E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
patients with long-standing type 1 diabetes |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Investigative Techniques [E05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | HLGT |
E.1.2 | Classification code | 10018424 |
E.1.2 | Term | Glucose metabolism disorders (incl diabetes mellitus) |
E.1.2 | System Organ Class | 10014698 - Endocrine disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10012602 |
E.1.2 | Term | Diabetes mellitus (incl subtypes) |
E.1.2 | System Organ Class | 10014698 - Endocrine disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10012601 |
E.1.2 | Term | Diabetes mellitus |
E.1.2 | System Organ Class | 10027433 - Metabolism and nutrition disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10067584 |
E.1.2 | Term | Type 1 diabetes mellitus |
E.1.2 | System Organ Class | 10027433 - Metabolism and nutrition disorders |
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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 |
The primary objective is to proof that the pancreatic uptake of EX in patients with long-standing DM1 and non-measurable production of C-peptide, i.e. patients with no relevant beta-cell mass left, is clearly lower than in healthy individuals with intact glucose homeostasis and insulin production. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives are:
• To determine the difference in uptake between the two groups in order to estimate the minimum difference in beta-cell mass (>15%) that would be measurable by GRS. (The beta cell mass in healthy subjects may vary considerably (circa 1-8% of the total pancreatic mass (Ritzel et al 2006)), so that it cannot be predicted what the difference in pancreatic uptake of 111In-DTPA-Exendin between healthy volunteers and patients with long standing T1D will be.)
• To perform dosimetric calculations in order determine the effective dose to the patient for EX-scanning and to determine the dose to the individual islets.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• 21-60 years of age
• Long-standing T1D (>5 years) or healthy volunteer with normal glucose tolerance
• Body mass index (BMI) of 21-27
• No previous treatment with synthetic Exendin (Exenatide, Byetta®) or Dipeptidyl-Peptidase IV inhibitors
• No measurable C-peptide in T1D patients (stimulated)
• Normal HbA1c (<7%) in healthy volunteers
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E.4 | Principal exclusion criteria |
• Age <21 or >60 years
• T1D duration <5 years or pathologic glucose tolerance in healthy volunteers
• BMI <21 or >27
• Previous treatment with Exenatide or Dipeptidyl-Peptidase IV inhibitors
• Measurable C-peptide in T1D patients
• Elevated HbA1c values (>7%) in healthy volunteers
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E.5 End points |
E.5.1 | Primary end point(s) |
Determination of the difference in uptake of the radiotracer into the pancreas between healthy volunteers and patinets with type 1 diabetes. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Determination of the minimal difference in beta cell mass that can be measured via the uptake of the radiotracer into the pancreas |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Proof of principle that scintigraphic measurement of the utpake of 111In-DTPA-[K40]-Exendin 4 into the pancreas allows to determine differences in beta cell mass. |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | Yes |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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E.8.2.4 | Number of treatment arms in the trial | 2 |
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.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial 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
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |