Clinical Trial Results:
A Multiple Center, Open Label, Prospective Study to Evaluate the Effectiveness and Ease-Of-Use of AMG504-1 Administered in the Home or School Environments for Treating Hypoglycemia in Children and Adolescents with T1D
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Summary
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EudraCT number |
2015-003732-12 |
Trial protocol |
Outside EU/EEA |
Global end of trial date |
21 Aug 2015
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Results information
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Results version number |
v1 |
This version publication date |
18 Jun 2017
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First version publication date |
18 Jun 2017
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Other versions |
v2 |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
16423
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT02402933 | ||
WHO universal trial number (UTN) |
- | ||
Other trial identifiers |
Trial Number: 16423, Trial Alias: I8R-MC-B001, Other: AMG109 | ||
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Sponsors
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Sponsor organisation name |
Eli Lilly and Company
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Sponsor organisation address |
Lilly Corporate Center, Indianapolis, IN, United States, 46285
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Public contact |
Available Mon - Fri 9 AM - 5 PM EST , Eli Lilly and Company, 1 877-CTLilly,
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Scientific contact |
Available Mon - Fri 9 AM - 5 PM EST , Eli Lilly and Company, 1 877-285-4559,
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
Yes
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EMA paediatric investigation plan number(s) |
EMEA-001657-PIP01-14 | ||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
21 Aug 2015
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
21 Aug 2015
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
Up to fifty (50) children and adolescents with type 1 diabetes (T1D) aged 4 to less than 18 years at time of enrolment will be selected for inclusion in the study. The target is to obtain treatment response and user-experience data following use of AMG504-1 (LY900018) in treating episodes of hypoglycemia.
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Protection of trial subjects |
This study was conducted in accordance with International Conference on Harmonization (ICH) Good Clinical Practice, and the principles of the Declaration of Helsinki, in addition to following the laws and regulations of the country or countries in which a study is conducted.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
10 Mar 2015
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
United States: 26
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Worldwide total number of subjects |
26
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EEA total number of subjects |
0
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
11
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Adolescents (12-17 years) |
15
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
Participants and their principal caregiver(s) (such as parents, family member, roommate, teacher, and coach) will be trained in the use of AMG504-1 (Dry-Mist nasal glucagon powder). | ||||||||||||||
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Pre-assignment
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Screening details |
No Text Entered | ||||||||||||||
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Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||||||
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Arms
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Arm title
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Nasal Glucagon | ||||||||||||||
Arm description |
Four doses of AMG504-1 (3 mg glucagon powder per dose) were dispensed to participants and each participant/caregiver were encouraged to keep one dose with them at all times and the other doses in a convenient location. | ||||||||||||||
Arm type |
Experimental | ||||||||||||||
Investigational medicinal product name |
Nasal Glucagon
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Investigational medicinal product code |
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Other name |
Dry-Mist nasal glucagon, AMG504-1, LY900018
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Pharmaceutical forms |
Inhalation solution
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Routes of administration |
Nasal use
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Dosage and administration details |
3 mg glucagon powder
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Baseline characteristics reporting groups
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Reporting group title |
Nasal Glucagon
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Reporting group description |
Four doses of AMG504-1 (3 mg glucagon powder per dose) were dispensed to participants and each participant/caregiver were encouraged to keep one dose with them at all times and the other doses in a convenient location. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Nasal Glucagon
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Reporting group description |
Four doses of AMG504-1 (3 mg glucagon powder per dose) were dispensed to participants and each participant/caregiver were encouraged to keep one dose with them at all times and the other doses in a convenient location. | ||
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End point title |
Number of Participants Awakening or Returning to a Normal Status Within 30 Minutes Following Studied Drug of Administration [1] | ||||||||
End point description |
Number of participants with effectiveness of nasal glucagon administered under clinical use in treating episodes of hypoglycemia in children and adolescents with T1D, as defined by clinical recovery following studied drug administration. Responses to questions completed by the caregiver are used to assess this outcome.
An episode of severe hypoglycemia is generally defined as an event associated with severe neuroglycopenia usually resulting in coma or seizure and requiring parenteral therapy (glucagon or intravenous glucose) administered by a third party. In this study moderate hypoglycemia is defined as an episode wherein the child/adolescent with diabetes has symptoms and/or signs of neuroglycopenia and has a blood glucose ≤3.9 millimole per liter (mmol/L) (70 milligram per deciliter [mg/dL]) based on a blood sample taken at or close to the time of treatment.
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End point type |
Primary
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End point timeframe |
Within 30 minutes after each drug administration for an episode of hypoglycemia
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: this is a single arm study and no statistical comparison was planned. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Assessment of Ease-of-use of Dry-Mist Nasal Glucagon by Completion of Questionnaires by the Caregiver | ||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Assess ease-of-use of intranasally administered glucagon in the hands of caregivers of participants who may be called upon to treat episodes of hypoglycemia.
Measurement for Degree of difficulty: opening the kit, Degree of difficulty: understanding the instructions on how to use the kit, Degree of difficulty: administering the medication into the nostril, Degree of satisfaction is 1 (Very Difficult) to 7 (Very Easy). Measurement for Dry Mist Nasal Glucagon will be easy to teach other caregivers, Nasal formulation of glucagon is less intimidating for caregivers, Dry Mist Nasal Glucagon is easy to carry and would be willing to carry it, Intranasal delivery of glucagon is preferable: level of agreement 1 (Strongly Disagree) to 7 (Strongly Agree).
Population: Proportions and n are based on the total number of hypoglycemic events (N=33) of 14 participants.
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End point type |
Secondary
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End point timeframe |
After each drug administration for an episode of hypoglycemia
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| Notes [2] - Except compare of administration (much easier, easier, about the same) is based on n=8 events. |
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||
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End point title |
Percentage of Participants With Adverse Events Solicited through Nasal Score Questionnaire | ||||||||
End point description |
An adverse event is a clinical event is a health change from the participant’s normal state, as defined during the pre-trial evaluation and enrollment. Adverse events were assessed based on participant's responses to the Nasal Score Questionnaire administered within 2 hours of recovery after the dosing event.
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End point type |
Secondary
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End point timeframe |
Within 2 hours of full recovery from a hypoglycemic event
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| No statistical analyses for this end point | |||||||||
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End point title |
Change in Blood Glucose Level Over Time | ||||||||||||||
End point description |
Glucometer-based measurements of blood glucose after the studied drug administration. The participants’ change in blood glucose level from baseline was measured by the caregiver using a glucometer at 15, 30 and 45 minutes after IN glucagon administration.
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End point type |
Secondary
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End point timeframe |
Baseline, 15, 30 and 45 minutes after drug administration for an episode of hypoglycemia
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| No statistical analyses for this end point | |||||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
Entire Study
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Adverse event reporting additional description |
Adverse events were collected systematically by the Caregiver using the Hypoglycemia Questionnaire, and by the Participant using the Nasal Score Questionnaire.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
18.1
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Reporting groups
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Reporting group title |
Nasal Glucagon
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Reporting group description |
3 mg glucagon powder | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 0% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| Participants from non-GCP compliant site were excluded from the analysis. | |||
