E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
prevention of type 1 diabetes mellitus in subjects at risk for T1DM |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10045228 |
E.1.2 | Term | Type I diabetes mellitus |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
determine whether intervention with repeated oral administration of recombinant human insulin will prevent or delay the development of clinical type 1 Diabetes Mellitus (T1DM) in non diabetic relatives of patients with T1DM who are positive for insulin autoantibodies but who do not have a metabolic defect. This intervention wil be compared with placebo given in a double-masked fashion. |
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E.2.2 | Secondary objectives of the trial |
Description of the effects of treatment with oral insulin versus placebo in other catgories of subjects defined using different combinations of autoantibodies and metabolic status (The Secondary Analysis Strata)and an assesment of the consistency of treatment effect among strata. Assesment of the effects of treatment on immunologic and metabolic markers. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. have a proband with T1Dm 2. if the proband is a first degree relative the partecipant must be 3-45 years of age. If the proband is a second or third degree relative the partecipant must be 3-20 years of age. 3. OGTT performed within 7 weeks prior to randomization with: fasting plasma glucose <110 mg/dl 2 hour plasma glucose <140 mg/dl 4. mIAA confirmed positive within the previous six months. 5. two samples with at least one autoantibody other than mIAA positive within the previous six months. 6. willing to sign Informed Consent Form. |
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E.4 | Principal exclusion criteria |
1. does not satisfy the above inclusion criteria. 2. has severe active disease. 3. prior partecipation in a trial for prevention of T1DM. 4. ongoing use of medications known to influence glucose. 5. Pregnant women or lactating. 6. OGTT that reveals diabetes, Impaired glucose tolerance (IGT) or impaired fasting glucose (IFG) 7. sunject has HLADQA1*0102,HLADQB1*0602. |
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E.5 End points |
E.5.1 | Primary end point(s) |
elapsed time from random treatment assignment to the development of diabetes among those enrolled in the primary analysis cohort consisting of subjects with insulin autoimmunity and absence of metabolic abnormality. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | Yes |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 3 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Yes |
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
|
la durata totale dello studio sara' quella necessaria per fornire una potenza dell'85% nel rilevare una riduzione del rischio del 40% di sviluppare il DMT1. Si potrebbe inoltre prevedere un'interruzione anticipata per assenza di effetto del farmaco |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 8 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 8 |
E.8.9.2 | In all countries concerned by the trial months | 0 |