E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Treatment of Type 2 diabetes mellitus |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Body processes [G] - Metabolic Phenomena [G03] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10012594 |
E.1.2 | Term | Diabetes |
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 of this study is to test the hypothesis that glycemic control, with exenatide is superior, to that of placebo after 28 weeks of treatment in adolescent patients. |
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E.2.2 | Secondary objectives of the trial |
The main secondary objectives of the study are to compare exenatide 5 μg twice daily and exenatide 10 μg twice daily versus each other and versus placebo with regards to:
the proportion of patients achieving an HbA1c at endpoint of <7%,≤6.5%, and <6.5%
body weight
fasting serum glucose
self-monitored blood glucose |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients are eligible to be included in the study only if they meet all of the following criteria:
[1] between ages 10 to 17 years, inclusive.
[2] have a history of type 2 diabetes
[3] have been treated with metformin, an SU, or both metformin and an SU (with or without diet and exercise), for at least 3 months or are naïve to anti-diabetes agents and being treated with diet and exercise alone. The dose of oral agent(s) should be stable for the 30 days prior to the screening visit
[4] have fasting C-peptide >0.6 ng/mL
[5] have no antibodies to glutamic acid decarboxylase (GAD65) or islet cell antigen (ICA512)
[6] have HbA1c between 6.5% and 10.5%, inclusive
[7] present an appropriately signed assent form
[8] a parent or adult guardian agrees in writing to participate in the patient’s treatment by signing a consent form
[9] both the patient and parent or responsible adult guardian are able to understand and comply with a lifestyle modification program
[10] the investigator determines that patient and parent or responsible adult guardian are able to fully participate in and likely to complete the trial. |
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E.4 | Principal exclusion criteria |
Patients will be excluded from the study if they meet any of the following criteria:
[1] have received treatment within the last 60 days with a drug that has not received regulatory approval for any indication at the time of study entry
[2] have previously been exposed to exenatide or, completed or withdrawn from this study or any other study investigating exenatide
[3] are unwilling or unable to inject the study medication
[4] have a genetic syndrome or disorder other than diabetes known to affect glucose tolerance
[5] have a known allergy or hypersensitivity to exenatide or excipients contained in the agent
[6] have used an alpha-glucosidase inhibitor, a meglitinide, or pramlintide for
more than 1 week during the 3 months prior to screening
[7] currently use inhaled steroids at a dose equal to or above 1000ug Flovent (fluticasone propionate) daily
[8] have used oral steroids within the last 60 days or more than 20 days use within
the past year
[9] have used a TZD within 120 days prior to screening
[10] have used any weight loss medication(s) within 30 days of screening
[11] are sexually active female of childbearing potential who is unwilling to appropriately use TWO methods of birth control for the duration of the study
[12] are female who is pregnant or planning to become pregnant within 6 months of study screening
[13] are female who is lactating.
[14] have history of renal disease
[15] have hepatic dysfunction,
[16] have had at least 1 episode of diabetic ketoacidosis after receiving antidiabetes medication. A history of diabetic ketoacidosis at the time of diagnosis will not be an exclusion criterion
[17] have physical limitations that prevent participation in lifestyle intervention
[18] have admitted use of anabolic steroids within the past 60 days
[19] have an active or untreated malignancy, or have been in remission from clinically significant malignancy (other than in situ carcinomas of the cervix) for less than 5 years
[20] have investigator-determined significant organ system illness or condition,
including but not limited to psychiatric or developmental disorder that prevent participation in lifestyle intervention
[21] fail to satisfy the investigator of suitability to participate for any other reason
[22] have used insulin for more than 10 weeks during the 3 months prior to screening |
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E.5 End points |
E.5.1 | Primary end point(s) |
to test the hypothesis that glycemic control, with exenatide is superior, to that of placebo after 28 weeks of treatment in adolescent patients |
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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) |
The following main secondary efficacy measures will be collected as per the Study Schedule.
proportion of patient achieving an HbA1c <7%, ≤6.5%, and <6.5%
body weight
Fasting Serum Glucose
Self monitoring blood glucose |
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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 | 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 | 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.8.2.4 | Number of treatment arms in the trial | 3 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
India |
Mexico |
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.8.9.2 | In all countries concerned by the trial years | 8 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 1 |