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    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7272   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    EudraCT Number:2018-000692-32
    Sponsor's Protocol Code Number:FARMPre-Proposalcode:TRS-2016-00
    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:2020-01-14
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2018-000692-32
    A.3Full title of the trial
    A randomized, double blind, placebo-controlled, multicenter clinical trial.
    Studio multicentrico, doppio cieco, randomizzato con placebo.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Trial to evaluate the efficacy of the drug metformin on the muscles in patients with myotonic distrophy type 1
    Studio per valutare l'efficacia del farmaco metformina sui muscoli in pazienti con distrofia miotonica di tipo I
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberFARMPre-Proposalcode:TRS-2016-00
    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.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 support
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFondazione Policlinico Tor Vergata
    B.5.2Functional name of contact pointDipartimento di Neuroscienze
    B.5.3 Address:
    B.5.3.1Street AddressViale Oxford 81
    B.5.3.2Town/ cityRoma
    B.5.3.3Post code00133
    B.5.4Telephone number0620903014
    B.5.5Fax number0620903014
    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. name METFORMINA - 500 30 COMPRESSE 500 MG
    D. of the Marketing Authorisation holderWYETH LEDERLE 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 nameMetformina
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMetformina
    D.3.9.2Current sponsor codeN/A
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number500 to 1500
    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. cell therapy medicinal product Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. 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 placeboFilm-coated tablet
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Myotonic Distrophy type 1, confirmed by genetic testing, with a CTG expansion size >100
    Distrofia Miotonica di tipo 1, confermata da test genetici, con una dimensione di espansione CTG> 100.
    E.1.1.1Medical condition in easily understood language
    Myotonic Distrophy type 1
    Distrofia Miotonica di tipo 1
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10068871
    E.1.2Term Myotonic dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10013987
    E.1.2Term Dystrophia myotonica
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To compare metformin hydrochloride against a placebo in the treatment of DM1. The intervention is focused on the effect of the experimental intervention on motility performances. This parameter is marker of the most involved motor capabilities in DM1 patients and will be measured with sensitive and validated scales. It is expected that the trial and outcome work will lead to new clinical guidelines for DM1 treatment.
    Confrontare la metformina cloridrato con un placebo nel trattamento di DM1. L'intervento è focalizzato sull'effetto del trattamento sperimentale sulle prestazioni della motilità.
    Questo parametro è marcatore delle capacità motorie più coinvolte nei pazienti affetti da DM1 e sarà misurato con scale sensibili e validate. Si prevede che il lavoro porterà a nuove linee guida cliniche per il trattamento di DM1.
    E.2.2Secondary objectives of the trial
    Secondary objectives of the study are categorized as: 1) Dexterity and motility; 2) Muscle quantitative testing of upper and lower limbs;
    3) Fatigue; 4) Quality of life.
    Gli obiettivi secondari dello studio sono classificati come: 1) Destrezza e motilità;
    2) Test quantitativo della forza degli arti superiori e inferiori; 3) Affaticamento;
    4) Qualità della vita.

    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Inclusion criteria are as stated below:
    Principal inclusion criteria
    - Male or female patients, aged between 18 and 64 years
    - A diagnosis of DM1, confirmed by genetic testing, with a CTG expansion size >100
    - MIRS score 3 or 4
    - Ambulatory, able to perform the 6 Minute Walk Test (6MWT)
    - Able to provide written informed consent
    - For women of child-bearing potential, blood screening for beta-HCG before randomization and use of one effective method of birth control during the conduct of the study

    I criteri di inclusione sono i seguenti:
    - Pazienti di sesso maschile o femminile, di età compresa tra 18 e 64 anni.
    - Una diagnosi di DM1, confermata da test genetici, con una dimensione di espansione CTG> 100.
    - Punteggio MIRS 3 o 4.
    - Deambulante, in grado di eseguire il test del cammino di 6 minuti (6MWT).
    - In grado di fornire il consenso informato scritto.
    - Per le donne in età fertile, screening del sangue per beta-HCG prima della randomizzazione e uso di un metodo contraccettivo efficace durante lo svolgimento dello studio
    E.4Principal exclusion criteria
    Exclusion criteria are as stated below:
    Principal exclusion criteria
    - Any medical contraindications to metformin including: known hypersensitivity to Metformin Hydrochloride or any of the other
    ingredients; Renal disease or renal dysfunction (serum creatinine levels ≥1.5 mg/dL [males], ≥1.4 mg/dL [females] or abnormal creatinine
    clearance, < 60 ml/min); Any acute condition which can lead to renal dysfunction (severe infection or injury, dehydration, shock); Heart
    complications such as heart failure (even if the condition is under control) or recent heart attack; Respiratory failure (patients with nocturnal non-invasive ventilation CPAP or BiPAP could be included); Liver disease; Excessive alcohol intake within 12 months from enrollment; Acute or chronic metabolic acidosis; Diabetic complications such as diabetic coma and ketoacidosis
    - Symptomatic insulin-requiring diabetes or type 2 diabetes requiring oral anti-diabetic agents
    - Any history of malignancy except for cases of remission > 12 months
    - Women who are pregnant or breast-feeding
    - Chronic administration of any drugs that may interfere with the actions of Metformin.
