E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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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.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the safety and tolerability of a single dose of NG 3 mg in children aged 1 to <4 years with T1D |
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E.2.2 | Secondary objectives of the trial |
To assess the PD of a single dose of NG 3 mg in children aged 1 to <4 years with T1D; To assess the PK of a single dose of NG 3 mg in children aged 1 to <4 years with T1D |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Have a Type 1 Diabetes diagnosis for at least 6 months
Have been receiving insulin therapy via multiple daily injections or using an insulin pump and have been stable for at least 3 months prior to screening
Have a HbA1c level of ≤ 9.5% at screening
Have sufficient venous access for collection of blood samples
Have good general health, apart from their Type 1 diabetes, with no prior history of choanal atresia, nasal/pharyngeal blockage or nasal anomaly |
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E.4 | Principal exclusion criteria |
Have a presence or history of glucagon hypersensitivity
Have a history of pheochromocytoma
Have a history of epilepsy or seizure disorder
Have 1 or more congenital anomalies to the anatomy of the nose, or require changes to the anatomy of the nose
Are using closed-loop insulin therapy, unless such a device is set to 'open loop/manual' mode on the day of the dosing visit
Have an episode of severe hypoglycemia or have had glucagon administered, during the 3 months prior to the screening visit and no severe hypoglycemia between the screening and dosing visit |
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E.5 End points |
E.5.1 | Primary end point(s) |
Number of Participants with One or More Treatment-Emergent Adverse Event(s) (TEAEs) and Serious Adverse Event(s) (SAEs) Considered by the Investigator to be Related to Study Drug Administration [ Time Frame: Baseline through Day 9 ]
A summary of SAEs, TEAEs and other non-serious adverse events (AEs), regardless of causality, will be reported in the Reported Adverse Events module |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
[ Time Frame: Baseline through Day 9 ] |
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E.5.2 | Secondary end point(s) |
Pharmacodynamics (PD): Change From Baseline (Predose Blood Glucose) in Maximum Observed Blood Glucose (BGmax) of Nasal Glucagon [Time Frame: Predose through Day 1 ] PD: Change From Baseline (Predose Blood Glucose) in BGmax of Nasal Glucagon
PD: Absolute BGmax of Nasal Glucagon [ Time Frame: Predose through Day 1 ] PD: Absolute BGmax of Nasal Glucagon
PD: Time of Maximum Observed Blood Glucose (TBGmax) of Nasal Glucagon [ Time Frame: Predose through Day 1 ] PD: TBGmax of Nasal Glucagon
PD: Area Under the Concentration Versus Time Curve (AUC) of Blood Glucose [ Time Frame: Predose through Day 1 ] PD: AUC of Blood Glucose
Pharmacokinetics (PK): AUC of Nasal Glucagon [ Time Frame: Predose through Day 1 ] PK: AUC of Nasal Glucagon |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
[Time Frame: Predose through Day 1 ] |
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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 | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
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) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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 | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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 |
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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.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |