Growth Hormone Deficiency, Idiopathic Short Stature
Conditions
Keywords
Genotropin Medical device Patient reported outcome
Brief summary
Assessment of Genotropin patient and caregiver (Dyad) perception of convenience and preference of Genotropin injection pen. Patients already on genotropin will be asked to use a genotropin pen for 2 months. Patient and caregiver will be asked to complete a questionnaire at baseline and 2 months.
Interventions
DEVICENew Genotropin Pen
Subjects will use the genotropin pen for 2 months. After 2 months patients and caregiver will be asked to fill out a questionnaire to assess perception of the genotropin pen
Sponsors
Pfizer
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
8 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Age 8-18 years * Using genotropin pen for at least 3 months prior to study enrollment * Compliance with genotropin treatment
Exclusion criteria
* Medical conditions that can affect participation in study * Insufficient command of English language to understand questionnaire * Using other growth hormone device and not Genotropin pen
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Dyads (Participant and Caregiver or Parent) Reporting no Difference or Easier to Use for New Genotropin Mark VII Injection Pen Compared to Pre-study Experience With the Genotropin Pen® | 2 months | Ease of use measured using the Injection Pen Assessment Questionnaire (IPAQ) patient-reported outcome (PRO) tool (based on 13 unique characteristics of injection pens). Section I measures ease of use of Genotropin® (very easy, somewhat easy, neither easy nor difficult, somewhat difficult, or very difficult). Section II measures ease of use of new Genotropin Mark VII Pen in comparison to pre-study experience with Genotropin® Pen (Genotropin® pen easier to use, new injection pen easier to use, or no difference) and preference (prefer Genotropin® Pen, prefer new injection pen, or no preference). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Dyads Reporting no Preference or Preference for New Genotropin Mark VII Injection Pen Compared to Pre-study Experience With the Genotropin Pen® | 2 months | Preference for use measured using IPAQ PRO tool (ease of use and preference based on 13 unique characteristics of injection pens). Section I measures ease of use of Genotropin® (very easy, somewhat easy, neither easy nor difficult, somewhat difficult, or very difficult). Section II measures ease of use of new Genotropin Mark VII Pen in comparison to pre-study experience with Genotropin® Pen (Genotropin® Pen easier to use, new injection pen easier to use, or no difference) and preference (prefer Genotropin® Pen, prefer new injection pen, or no preference). |
| Percentage of Dyads Reporting New Genotropin Mark VII Injection Pen Easier to Use Compared to Pre-study Experience With the Genotropin Pen® | 2 months | Ease of use measured using the IPAQ PRO tool (ease of use and preference based on 13 unique characteristics of injection pens). Section I measures ease of use of Genotropin® Pen (very easy, somewhat easy, neither easy nor difficult, somewhat difficult, or very difficult). Section II measures ease of use of new Genotropin Mark VII Pen in comparison to pre-study experience with Genotropin® Pen (Genotropin® Pen easier to use, new injection pen easier to use, or no difference) and preference (prefer Genotropin® Pen, prefer new injection pen, or no preference). |
| Percentage of Dyads Reporting New Genotropin Mark VII Injection Pen Preferable Compared to Pre-study Experience With the Genotropin Pen® | 2 months | Preference for use measured using IPAQ PRO tool (ease of use and preference based on 13 unique characteristics of injection pens). Section I measures ease of use of Genotropin® (very easy, somewhat easy, neither easy nor difficult, somewhat difficult, or very difficult). Section II measures ease of use of new Genotropin Mark VII Pen in comparison to pre-study experience with Genotropin® Pen (Genotropin® Pen easier to use, new injection pen easier to use, or no difference) and preference (prefer Genotropin® Pen, prefer new injection pen, or no preference). |
Countries
United States
Participant flow
Pre-assignment details
Participants who used the Genotropin® Pen for at least 3 months prior to enrollment were eligible to participate. Genotropin (somatropin) dose was not adjusted for the purposes of the study, but only based on clinical management requirements as determined by the treating physician.
