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    Summary
    EudraCT Number:2014-004110-28
    Sponsor's Protocol Code Number:1/2014
    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:2014-12-22
    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 number2014-004110-28
    A.3Full title of the trial
    A monocentric, open-label pilot study to assess the safety and efficacy of minimal islet transplantation in patients with new-onset type 1 diabetes.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Islet transplantation at the onset of diabetes mellitus type 1
    A.3.2Name or abbreviated title of the trial where available
    MITO Trial
    A.4.1Sponsor's protocol code number1/2014
    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 SponsorIRCCS Ospedale San Raffaele
    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 supportMinistero
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIRCCS Ospedale San Raffaele
    B.5.2Functional name of contact pointMedicina Generale Indirizzo Diabeto
    B.5.3 Address:
    B.5.3.1Street AddressVia Olgettina 60
    B.5.3.2Town/ cityMilano
    B.5.3.3Post code20132
    B.5.3.4CountryItaly
    B.5.4Telephone number00390226432961
    B.5.5Fax number00390226433960
    B.5.6E-mailpiemonti.lorenzo@hsr.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 THYMOGLOBULIN®
    D.2.1.1.2Name of the Marketing Authorisation holderGenzyme SRL
    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 nameTHYMOGLOBULINE
    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 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 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) Yes
    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 NEULASTA®
    D.2.1.1.2Name of the Marketing Authorisation holderAmgen 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 nameNeulasta
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    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 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: 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 RAPAMUNE®
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Limited
    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 nameRapamune
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Type 1 diabetes
    E.1.1.1Medical condition in easily understood language
    Diabetes
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level PT
    E.1.2Classification code 10067584
    E.1.2Term Type 1 diabetes mellitus
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The aim of this study is to test the efficacy and safety of a marginal islet transplantation associated with immunosuppressive treatment / modulatory transient therapy for type 1 diabetes onset
    E.2.2Secondary objectives of the trial
    - Assess the impact of treatment on the immune response of memory and autoimmune in new onset type 1 diabetes
    - Identify biomarkers that reflect the sensitivity or resistance to the potential benefits of treatment on β-cell function
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Males and females aged 18-45 years
    - Ability to provide written informed consent
    - Mentally stable and able to comply with the procedures of the Protocol for the duration of the study, including follow-up visits and examinations scheduled
    - T1D new onset (diagnosis of diabetes within 180 days prior to enrollment).
    - Function beta cell residual (C-peptide fasting> 0.3 ng / mL in plasma glucose levels> 70 mg / dL and ≤200 mg / dL).
    - Positivity for at least one of the following autoantibodies typically associated with T1D: anti-GAD, anti-IA-2, anti ZnT8, IAA. A person who is positive for IAA and negative for other autoantibodies is not admissible if used insulin for more than 7 days.
    - Ongoing treatment with insulin, or has required insulin therapy (for 7 days) for diabetes at some time between the date of diagnosis and recruitment
    - Incompatibility for MHC class IA and B and compatibility for MHC class II DR with the donor of islet
    E.4Principal exclusion criteria
    - Body mass index (BMI) ≥ 32.0 kg / m2 or patient ≤ 50kg weight
    - Requirement of insulin> 1.0 IU / kg / day
    - HbA1c> 10%
    - Blood pressure: systolic> 160 mmHg or diastolic blood pressure> 100 mmHg.
    - Chronic disease than type 1 diabetes.
    - Moderate and severe renal impairment (creatinine clearance <90 mL / min)
    - Presence or history of macroalbuminuria (> 300 mg / g creatinine).
    - Hepatic dysfunction defined by an increase in ALT / AST above the limit of the normal value and increases in total bilirubin> 3 mg / dl
    - Pregnant or breast-feeding.
    - Reluctance to use effective contraception up to 4 months after the end of the administration of the study drug (females and males)
    - Active infection including hepatitis B, hepatitis C, HIV or tuberculosis (TB). Positive tests are acceptable only if associated with a history of previous vaccination in the absence of any sign of active infection.
    - Negative for Epstein-Barr Virus (EBV) to the determination of IgG
    - Invasive aspergillosis, histoplasmosis, coccidioidomycosis year prior to study enrollment
    - Any history of malignancy except basal or squamous cell carcinoma of the skin eradicated
    - Alcohol or substance abuse known active
    - Haemoglobin base below the lower limit of normal; lymphopenia (<1,000 / microL), neutropenia (<1,500 / microL), or thrombocytopenia (platelets <100,000 / microL).
    - A history of lack of factor V
    - Any coagulopathy or diseases requiring long-term anticoagulant therapy (eg, warfarin) after transplantation (treatment with low-dose aspirin is permitted) or patients with an international normalized ratio (INR)> 1.5
    - Heart Disease serious coexisting:
    recent myocardial infarction (within the last 6 months)
    left ventricular ejection fraction <30%
    - Calculus symptomatic gallbladder
    - Acute or chronic pancreatitis
    - Peptic ulcer symptomatic
    - Severe diarrhea incessant vomiting or other gastrointestinal disorders potentially interfering with the ability to absorb oral medications
    - Hyperlipidemia despite medical therapy (fasting low-density lipoprotein [LDL] cholesterol> 130 mg / dL, treated or untreated, and or fasting triglycerides> 200 mg / dL)
    - Treatment for a medical condition requiring chronic use of systemic steroids, except for the use of 5 mg of prednisone per day, or an equivalent dose of hydrocortisone, for the replacement saline.
    - Treatment with any anti-diabetic drug other than insulin within 4 weeks of enrollment
    - Use of investigational drugs within 4 weeks of enrollment.
    - The administration of live attenuated vaccine within 2 months thereafter
    - Any medical condition that, in the opinion of the investigator, would interfere with the safe participation in the study.
    - Treatment with any immunosuppressive regimen at enrollment
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in the insulin secretory response evaluated as Area Under the Curve (AUC) to two hours of C-peptide plasma in response to mixed meal (MMTT)
    The incidence and severity of adverse events (AE) on the procedure for islet transplantation or treatment-related immunosuppressive
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 52 ± 2
    Weeks 4 ± 1, 12± 2 and week 52 ± 2
    E.5.2Secondary end point(s)
    Change from baseline in the insulin secretory response evaluated as Area Under the Curve (AUC) to two hours of C-peptide plasma in response to mixed meal (MMTT) one year after transplant
    Change from baseline C-peptide stimulated (the highest value at any point in time during the MMTT after injection mixed meal)
    Change from baseline in the secretory response gucagone evaluated as Area Under the Curve (AUC) to two hours of C-peptide plasma in response to mixed meal (MMTT)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Weeks: 4 ± 1, 12± 2, 26±2
    Weeks: 4 ± 1, 12± 2, 26±2, 52 ± 2
    Weeks: 4±1, 12±2, 26±2, 52±2
    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 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 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 Yes
    E.8.1.7.1Other trial design description
    pilot
    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.8.5The 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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned 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: 6
    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 state6
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 6
    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 end of the follow-up of one year, the subjects will be invited to join a separate protocol for monitoring the safety / efficacy. Those who agree to participate will be monitored for another five years.
    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 Decision2015-03-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-04-01
    P. End of Trial
    P.End of Trial StatusOngoing
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