Type 1 Diabetes
Conditions
Keywords
Severe Hypoglycemia, Nasal Powder, Hypoglycemia Rescue Therapy, Ready to use Glucagon
Brief summary
The main purpose of this study is to evaluate the safety and tolerability of a study drug called nasal glucagon (Baqsimi) in pediatric participants with type 1 diabetes (T1D) aged 1 to less than 4 years. Blood tests will be performed to check how much nasal glucagon gets into the bloodstream. Blood sugar will also be measured to understand the effect of the drug on blood sugar levels. The study consists of a screening period up to 35 days before dosing, 1 day when a dose of nasal glucagon will be given and then 2 telephone follow up calls; first follow-up call on the day after the nasal glucagon was given and second call about one week after nasal glucagon was given. The study will last up to 9 days, not including the screening period.
Interventions
DRUGGlucagon Nasal Powder [Baqsimi]
Administered intranasally
Sponsors
Eli Lilly and Company
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 4 Years
Healthy volunteers
No
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
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Baseline to Day 9 | A TEAE is defined as an adverse event which occurs post-dose or which is present prior to dosing and becomes more severe post-dose. An SAE is any AE from the study that results in 1 of the following: Death, initial or prolonged inpatient hospitalization, a life-threatening experience (i.e., immediate risk of dying), persistent or significant disability/incapacity, congenital anomaly/birth defect, important medical events that may not be immediately life-threatening or result in death or hospitalization but may jeopardize the subject or may require intervention to prevent 1 of the other outcomes listed in the definition above. The number of participants with one or more TEAEs, SAEs considered by the investigator to be related to study drug administration is reported here. A summary of SAEs and other non-serious AEs, regardless of causality is located in the Reported Adverse Events section of this record. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacodynamics (PD): Change From Baseline in Maximum Observed Blood Glucose (BGmax) | Baseline, Day 1 (pre-dose, 10, 30, 60, 90 minutes post-dose) | Change from baseline in BGmax was measured to investigate the PD effect of nasal glucagon on blood glucose level following 3 mg nasal glucagon administration on day 1. Baseline is defined as Day 1 pre-dose. The BGmax on Day 1 was determined using plasma samples collected pre-dose, 10, 30, 60, and 90 minutes post nasal glucagon dose. |
| PD: Absolute BGmax of Nasal Glucagon | Day 1 (pre-dose, 10, 30, 60, 90 minutes post-dose) | PD: Absolute BGmax of Nasal Glucagon |
| PD: Time of Maximum Observed Blood Glucose (TBGmax) of Nasal Glucagon | Day 1 (pre-dose, 10, 30, 60, 90 minutes post-dose) | PD: TBGmax of Nasal Glucagon |
| PD: Area Under the Concentration Versus Time Curve (AUC) of Blood Glucose | Day 1 (pre-dose, 10, 30, 60, 90 minutes post-dose) | AUC from time 0 to the last measured concentration of blood glucose at 90 minutes \[AUC(0-90)\] is reported. |
| Pharmacokinetics (PK): AUC of Nasal Glucagon | Day 1 (10, 30, 60 minutes post-dose) | PK: AUC of Nasal Glucagon |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 3 mg Nasal Glucagon Participants received a single dose of 3 mg glucagon powder administered intranasally on day 1. | 7 |
| Total | 7 |
Baseline characteristics
| Characteristic | 3 mg Nasal Glucagon |
|---|---|
| Age, Continuous | 2.98 years STANDARD_DEVIATION 0.82 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 6 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 7 Participants |
| Region of Enrollment United States | 7 Participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 7 |
| other Total, other adverse events | 5 / 7 |
| serious Total, serious adverse events | 0 / 7 |
Outcome results
Primary
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
A TEAE is defined as an adverse event which occurs post-dose or which is present prior to dosing and becomes more severe post-dose. An SAE is any AE from the study that results in 1 of the following: Death, initial or prolonged inpatient hospitalization, a life-threatening experience (i.e., immediate risk of dying), persistent or significant disability/incapacity, congenital anomaly/birth defect, important medical events that may not be immediately life-threatening or result in death or hospitalization but may jeopardize the subject or may require intervention to prevent 1 of the other outcomes listed in the definition above. The number of participants with one or more TEAEs, SAEs considered by the investigator to be related to study drug administration is reported here. A summary of SAEs and other non-serious AEs, regardless of causality is located in the Reported Adverse Events section of this record.
Time frame: Baseline to Day 9
Population: All participants who received at least one dose of study drug.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| 3 mg Nasal Glucagon | 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 | TEAEs | 5 Participants |
| 3 mg Nasal Glucagon | 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 | SAEs | 0 Participants |
Secondary
PD: Absolute BGmax of Nasal Glucagon
PD: Absolute BGmax of Nasal Glucagon
Time frame: Day 1 (pre-dose, 10, 30, 60, 90 minutes post-dose)
Population: All participants who received at least one dose of study drug and had evaluable PD data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| 3 mg Nasal Glucagon | PD: Absolute BGmax of Nasal Glucagon | 242.1 mg/dL | Standard Deviation 46 |
Secondary
PD: Area Under the Concentration Versus Time Curve (AUC) of Blood Glucose
AUC from time 0 to the last measured concentration of blood glucose at 90 minutes \[AUC(0-90)\] is reported.
Time frame: Day 1 (pre-dose, 10, 30, 60, 90 minutes post-dose)
Population: All participants who received at least one dose of study drug and had evaluable PD data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| 3 mg Nasal Glucagon | PD: Area Under the Concentration Versus Time Curve (AUC) of Blood Glucose | 18200 milligram*minute per deciliter (mg*m/dL) | Standard Deviation 3000 |
Secondary
PD: Time of Maximum Observed Blood Glucose (TBGmax) of Nasal Glucagon
PD: TBGmax of Nasal Glucagon
Time frame: Day 1 (pre-dose, 10, 30, 60, 90 minutes post-dose)
Population: All participants who received at least one dose of study drug and had evaluable PD data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| 3 mg Nasal Glucagon | PD: Time of Maximum Observed Blood Glucose (TBGmax) of Nasal Glucagon | 55.6 minutes | Standard Deviation 20.9 |
Secondary
Pharmacodynamics (PD): Change From Baseline in Maximum Observed Blood Glucose (BGmax)
Change from baseline in BGmax was measured to investigate the PD effect of nasal glucagon on blood glucose level following 3 mg nasal glucagon administration on day 1. Baseline is defined as Day 1 pre-dose. The BGmax on Day 1 was determined using plasma samples collected pre-dose, 10, 30, 60, and 90 minutes post nasal glucagon dose.
Time frame: Baseline, Day 1 (pre-dose, 10, 30, 60, 90 minutes post-dose)
Population: All participants who received at least one dose of study drug and had evaluable PD data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| 3 mg Nasal Glucagon | Pharmacodynamics (PD): Change From Baseline in Maximum Observed Blood Glucose (BGmax) | 132.4 milligrams per deciliter (mg/dL) | Standard Deviation 52.4 |
Secondary
Pharmacokinetics (PK): AUC of Nasal Glucagon
PK: AUC of Nasal Glucagon
Time frame: Day 1 (10, 30, 60 minutes post-dose)
Population: All participants who received at least one dose of study drug and had evaluable PK data.
| Arm | Measure | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|
| 3 mg Nasal Glucagon | Pharmacokinetics (PK): AUC of Nasal Glucagon | 1560 picogram*hour per milliliter (pg*hr/mL) | Geometric Coefficient of Variation 25.3 |