Participants by arm
| Arm | Count |
|---|---|
| Genotropin / Genotropin Mark VII Pen Genotropin (somatropin) injection administered using the Genotropin Mark VII Pen subcutaneously (sc) at a dose prescribed by the physician. | 136 |
| Total | 136 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Protocol Violation | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Genotropin / Genotropin Mark VII Pen |
|---|---|
| Age, Customized ≤10 years of age | 37 participants |
| Age, Customized ≥15 years of age | 29 participants |
| Age, Customized Between 11 and 12 years of age | 30 participants |
| Age, Customized Between 13 and 14 years of age | 40 participants |
| Sex: Female, Male Female | 45 Participants |
| Sex: Female, Male Male | 91 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 28 / 136 |
| serious Total, serious adverse events | 0 / 136 |
Outcome results
Primary
Percentage of Dyads (Participant and Caregiver or Parent) Reporting no Difference or Easier to Use for New Genotropin Mark VII Injection Pen Compared to Pre-study Experience With the Genotropin Pen®
Ease of use measured using the Injection Pen Assessment Questionnaire (IPAQ) patient-reported outcome (PRO) tool (based on 13 unique characteristics of injection pens). Section I measures ease of use of Genotropin® (very easy, somewhat easy, neither easy nor difficult, somewhat difficult, or very difficult). Section II measures ease of use of new Genotropin Mark VII Pen in comparison to pre-study experience with Genotropin® Pen (Genotropin® pen easier to use, new injection pen easier to use, or no difference) and preference (prefer Genotropin® Pen, prefer new injection pen, or no preference).
Time frame: 2 months
Population: Full analysis set (FAS): all participants who used the new pen at least once to administer Genotropin and who completed the 2-month follow-up questionnaire. Dyad defined as the participant (child being treated) and adult partner (parent or caregiver).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Genotropin / Genotropin Mark VII Pen | Percentage of Dyads (Participant and Caregiver or Parent) Reporting no Difference or Easier to Use for New Genotropin Mark VII Injection Pen Compared to Pre-study Experience With the Genotropin Pen® | 73.68 percentage of dyads |
Secondary
Percentage of Dyads Reporting New Genotropin Mark VII Injection Pen Easier to Use Compared to Pre-study Experience With the Genotropin Pen®
Ease of use measured using the IPAQ PRO tool (ease of use and preference based on 13 unique characteristics of injection pens). Section I measures ease of use of Genotropin® Pen (very easy, somewhat easy, neither easy nor difficult, somewhat difficult, or very difficult). Section II measures ease of use of new Genotropin Mark VII Pen in comparison to pre-study experience with Genotropin® Pen (Genotropin® Pen easier to use, new injection pen easier to use, or no difference) and preference (prefer Genotropin® Pen, prefer new injection pen, or no preference).
Time frame: 2 months
Population: FAS
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Genotropin / Genotropin Mark VII Pen | Percentage of Dyads Reporting New Genotropin Mark VII Injection Pen Easier to Use Compared to Pre-study Experience With the Genotropin Pen® | 63.16 percentage of dyads |
Secondary
Percentage of Dyads Reporting New Genotropin Mark VII Injection Pen Preferable Compared to Pre-study Experience With the Genotropin Pen®
Preference for use measured using IPAQ PRO tool (ease of use and preference based on 13 unique characteristics of injection pens). Section I measures ease of use of Genotropin® (very easy, somewhat easy, neither easy nor difficult, somewhat difficult, or very difficult). Section II measures ease of use of new Genotropin Mark VII Pen in comparison to pre-study experience with Genotropin® Pen (Genotropin® Pen easier to use, new injection pen easier to use, or no difference) and preference (prefer Genotropin® Pen, prefer new injection pen, or no preference).
Time frame: 2 months
Population: FAS; number of participants with measurement.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Genotropin / Genotropin Mark VII Pen | Percentage of Dyads Reporting New Genotropin Mark VII Injection Pen Preferable Compared to Pre-study Experience With the Genotropin Pen® | 59.85 percentage of dyads |
Secondary
Percentage of Dyads Reporting no Preference or Preference for New Genotropin Mark VII Injection Pen Compared to Pre-study Experience With the Genotropin Pen®
Preference for use measured using IPAQ PRO tool (ease of use and preference based on 13 unique characteristics of injection pens). Section I measures ease of use of Genotropin® (very easy, somewhat easy, neither easy nor difficult, somewhat difficult, or very difficult). Section II measures ease of use of new Genotropin Mark VII Pen in comparison to pre-study experience with Genotropin® Pen (Genotropin® Pen easier to use, new injection pen easier to use, or no difference) and preference (prefer Genotropin® Pen, prefer new injection pen, or no preference).
Time frame: 2 months
Population: FAS; N=number of participants with measurement.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Genotropin / Genotropin Mark VII Pen | Percentage of Dyads Reporting no Preference or Preference for New Genotropin Mark VII Injection Pen Compared to Pre-study Experience With the Genotropin Pen® | 65.15 percentage of dyads |