E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Diabetes Mellitus, Type 2 |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10045242 |
E.1.2 | Term | Type II diabetes mellitus |
E.1.2 | System Organ Class | 100000004861 |
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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 |
To confirm the superiority of liraglutide at the maximum tolerated dose (0.6 mg, 1.2 mg or 1.8 mg)versus placebo when added to in combination with metformin with or without basal insulin treatment in controlling glycaemia versus metformin and liraglutide placebo in children, and adolescents (ages 10–17 years) with type 2 diabetes. |
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E.2.2 | Secondary objectives of the trial |
To assess and compare the effect of liraglutide versus placebo in combination with metformin with or without basal insulin treatment. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
-Children and adolescents between the ages of 10–16 years. Subjects cannot turn 17 years and 11 months before the end of treatment (52 weeks)
- Diagnosis of type 2 diabetes mellitus and treated for at least 30 days with:
- diet and exercise alone
- diet and exercise in combination with metformin monotherapy
- diet and exercise in combination with metformin and a stable* dose of basal insulin
- diet and exercise in combination with a stable* dose of basal insulin.
*Stable is defined as basal insulin adjustments up to 15%
- HbA1c
- ≥7.0% and ≤11% if diet and exercise treated
- ≥6.5% and ≤11% if treated with metformin as monotherapy, basal insulin as monotherapy or metformin and basal insulin in combination
- Body mass index (BMI) >85% percentile of the general age and gender
matched population |
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E.4 | Principal exclusion criteria |
- Type 1 diabetes
- Maturity onset diabetes of the young (MODY)
- Use of any antidiabetic agent other than metformin and/or basal insulin within 90 days prior to screening
- Recurrent severe hypoglycaemia or hypoglycaemic unawareness as judged by the investigator
- History of chronic pancreatitis or idiopathic acute pancreatitis
- Any clinically significant disorder, except for conditions associated with type 2 diabetes history which in the investigator’s opinion could interfere with results of the trial
- Uncontrolled hypertension, treated or untreated >99th percentile for age and gender in children
- Known or suspected abuse of alcohol or narcotics |
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E.5 End points |
E.5.1 | Primary end point(s) |
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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) |
1. HbA1c <7.0% (yes/no)
2. HbA1c ≤6.5% (yes/no)
3. HbA1c <7.0% without severe or minor hypoglycaemic episodes (yes/no)
Change from baseline:
4. Fasting plasma glucose (FPG)
5. 7-point self-measured plasma glucose
6. body weight
7. BMI standard deviation score (SDS)
Safety
8. Adverse events (AEs) and serious adverse events (SAEs)
9. Safety follow-up after 1 and 2 years: AEs and SAEs, growth velocity and pubertal progression |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1-7: at 26 and 52 weeks of treatment
8-9: After 53 weeks, and after 1 and 2 years after LPLV |
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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 | 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 |
Followed by 26-week open-label extension |
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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.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 30 |
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 | No |
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 |
Australia |
Austria |
Belgium |
Canada |
Croatia |
Denmark |
Egypt |
France |
Greece |
Hungary |
India |
Israel |
Lebanon |
Macedonia, the former Yugoslav Republic of |
Malaysia |
Mexico |
Morocco |
Netherlands |
New Zealand |
Norway |
Poland |
Portugal |
Romania |
Russian Federation |
Serbia |
Spain |
Sweden |
Taiwan |
Thailand |
Turkey |
United Kingdom |
United States |
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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
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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 | 5 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |