E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
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 |
|
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 study is intended to evaluate the effect of 20 µg Diamyd on the preservation of pancreatic beta cell function as defined by an increase in fasting C-peptide. In addition, the proposed study will provide further data to evaluate the safety of Diamyd treatment.
The primary objective is to evaluate the efficacy of 20 µg Diamyd, versus placebo.
|
|
E.2.2 | Secondary objectives of the trial |
The secondary objective is to evaluate the safety of 20 µg Diamyd treatment.
|
|
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1. Male and female patients of any race between 10 and 18 years of age. 2. Type 1 diabetes mellitus diagnosed within the previous 18 months and the onset at least 14 days prior to Primary injection 3. C-peptide level above 0.1 nmol/l. 4. Presence of GAD65 antibodies 5. Written informed consent both from the patient and the guardian where applicable.
|
|
E.4 | Principal exclusion criteria |
1. Secondary diabetes mellitus. 2.Treatment with immunosuppressant(s). 3. A history of anaemia or significantly abnormal haematology results at screening. 4. A history of epilepsy, head trauma or cerebrovascular accident or clinical features of continuous motor unit activity in proximal muscles. 5. Clinically significant history of acute reaction to drugs in the past. 6. Treatment with any vaccine within one month prior to planned first Diamyd dose or planned treatment with vaccine up to 2 months after the last injection with Diamyd. 7. Participation in other clinical trials with a new chemical entity within the previous 3 months. 8. Inability or unwillingness to comply with the provisions of this protocol. 9. A history of alcohol or drug abuse. 10. A significant illness other than diabetes within 2 weeks prior to first dosing. 11. HIV or hepatitis. 12. Females pregnant at the time of injection or plans for pregnancy during the next 13 months ( one year after 2 nd administration). 13. MODY- Maturity Onset Diabetes in the young, known or likely 14. Presence of associated serious disease or condition which in the opinion of the investigator makes the patient non-eligible for the study.
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy endpoint is the change in fasting concentration of C-peptide from baseline to 15 months after the first injection. The secondary efficacy endpoints are changes from baseline to various time-points for the following variables: The concentration of HbA1c Fasting concentration of C-peptide Concentration of C-peptide after challenge with Sustacal
|
|
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 | Yes |
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) | 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 | 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.8.5 | 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 | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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.8.9.1 | In the Member State concerned years | 7 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |