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    Summary
    EudraCT Number:2019-004426-24
    Sponsor's Protocol Code Number:TREAT-LMNA
    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:2021-06-15
    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 number2019-004426-24
    A.3Full title of the trial
    Deflazacort TREATment in LMNA related congenital muscular dystrophy: study of clinical effectiveness and search for reliable biomarkers.
    Deflazacort TRATTAMENTO nella distrofia muscolare congenita correlata a LMNA: studio dell'efficacia clinica e ricerca di biomarcatori affidabili
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Deflazacort TREATment in LMNA related congenital muscular dystrophy: study of clinical effectiveness and search for reliable biomarkers.
    Deflazacort TRATTAMENTO nella distrofia muscolare congenita correlata a LMNA: studio dell'efficacia clinica e ricerca di biomarcatori affidabili
    A.3.2Name or abbreviated title of the trial where available
    TREAT-LMNA
    TREAT-LMNA
    A.4.1Sponsor's protocol code numberTREAT-LMNA
    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 SponsorAZIENDA OSPEDALIERO-UNIVERSITARIA PISANA
    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 - Italian Medicines Agency
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAzienda Ospedaliero - Universitaria Pisana
    B.5.2Functional name of contact pointU.O. Farmacologia Clinica e Farmaco
    B.5.3 Address:
    B.5.3.1Street AddressVia Roma 67
    B.5.3.2Town/ cityPisa
    B.5.3.3Post code56126
    B.5.3.4CountryItaly
    B.5.4Telephone number050996215
    B.5.5Fax number050993570
    B.5.6E-mailv.gori@ao-pisa.toscana.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.2.1.2Country 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 nameDeflazacort
    D.3.2Product code [Deflazacort]
    D.3.4Pharmaceutical form Oral drops, solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPBuccal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNdeflazacort
    D.3.9.2Current sponsor codedeflazacort
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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.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 Lucen
    D.2.1.1.2Name of the Marketing Authorisation holderMalesci
    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 nameLucen
    D.3.2Product code [-]
    D.3.4Pharmaceutical form Gastro-resistant granules
    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 INNesomeprazolo
    D.3.9.2Current sponsor code-
    D.3.9.3Other descriptive nameesomeprazole
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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
    Congenital Muscolar distrophy LMNA related
    Distrofia muscolare congenita correlata a LMNA ( L-CMD)
    E.1.1.1Medical condition in easily understood language
    Congenital muscolar distrophy coused by lamina gene mutation
    distrofia muscolare congenita causata da mutazione del gene lamina
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10003718
    E.1.2Term Atrophy skeletal muscle
    E.1.2System Organ Class 100000004859
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objectives of the study will be:
    1. Evaluation of the effect of therapy with Deflazacort drops 0.9 mg / kg / day on clinical outcome and secretoma in adult and pediatric patients with L-CMD.
    2. Evaluation of the safety and tolerability of the treatment with Deflazacort drops 0.9 mg / kg / day
    Gli obiettivi primari dello studio saranno:
    1. Valutazione dell'effetto della terapia con Deflazacort gocce 0,9 mg / kg / die sull'esito clinico e sul secretoma in pazienti adulti e pediatrici affetti da L-CMD.
    2. Valutazione della sicurezza e della tollerabilità del trattamento con Deflazacort gocce 0,9 mg / kg / die
    E.2.2Secondary objectives of the trial
    The secondary objective will be to study in vitro whether LCMD specific secreted molecules influence myogenic differentiation and fibrosis in normal human myoblasts in culture, thus validating these molecules as biomarkers in L-CMD.
    L'obiettivo secondario sarà quello di studiare in vitro se le molecole secrete specifiche della LCMD influenzano la differenziazione miogenica e la fibrosi nei mioblasti normali umani in coltura, convalidando così queste molecole come biomarcatori nell'L-CMD.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Corticosteroid-naive patients, aged between 3 and 40 years of both sexes and of Caucasian origin, with clinical and genetic diagnosis of L-CMD or EDMD2 with an onset age before 5 years.
    • Patients will be eligible if at the time of recruitment they are able to provide reproducible measurements of forced vital capacity (FVC) (variability <15% between two repeated measures of FVC).
    • Signing of informed consent by the patient, in the case of an adult individual, or by the parents / legal guardian, in the case of an individual under the age of 18.

    Then, patients will be divided into two subgroups based on clinical characteristics:
    i) subgroup 1: patients who maintain the following abilities:
    -to walk for at least 50 meters at 6 minutes of walking test (6MWT)
    - get up from the floor, with or without support.
    ii) subgroup 2: patients with loss of gait or more severe motor impairment who do not maintain the abilities described above.
    • Pazienti naïve ai corticosteroidi, di età compresa tra 3 e 40 anni di entrambi i sessi e di origini caucasiche, con diagnosi clinica e genetica di L-CMD o EDMD2 con età insorgente prima dei 5 anni.
    • I pazienti saranno ammissibili se al momento del reclutamento sono in grado di fornire misurazioni riproducibili della capacità vitale forzata (FVC) (variabilità <15% tra due misure ripetute di FVC).
    • Sottoscrizione del consenso informato da parte del paziente, in caso di individuo adulto, o dei genitori/tutore legale, in caso di individuo minorenne.

    Quindi, i pazienti saranno suddivisi in due sottogruppi in base alle caratteristiche cliniche:
    i) sottogruppo 1: pazienti che mantengono le seguenti capacità:
    -per camminare per almeno 50 metri a 6 minuti di test di camminata (6MWT)
    -alzarsi dal pavimento, con o senza supporto.
    ii) sottogruppo 2: pazienti con perdita di deambulazione o compromissione motoria più grave che non mantengono le capacità sopra descritte.
    E.4Principal exclusion criteria
    Exclusion criteria
    • Hypersensitivity to the active substance or to any of the excipients
    • Chronic therapy with corticosteroids or immunosuppressive drugs
    • Diabetes mellitus or other comorbidities which strongly contraindicate the use of corticosteroids
    • Pregnancy and breastfeeding.
    Criteri di esclusione
    • Ipersensibilità al principio attivo o ad uno qualsiasi degli eccipienti
    • Terapia cronica con corticosteroidi o farmaci immunosoppressori
    • Diabete mellito o altre comorbidità che controindicano fortemente l'uso di corticosteroidi
    • Gravidanza e allattamento.
    E.5 End points
    E.5.1Primary end point(s)
    1. Safety evaluation of the administration of Deflazacort drops (0.9mg / kg / day)

    2. Evaluation of the effects of treatment with Deflazacort drops (0.9mg / kg / day in relation to the change of clinical parameters at each treatment time and follow up (T0-T6-T12-T15-T18).
    For subgroup 1, the primary clinical outcome variable will be chosen as a three-dimensional (multivariate) outcome consisting of the following three components:
    • relaxation time
    • FVC
    • motor measurement (MFM)

    For subgroup 2, the primary clinical outcome variable will be:
    • FVC
    • motor measurement (MFM)
    1. Valutazione di sicurezza della somministrazione di Deflazacort gocce (0.9mg/kg/die)

    2. Valutazione degli effetti del trattamento con Deflazacort gocce (0.9mg/kg/die in relazione al cambiamento di parametri clinici ad ogni tempo di trattamento e follow up (T0-T6-T12-T15-T18).
    Per il sottogruppo 1, la variabile di esito clinico primario sarà scelta come esito tridimensionale (multivariato) costituito dai seguenti tre componenti:
    • tempo di distensione
    • FVC
    • misurazione motoria ( MFM)

    Per il sottogruppo 2, la variabile di esito clinico primario sarà:
    • FVC
    • misurazione motoria (MFM)
    E.5.1.1Timepoint(s) of evaluation of this end point
    18 mesi
    18 mesi
    E.5.2Secondary end point(s)
    Secondary outcome variables will include the following continuous variables, calculated on average for all post-baseline follow-up visits:
    Time to run / walk 10 m (subgroup 1);
    • distance traveled in 6 minutes (subgroup 1);
    • range of motion in the ankle, elbow and knee joints (subgroup 1 and 2);
    • cardiac function (measured by transthoracic echocardiography and 12-lead ECG) (subgroup 1 and 2);
    Le variabili di risultato secondarie includeranno le seguenti variabili continue, calcolate in media per tutte le visite di follow-up post-basale:
    • tempo di correre / camminare 10 m (sottogruppo 1);
    • distanza percorsa in 6 minuti (sottogruppo 1);
    • gamma di movimento nelle articolazioni della caviglia, del gomito e del ginocchio (sottogruppo 1 e 2);
    • funzione cardiaca (misurata mediante ecocardiografia transtoracica ed ECG a 12 derivazioni) (sottogruppo 1 e 2);
    E.5.2.1Timepoint(s) of evaluation of this end point
    18 mesi
    18 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 No
    E.6.4Safety No
    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 Yes
    E.6.13.1Other scope of the trial description
    specific biomarkers
    ricerca biomarcatori
    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 No
    E.8.1.2Open Yes
    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) Yes
    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 concerned6
    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 months25
    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 months25
    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: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    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 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 20
    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)
    Fr each patient, the study will be completed at the end of treatmentand of follow up
    Per ogni paziente lo studio sarà completato alla fine del trattamento e del follow up
    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 Decision2020-07-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-06-11
    P. End of Trial
    P.End of Trial StatusOngoing
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