A.4.1Sponsor's protocol code numberSTEMALS-II
    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 SponsorUniversità degli Studi di Torino e Azienda Ospedaliera Città della Salute e della Scienza di Torino
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAIFA
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversità degli Studi di Torino e Azienda Ospedaliera Città della Salute e della Scienza di Torino
    B.5.2Functional name of contact pointCRESLA, SCDU Neurologia 2
    B.5.3 Address:
    B.5.3.1Street AddressVia Cherasco 15
    B.5.3.2Town/ cityTORINO
    B.5.3.3Post code10126
    B.5.3.4CountryItaly
    B.5.4Telephone number00390116335439
    B.5.5Fax number00390116963487
    B.5.6E-mailadriano.chio@unito.it
    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 TEVAGRASTIM
    D.2.1.1.2Name of the Marketing Authorisation holderTeva Italia S.r.l.
    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 namefilgrastim
    D.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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) Yes
    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 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 Yes
    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.IMP: 2
    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 MANNITOLO 18% LDB
    D.2.1.1.2Name of the Marketing Authorisation holderLaboratori Diaco Biomedicali S.p.A.
    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 nameMANNITOLO 18%
    D.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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 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.IMP: 3
    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 PERFALGAN
    D.2.1.1.2Name of the Marketing Authorisation holderBristol-Myers Squibb S.r.l.
    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 namePARACETAMOLO
    D.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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 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 placeboInjection
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Amyotrophic lateral sclerosis (ALS) is a severe progressive neurological disorder characterized by a selective degeneration of spinal, bulbar, and cortical motor neurons.
    La sclerosi laterale amiotrofica (SLA) è una malattia neurologica progressiva grave caratterizzata da una degenerazione selettiva dei spinale, bulbare, e motoneuroni corticali.
    E.1.1.1Medical condition in easily understood language
    Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease of adulthood characterized by progressive degeneration of spinal motor neurons, leading to cortical and bulbar muscle weakness.
    La (SLA) è una malattia neurodegenerativa dell'età adulta caratterizzata dalla degenerazione progressiva dei motoneuroni spinali, portando a corticale e bulbare debolezza muscolare.


    E.1.1.2Therapeutic area Not possible to specify
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    assessment of the safety and efficacy of granulocyte-colony stimulating factor compared with placebo in ALS patients, evaluated with a placebo-controlled, double blind, parallel groups design.
    valutare la sicurezza l’efficacia del trattamento con G-CSF (filgrastim) rispetto al placebo in pazienti affetti da SLA;
    valutare le modificazioni dei parametri infiammatori a livello sierico e liquorale (chemochine e citochine) nei pazienti trattati con filgrastim rispetto a quelli trattati con placebo.

    E.2.2Secondary objectives of the trial
    evaluation of cytokine and chemokine levels in the cerebrospinal fluid and serum of patients before and after the treatment with granulocyte-colony stimulating factor and placebo
    valutare le modificazioni dei parametri infiammatori a livello sierico e liquorale (chemochine e citochine) nei pazienti trattati con filgrastim rispetto a quelli trattati con placebo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    a. patients with definite, probable, probable laboratory-supported or possible ALS
    b. age 20-75 years;
    c. disease duration <=24 months;
    d. respiratory function (FVC) ≥ 65% of expected;
    e. patients must understand and accept the protocol requirements, and must give a written informed consent.
    a. SLA definita, probabile, probabile con supporto di laboratorio o possibile;
    b. età tra 20 e 75 anni;
    c. durata di malattia inferiore o uguale a 24 mesi;
    d. funzione respiratoria soddisfacente (capacità vitale forzata (FVC) ≥ 65% dell’atteso);
    e. progressione di malattia documentata negli ultimi 3 mesi;
    E.4Principal exclusion criteria
    a. previous polio infection ;
    b. motor neuron diseases other than ALS (progressive bulbar palsy , progressive muscular atrophy , primary lateral sclerosis );
    c. clinical involvement of other neurological systems (sensory, extrapyramidal , oculomotor, cerebellar);
    d. serious clinical conditions such as cardiovascular disease, uncontrolled hypertension, renal or hepatic impairment , dysthyroidism , Respiratory Distress Syndrome (ARDS) , sickle-cell anaemia;
    e. subjects who have participated in any drug trial within 12 weeks prior to recruitment;
    f . concomitant malignancy, present or past, with the exception of different melanoma skin cancers and carcinoma in situ of the cervix;
    g . patients with severe congenital neutropaenia syndrome with abnormal cytogenetics;
    h . patients with rare hereditary problems of fructose intolerance;
    i . present or past malignant myeloproliferative diseases, secondary polycythemia, splenomegaly with a diameter> 14 cm, thrombophilia was found;
    j . presence of percutaneous gastrostomy at the time of recruitment ;
    k . presence of NIV at the time of recruitment;
    l. subjects who have participated in any drug trial within 12 weeks prior to recruitment;
    n . drug addiction , alcoholism, or psychiatric disorders;
    o . women who are pregnant or breastfeeding.
    a. pregressa infezione poliomielitica;
    b. malattie del motoneurone diverse dalla SLA (paralisi bulbare progressiva, atrofia muscolare progressiva, sclerosi laterale primaria);
    c. coinvolgimento clinico di altri sistemi neurologici (sensoriale, extrapiramidale, oculomotore, cerebellare, vegetativo);
    d. gravi condizioni cliniche internistiche, come malattie cardiovascolari, ipertensione arteriosa non controllata, insufficienza renale o epatica, distiroidismo, Sindrome da Sofferenza Respiratoria dell'Adulto (ARDS), anemia falciforme
    e. soggetti che abbiano partecipato ad un qualsiasi trial farmacologico nelle 12 settimane precedenti il reclutamento;
    f. concomitante neoplasia, presente o pregressa, ad eccezione di neoplasie cutanee differenti dal melanoma e del carcinoma in situ della cervice;
    g. pazienti con neutropenia congenita grave (sindrome di Kostman) portatori di anomalie citogenetiche (vedere più avanti);
    h. pazienti affetti da rari problemi ereditari di intolleranza al fruttosio
    i. malattie mieloproliferative maligne presenti o pregresse, poliglobulia secondaria, splenomegalia con diametro > 14 cm, stato trombofilico accertato;
    j. presenza di gastrostomia percutanea o punteggio inferiore a 2 alla voce deglutizione della scala ALSFRS-R al momento del reclutamento;
    k. presenza di NIV al momento del reclutamento (punteggio pari a 4 al punto 12 della scala ALSFRS-R);
    l. soggetti che abbiano partecipato ad un qualsiasi trial farmacologico nelle 12 settimane precedenti il reclutamento
    m. scarsa compliance con pregressi trattamenti;
    n. tossicodipendenza, alcolismo o disturbi psichiatrici;
    o. donne in gravidanza o allattamento.
    E.5 End points
    E.5.1Primary end point(s)
    a. modification of progression rates of total disability score (ALS-FRS-R) during the 72 weeks of the study;
    b. modification of manual muscle testing scores (MRC scale), FVC and QoL during the 72 weeks of the study;
    c. time to death, tracheostomy or use of Non-Invasive Ventilation ≥18 h/day;
    d. evaluation of treatment safety and tolerability
    Modificazione della velocità di progressione del punteggio totale di disabilità (scala ALSFRS-R) nel corso delle 72 settimane dello studio (48 settimane di trattamento + 24 settimane di follow-up) calcolata come segue:

    ALSFRS-R slope = [punteggio ALSFRS-R (tempo 0) – punteggio ALSFRS-R (tempo 72)]/72

    Per i pazienti che usciranno dallo studio per decesso o ritiro del consenso prima del termine delle 72 settimane di trattamento lo slope sarà calcolato utilizzando l’ultima osservazione prima dell’uscita dallo studio suddividendolo per il numero di settimane di osservazione.
    E.5.1.1Timepoint(s) of evaluation of this end point
    all the end points will be assessed for the entire duration of the study at week 0, 4, 12, 24, 36, 48 and including time point of follow-up at weeks 60 and 72.
    tutti gli end-point saranno valutati per l'intera durata dello studio alla settimana 0, 4, 12, 24, 36, 48 e compreso punto di tempo di follow-up alle settimane 60 e 72.
    E.5.2Secondary end point(s)
    a. modification of progression rates of total disability score (ALS-FRS-R) during the 72 weeks of the study;
    b. modification of manual muscle testing scores (MRC scale), FVC and QoL during the 72 weeks of the study;
    c. time to death, tracheostomy or use of Non-Invasive Ventilation ≥18 h/day;
    d. evaluation of treatment safety and tolerability
    a. Modificazione delle velocità di progressione dei punteggi di forza muscolare (scala MRC), della funzionalità respiratoria (FVC) e della qualità di vita (QoL) (misurata con il McGill Quality of Life Questionnaire) nel corso dello studio (tempo 0, 24, 48 e 72 settimane); anche in questo caso si utilizzerà la formula riportata in 9.1;
    b. tempo al decesso o al posizionamento di tracheostomia (analisi di sopravvivenza) o all’utilizzo di ventilazione non invasiva (NIV) ≥18 h/die;
    c. interruzione del trattamento per progressione di malattia o per evento avverso;
    d. analisi di sicurezza e tollerabilità: confronto fra i SAE nei pazienti in farmaco e placebo.
    E.5.2.1Timepoint(s) of evaluation of this end point
    all the end points will be assessed for the entire duration of the study at week 0, 4, 12, 24, 36, 48 and including time point of follow-up at weeks 60 and 72.
    tutti gli end-point saranno valutati per l'intera durata dello studio alla settimana 0, 4, 12, 24, 36, 48 e compreso punto di tempo di follow-up alle settimane 60 e 72.
    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 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 concerned11
    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 No
    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
    ULTIMA VISITA ULTIMO SOGGETTO
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    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: 90
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 90
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state90
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 90
    F.4.2.2In the whole clinical trial 90
    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)
    Subjects are required to return to clinic for a planned follow-up of 24 weeks (2 visits every 12 weeks) after last infusion for the following assessments: ALSFRS-R, Manual Muscle Testing (MRC Scale), Physical Examination, Neurological Examination, Concomitant Medication, Vital Signs and Weight, Blood samples (haematology and clinical chemistry).
    I soggetti sono tenuti a restituire alla clinica per un follow-up previsto di 24 settimane (2 visite ogni 12 settimane) dopo l'ultima infusione per i seguenti valutazioni: ALSFRS-R, Muscolo Manuale Testing (MRC Scale), esame fisico, neurologico esame, concomitanti farmaci, Segnali e peso vitali, campioni di sangue (ematologia e chimica clinica).
    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 Decision2014-08-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-09-01
    P. End of Trial
    P.End of Trial StatusOngoing
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