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    Summary
    EudraCT Number:2018-000368-27
    Sponsor's Protocol Code Number:MAN-ALG-18-001
    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:2020-11-04
    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 number2018-000368-27
    A.3Full title of the trial
    ND
    CHANGE IN PLASMA LYSO GL3 AND RELIEF OF GASTRO INTESTINAL SYMPTOMS IN PATIENTS SWITCHED FROM AGALSIDASE ALFA TO AGALSIDASE BETA
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    ND
    ND
    A.3.2Name or abbreviated title of the trial where available
    ND
    ND
    A.4.1Sponsor's protocol code numberMAN-ALG-18-001
    A.5.4Other Identifiers
    Name:NDNumber:ND
    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 SponsorFONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
    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 supportGenzyme-Sanofi
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationClinical Trial Center
    B.5.2Functional name of contact pointUnità CRO
    B.5.3 Address:
    B.5.3.1Street Addresslargo Agostino Gemelli 8
    B.5.3.2Town/ cityRoma
    B.5.3.3Post code00168
    B.5.3.4CountryItaly
    B.5.4Telephone number0688805565
    B.5.5Fax number0688808867
    B.5.6E-mailbetty.polikar@clinicaltrialcenter.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 FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO
    D.2.1.1.2Name of the Marketing Authorisation holderGENZYME EUROPE B.V.
    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 namefabrazyme
    D.3.2Product code [A16AB04]
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAGALSIDASI BETA
    D.3.9.2Current sponsor codeNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number35
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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 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 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.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
    Fabry disease
    Malattia di Fabry
    E.1.1.1Medical condition in easily understood language
    ND
    ND
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level SOC
    E.1.2Classification code 10010331
    E.1.2Term Congenital, familial and genetic disorders
    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 any difference in plasma Lyso gl3 measure and clinical outcomes in gastrointestinal involvement in patients with GI involvement switched from agalsidase alfa to beta.
    valutare eventuali differenze nella misurazione del Lyso GL3 plasmatico e gli esiti clinici nel coinvolgimento gastrointestinale in pazienti con coinvolgimento GI passati da agalsidasi alfa a beta
    E.2.2Secondary objectives of the trial
    to identify differences in intestinal microbiota composition in a cohort of patients switched from a standard dose of agalsidase alfa to agalsidase beta.
    identificare differenze nella composizione del microbiota intestinale in una coorte di pazienti passati da una posologia standard di agalsidasi alfa ad agalsidasi beta.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Informed Consent signed, before any trial procedures are performed
    • Male or female patients
    • Age =18 years
    • Confirmed diagnosis of Fabry disease and concomitant treatment with standard dose of agalsidase alfa for at least 1 years, without any dose modification
    • Presence of continuous gastrointestinal involvement for the previous 4 months before the screening, with at least 1 symptom reported in the GRSG questionnaire
    • Elevated plasma lyso-GL3 (plasma lyso-Gl3 > 7 ng/ml)
    • Firma del consenso informato, prima di qualsiasi procedura prevista dallo studio
    • Pazienti di sesso maschile o femminile
    • Età =18 anni
    • Diagnosi di malattia di Fabry confermata, e trattamento concomitante con dose standard di agalsidasi alfa da almeno un anno, senza alcuna modifica della posologia
    • Presenza di manifestazioni gastrointestinali in maniera continua nei 4 mesi precedenti la visita di screening, con almeno uno dei sintomi riportati nel questionario GSRS
    • valori elevati di lyso-GL3 plasmatico (plasma lyso-Gl3 > 7 ng/ml)
    E.4Principal exclusion criteria
    • plasma lyso-Gl3= 7 ng/ml at baseline and/or no evidences of GI symptoms.
    • Patients with a diagnosis of chronic inflammatory diseases defined according to the criteria of the International Classification: gastroenterological, rheumatologic, immunologic autoimmune diseases
    • Patients diagnosed with any type of cancer in evolution
    • Patients diagnosed with infectious disease certified through microbiological tests performed in the last 12 months (included in the medical history of the patient)
    • Patients with IBD, Celiac disease or other inflammatory chronic disease such as Crohn disease or rectal ulcerative colitis (RUC )
    • Pregnancy or lactation
    • Concomitant medication with chloroquine, amiodarone, benoquin or gentamycin, due to a theoretical risk of inhibition of intra-cellular a- Gal A activity.
    • Known allergy to one or more of the components of agalsidase beta
    • Livelli plasmatici di lyso-Gl3= 7 ng/ml al basale e/o nessuna evidenza di manifestazioni gastrointestinali .
    • Pazienti con diagnosi di malattie infiammatorie croniche, definite secondo I criteri di classificazione internazionale: gastrointestinali, reumatologiche, immunologiche ed autoimmunitarie
    • Pazienti con diagnosi di neoplasie
    • Pazienti con diagnosi di malattie infettive certificate da test microbiologici eseguiti negli ultimi 12 mesi (incluso nella storia clinica del paziente)
    • Pazienti con sindrome dell’intestino irritabile, con malattia celiaca o altre malattie infiammatorie croniche intestinali come malattia di Crohn o rettocolute ulcerosa
    • stato di gravidanza o allattamento
    • concomitante terapia con clorochina, amiodarone, benoquina o gentamicina, in relazione al rischio teorico di inibilre l’attività intracellultare dell’enzima a- Gal A.
    • Allergie note ad uno o più componenti dell’agalsidasi beta
    E.5 End points
    E.5.1Primary end point(s)
    1. The primary endpoint will be focused on the evaluation of plasma level of Lyso GL3 and gastrointestinal symptoms evaluated through the validated scale “Gastrointestinal Symptom Rating Scale” (GSRS) at baseline and after the switch from agalsidase alfa to beta.
    L'endpoint primario sarà focalizzato sulla valutazione del livello plasmatico di Lyso GL3 e dei sintomi gastrointestinali valutati attraverso la scala validata "Gastrointestinal Symptom Rating Scale" (GSRS) al basale e dopo il passaggio da agalsidasi alfa a beta.
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 months
    12 mesi
    E.5.2Secondary end point(s)
    Intestinal microbiota: three samples intestinal fecal will be recollected at baseline and at the time of standard follow up to evidence any differences in the colonization of intestinal bacteria.
    Analisi del microbiota intestinale: tre campioni di feci intestinali saranno raccolte al basale e al momento del follow up standard per evidenziare eventuali differenze nella colonizzazione dei batteri intestinali.
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 months
    12 mesi
    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 Yes
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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
    Clinical Study Report availability
    Disponibilità del rapporto clinico finale
    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 months27
    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 months27
    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: 7
    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 state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 14
    F.4.2.2In the whole clinical trial 14
    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)
    Normal clinical practice
    Normale pratica 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 Decision2018-07-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-10-22
    P. End of Trial
    P.End of Trial StatusCompleted
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