    - Participation in another experimental therapeutic protocol within 6 months prior to baseline and during the study period (participation in natural history study is allowed)
    - Any other condition that, in the opinion of the investigator, may compromise the safety or compliance of the patient or would preclude
    the patient from successful completion of the study or would impair interpretation of results
    - Any neurological disease with motor impairment or other neuromuscular disease than DM1
    I criteri di esclusione sono i seguenti:
    Qualsiasi controindicazione medica alla metformina, inclusa: nota ipersensibilità a metformina cloridrato o a uno qualsiasi degli altri ingredienti; malattia renale o disfunzione renale (livelli di creatinina sierica ≥ 1,5 mg / dL [maschi], ≥ 1,4 mg / dL [femmine] o creatinina anormale clearance, <60 ml / min); Qualsiasi condizione acuta che può portare a disfunzione renale (grave infezione o lesione, disidratazione, shock); Complicanze cardiache come insufficienza cardiaca (anche se la condizione è sotto controllo) o recente infarto; Insufficienza respiratoria (pazienti in ventilazione notturna non invasiva CPAP o BiPAP potranno essere inclusi);
    Malattia del fegato;
    Eccessiva assunzione di alcol entro 12 mesi da arruolamento;
    Acidosi metabolica acuta o cronica;
    Complicanze diabetiche come coma diabetico e chetoacidosi . Diabete sintomatico che richiede insulina o diabete di tipo 2 che richiede agenti antidiabetici orali. Qualsiasi storia di neoplasia tranne che per casi di remissione da > 12 mesi.
    Donne in gravidanza o in allattamento.
    Somministrazione cronica di qualsiasi farmaco che possa interferire con le azioni di metformina.
    Partecipazione a un altro protocollo terapeutico sperimentale entro 6 mesi prima del basale e durante il periodo di studio (la partecipazione nella storia del paziente è permessa).
    Qualsiasi altra condizione che, secondo il parere dello sperimentatore, possa compromettere la sicurezza o la conformità del paziente o precluderlo dal corretto completamento dello studio o compromettere l'interpretazione dei risultati.
    Qualsiasi malattia neurologica con compromissione motoria o altra malattia neuromuscolare rispetto a DM1.
    E.5 End points
    E.5.1Primary end point(s)
    The primary outcome measure will be the 6 minute walk test (6MWT) with BORG Scale assessment (0 to 10 rating of perceived exertion score) measured at the end of the 24 months intervention period. The 6MWT is a widely used walking test: the distance walked in 6 minutes is measured and a greater distance indicates a better performance. The 6MWT has been validated for DM1 patients and is a sensitive measure that shows deterioration of motility in a relative short term within the natural history of the disease
    La misura dell’obiettivo primario sarà valutata con il test del cammino di 6 minuti (6MWT) con valutazione BORG Scale (valutazione dello sforzo percepito con punteggio da 0 a 10) misurato alla fine del periodo di intervento di 24 mesi. Il 6MWT è un test ambulatorio molto utilizzato: viene valutata la distanza percorsa in 6 minuti a piedi, dove una maggiore distanza percorsa indica migliore condizione. Il 6MWT è stato validato per pazienti con DM1 ed è un misura sensibile che mostra il deterioramento della motilità in un determinato breve periodo all'interno della storia naturale della malattia.
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 24 months of treatment
    Dopo 24 mesi di trattamento
    E.5.2Secondary end point(s)
    1. Dexterity and motility (measurement time 20 min); 2. Muscle strength (20 min); 3. Fatigue (10 min); 4. Quality of life (10 min); 4. Analysis of alternative splicing regulated by metformin; 5. Biochemical analysis to quantify markers of oxidative stress in order to assess their sensitivity to metformin treatment.
    1. Destrezza e motilità (tempo di misurazione 20 min);
    2. Forza muscolare (20 min);
    3. Affaticamento (10 minuti);
    4. Qualità della vita (10 min);
    5. Analisi dello splicing alternativo regolato da metformina;
    6. Analisi biochimiche per quantificare i marcatori dello stress ossidativo al fine di valutare la loro sensibilità al trattamento con metformina.
    E.5.2.1Timepoint(s) of evaluation of this end point
    After 24 months of treatment
    Dopo 24 mesi di trattamento
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 concerned4
    E.8.5The trial involves multiple Member States No
    E.8.5.1Number of sites anticipated in the EEA4
    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
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    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 No
    F.1.1Number of subjects for this age range: 1
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 194
    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 No
    F.3.3.1Women of childbearing potential not using contraception Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally Information not present in EudraCT
    F.3.3.7Others Information not present in EudraCT
    F.4 Planned number of subjects to be included
    F.4.1In the member state194
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 194
    F.4.2.2In the whole clinical trial 194
    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)
    At the discretion of the investigator the patient will be treated in the manner appropriate to his pathology
    A discrezione dello Sperimentatore il paziente sarà trattato nel modo adeguato alla sua patologia
    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 Decision2019-05-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-07-17
    P. End of Trial
    P.End of Trial StatusOngoing
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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