Growth Hormone Deficiency
Conditions
Brief summary
This study will help to determine whether an injection log on a recombinant human growth hormone (rhGH) delivery device improves subjects' adherence with injection schedules. Adherence will be compared between a group of subjects who are aware of the injection log on the easypod™ rhGH delivery device and a group of subjects who are not aware of the easypod™ injection log. It is the study hypothesis that subject non-adherence rate is different for those who are aware of the injection log capability versus those who are unaware of the injection log capability. Subject perception will also be evaluated by comparing the ease and convenience of use and subject preference for the easypod™ compared to two other rhGH pen injection devices.
Interventions
DEVICEeasypod
Subjects is aware that the device records injection information that can be viewed by user
Sponsors
EMD Serono
Study design
Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Masking
SINGLE (Subject)
Eligibility
Sex/Gender
ALL
Age
10 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Be male or female from 10 to 18 years of age, inclusive, with open epiphyses * Have GH deficiency diagnosed by treating pediatric endocrinologist for at least 6 months * Be undergoing rhGH treatment via self-injection using the Lilly Humatrope® or Pfizer Genotropin® pen injection device for at least 6 months * Be willing and able to comply with the protocol for the duration of the trial * Have access to the Internet * Be able to read, speak and understand English * If female of childbearing potential, have a negative urine pregnancy test at Screening and use an acceptable form of birth control during the trial, including abstinence, a hormonal contraceptive, intrauterine device, diaphragm with spermicide, or condom with spermicide
Exclusion criteria
* Known allergy or hypersensitivity to rhGH or prescribed diluent (0.3% metacresol for click.easy® cartridge) * Severe illness during the previous 6 months * Active malignancy (except non-melanomatous skin malignancies) * Diabetes mellitus (type I or II) * Pregnancy or lactation * Any medical condition that, in the opinion of the Investigator, would jeopardize the subject's safety following exposure to the investigational device * Participation in any other investigational study during the duration of participation in this trial
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percent rhGH Injections Missed During the Treatment Period (Based on the Easypod™ Injection Log) | 90 Days |
Secondary
| Measure | Time frame |
|---|---|
| Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | 90 Days |
| Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | 90 Days |
| Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | 90 Days |
Countries
United States
Participant flow
Recruitment details
43 Subjects were randomized from 14 Clinics in the US from March 2008 through January 2009. One subject withdrew consent prior to first dose. Recruitment was ended early due to slow enrollment.
Pre-assignment details
Screening and randomization took place at Visit 1 (Study Day 1) which consisted of informed consent, medical/disease history, physical exam and laboratory assessments.
Participants by arm
| Arm | Count |
|---|---|
| Log Aware Dose Log Aware and Daily Diary Enabled | 21 |
| Log Unaware Dose Log UnaAware and Daily Diary Disabled | 21 |
| Total | 42 |
Baseline characteristics
| Characteristic | Log Unaware | Log Aware | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 21 Participants | 21 Participants | 42 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Continuous | 13.0 years STANDARD_DEVIATION 2.56 | 12.4 years STANDARD_DEVIATION 1.5 | 12.7 years STANDARD_DEVIATION 2.1 |
| Region of Enrollment United States | 21 participants | 21 participants | 42 participants |
| Sex: Female, Male Female | 3 Participants | 2 Participants | 5 Participants |
| Sex: Female, Male Male | 18 Participants | 19 Participants | 37 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 2 / 21 | 5 / 21 |
| serious Total, serious adverse events | 1 / 21 | 1 / 21 |
Outcome results
Primary
Percent rhGH Injections Missed During the Treatment Period (Based on the Easypod™ Injection Log)
Time frame: 90 Days
Population: The modified full analysis set includes all randomized subjects who received at least one injection using the easypod device and had valid device data. Seven subjects who had damaged devices or incorrect device setting were excluded from this analysis set.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Log Aware | Percent rhGH Injections Missed During the Treatment Period (Based on the Easypod™ Injection Log) | 20 Percent of injections missed |
| Log Unaware | Percent rhGH Injections Missed During the Treatment Period (Based on the Easypod™ Injection Log) | 19.46 Percent of injections missed |
| Total | Percent rhGH Injections Missed During the Treatment Period (Based on the Easypod™ Injection Log) | 20 Percent of injections missed |
Comparison: P-Value based on two-sided Wilcoxon rank-sum test for difference in medians between log aware and log unawarep-value: 0.908Wilcoxon (Mann-Whitney)
Secondary
Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices.
