E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients for the proposed trial are those with recent onset type 1 diabetes (diagnosed within 3 months at screening). |
|
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 primary objective is to evaluate the efficacy of i) a prime-and-boost regimen with 20 µg Diamyd and ii) a prime-and-boost regimen with 20 µg Diamyd, followed by 2 additional single doses with 20 µg Diamyd, compared to placebo with respect to preserving insulin secretion. This will be evaluated by the change from baseline to Month 15 in C peptide area under the curve mean 0-120 minutes (AUCmean 0-120 min) during an MMTT (Mixed Meal Tolerance Test). In addition, the proposed study will provide further data to evaluate the safety of Diamyd® treatment.
|
|
E.2.2 | Secondary objectives of the trial |
The secondary objectives are to compare diabetes status variables between Diamyd and placebo before and after treatment, and to further confirm the safety of Diamyd. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male and female patients between 10 and 20 years of age 2. Insulin dependent type 1 diabetes mellitus diagnosed within the previous 3 months at time of screening 3. Fasting C-peptide level at time of screening above 0.1 nmol/L 4. Elevated GAD65 antibodies (GADA) at time of screening 5. Menarchal females must agree to avoid pregnancy and have a negative urine pregnancy test 6. Patients must agree to using adequate contraception, if sexually active, until 1 year after the last study drug administration 7. Must be willing to comply with intensive diabetes management 8. Written informed consent obtained from the patient and/or patient’s parents or legal acceptable representative(s) according to local regulations.
|
|
E.4 | Principal exclusion criteria |
1. Previous or current treatment with immunosuppressant therapy (although topical or inhaled steroids are accepted) 2. Treatment with any oral or injected anti-diabetic medications other than insulin 3. A history of anemia or significantly abnormal hematology 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 vaccines or other drugs in the past 6. Treatment with any vaccine within 1 month prior to planned first Diamyd dose or planned treatment with vaccine up to 2 months after the last injection with Diamyd, excluding the influenza vaccine 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. Known human immunodeficiency virus (HIV) or hepatitis 12. Females who are lactating or pregnant (for females who have started menstruating the possibility of pregnancy must be excluded by urine βHCG on-site within 24 hours prior to the investigational product administration) 13. Males or females not willing to use adequate contraception, if sexually active, until 1 year after the last study drug administration 14. Presence of associated serious disease or condition, including active skin infections that preclude subcutaneous injection, 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 change from baseline (Visit 2) to Month 15 (Visit 6) in C-peptide (AUCmean 0-120 min) during an MMTT. |
|
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) | 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 | Yes |
E.8.1.7.1 | Other trial design description |
|
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 | 20 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 50 |
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 | 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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 3 |