Time frame: 90 Days
Population: The modified full analysis set includes all randomized subjects who received at least one injection using the easypod device and had valid device data. In addition, this total of 35 subjects was divided into 2 groups, Humatrope and Genotropin, based on their previous rhGH use.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Log Aware | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Agree | 2 Participants |
| Log Aware | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Missing | 5 Participants |
| Log Aware | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Strongly Agree | 2 Participants |
| Log Aware | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Neutral | 2 Participants |
| Log Aware | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Disagree | 2 Participants |
| Log Aware | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Strongly Disagree | 3 Participants |
| Log Unaware | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Disagree | 3 Participants |
| Log Unaware | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Strongly Disagree | 4 Participants |
| Log Unaware | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Agree | 2 Participants |
| Log Unaware | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Neutral | 1 Participants |
| Log Unaware | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Missing | 3 Participants |
| Log Unaware | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Strongly Agree | 6 Participants |
| Total | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Missing | 8 Participants |
| Total | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Agree | 4 Participants |
| Total | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Neutral | 3 Participants |
| Total | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Disagree | 5 Participants |
| Total | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Strongly Disagree | 7 Participants |
| Total | Subject Perceptions of Easypod: Preference to Use Easypod Over Two Other rhGH Pen Injection Devices. | Strongly Agree | 8 Participants |
Secondary
Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices.
Time frame: 90 Days
Population: The modified full analysis set includes all randomized subjects who received at least one injection using the easypod device and had valid device data. In addition, this total of 35 subjects was divided into 2 groups, Humatrope and Genotropin, based on their previous rhGH use.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Log Aware | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Very Convenient | 3 Participants |
| Log Aware | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Convenient | 3 Participants |
| Log Aware | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Neutral | 5 Participants |
| Log Aware | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Not Very Convenient | 0 Participants |
| Log Aware | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Not at all Convenient | 0 Participants |
| Log Aware | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Missing | 5 Participants |
| Log Unaware | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Missing | 3 Participants |
| Log Unaware | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Very Convenient | 7 Participants |
| Log Unaware | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Not Very Convenient | 0 Participants |
| Log Unaware | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Not at all Convenient | 1 Participants |
| Log Unaware | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Convenient | 5 Participants |
| Log Unaware | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Neutral | 3 Participants |
| Total | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Convenient | 8 Participants |
| Total | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Neutral | 8 Participants |
| Total | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Missing | 8 Participants |
| Total | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Not Very Convenient | 0 Participants |
| Total | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Very Convenient | 10 Participants |
| Total | Subject Perceptions of Easypod: Storage Convenience Compared to Two Other rhGH Pen Injection Devices. | Not at all Convenient | 1 Participants |
Secondary
Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices
Time frame: 90 Days
Population: The modified full analysis set includes all randomized subjects who received at least one injection using the Easypod device and had valid device data. In addition, this total of 35 subjects was divided into 2 groups, Humatrope and Genotropin, based on their previous rhGH use.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Log Aware | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Neutral | 0 Participants |
| Log Aware | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Strongly Agree | 5 Participants |
| Log Aware | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Agree | 1 Participants |
| Log Aware | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Disagree | 3 Participants |
| Log Aware | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Strongly Disagree | 2 Participants |
| Log Aware | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Missing | 5 Participants |
| Log Unaware | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Agree | 3 Participants |
| Log Unaware | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Neutral | 4 Participants |
| Log Unaware | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Disagree | 1 Participants |
| Log Unaware | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Missing | 3 Participants |
| Log Unaware | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Strongly Disagree | 3 Participants |
| Log Unaware | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Strongly Agree | 5 Participants |
| Total | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Strongly Agree | 10 Participants |
| Total | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Strongly Disagree | 5 Participants |
| Total | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Neutral | 4 Participants |
| Total | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Missing | 8 Participants |
| Total | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Agree | 4 Participants |
| Total | Subjects Perception of Easypod Ease of Use Compared to Two Other rhGH Pen Injection Devices | Disagree | 4 Participants |