Growth Hormone Deficiency
Conditions
Keywords
Growth Hormone Deficiency, Somatropin, Injection Pen, Medical Device, Cross Over Study, Preference, Convenience, Children, Dyads, Adults
Brief summary
Convenience and preference for the new Mark VII pen compared to the current Genotropin pen will be assessed using a questionaire. it is expected that the new pen will be preferred or at least no different to the current pen.
Interventions
DEVICEGenotropin Pen
Pen provided in 5, 5.3 and 12 mg doses, Dose based on body weight, subcutaneous injection daily for 2 months while in study.
DEVICEMARK VII pen
Pen provided in 5, 5.3 and 12mg doses, dose based on body weight, subcutaneous injection daily for 2 months while in study.
Sponsors
Pfizer
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
4 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Age greater than or equal to 8 years. * Subjects who are starting treatment with growth hormone (Genotropin) for the first time.
Exclusion criteria
* Subjects with Prader-Willi syndrome or chronic renal insufficiency. * Subjects and caregivers who, in the opinion of the investigator are not able to adequately participate in the study. * Subjects with chronic systemic disorders, such as diabetes and heart disease.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Dyads (Participant and Caregiver or Parent) and Adult Participants Reporting no Difference or Easier to Use for the New Genotropin Mark VII Injection Pen Compared to the Genotropin Pen® | Month 4 | Participants were asked the following question from Section II of the Injection Pen Assessment Questionnaire (IPAQ) patient-reported outcome (PRO) tool, Thinking about the Genotropin pen and the new injection pen you used over the past few months, please compare both injection pens and choose which one is easier to use overall? Choices included: Genotropin Pen® easier to use, new injection pen easier to use, or no difference. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Dyads and Adult Participants Reporting no Preference or Preference for the New Genotropin Mark VII Injection Pen Compared to the Genotropin Pen® | Month 4 | Participants were asked the following question from Section II of the IPAQ PRO tool, Thinking about both injection pens over the past few months, please choose which injection pen you prefer overall. Choices included: prefer Genotropin Pen®, prefer new injection pen, or no preference. |
| Percentage of Dyads and Adult Participants Reporting the New Genotropin Mark VII Injection Pen Easier to Use Compared to the Genotropin Pen® | Month 4 | Participants were asked the following question from Section II of the IPAQ PRO tool, Thinking about the Genotropin pen and the new injection pen you used over the past few months, please compare both injection pens and choose which one is easier to use overall? Choices included: Genotropin Pen® easier to use, new injection pen easier to use, or no difference. |
| Percentage of Dyads and Adult Participants Reporting the New Genotropin Mark VII Injection Pen Preferable Compared to the Genotropin Pen® | Month 4 | Participants were asked the following question from Section II of the IPAQ PRO tool, Thinking about both injection pens over the past few months, please choose which injection pen you prefer overall. Choices included: prefer Genotropin Pen®, prefer new injection pen, or no preference. |
| Percentage of Dyads and Adult Participants Who Would Choose the New Genotropin Mark VII Injection Pen in Preference to the Genotropin® Pen | Month 4 | Investigators were asked the following study treatment continuation question, Which device did the participant choose for continued treatment? Choices included the Genotropin® Pen or the new injection pen. |
| Ease of Use of Each Injection Pen | Month 2 and Month 4 | Participants were asked the following question from Section I of the IPAQ PRO tool, Thinking about the injection pen you have been using for the past few months, how easy or difficult it is for you to use the injection pen overall? Responses were provided using a 5 point scale which ranged from very easy (5), somewhat easy (4), neither easy nor difficult (3), somewhat difficult (2), or very difficult (1). |
Countries
Czechia, Germany, Netherlands, Slovakia, Sweden, Turkey (Türkiye), United Kingdom
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Entire Study Population Includes participants randomized to first use the Mark VII pen (subcutaneous injections daily for 2 months) and participants randomized to first use the current Genotropin® pen (subcutaneous injections daily 2 months). Both pens provided in 5, 5.3, and 12 mg doses of Genotropin (somatropin \[rDNA origin\]); doses received by participants based on body weight. | 120 |
| Total | 120 |
Baseline characteristics
| Characteristic | Entire Study Population |
|---|---|
| Age, Customized ≥65 years | 2 participants |
| Age, Customized ≤7 years | 28 participants |
| Age, Customized Between 18 and 44 years | 18 participants |
| Age, Customized Between 45 and 64 years | 22 participants |
| Age, Customized Between 8 and 17 years | 50 participants |
| Sex: Female, Male Female | 51 Participants |
| Sex: Female, Male Male | 69 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 48 / 120 |
| serious Total, serious adverse events | 2 / 120 |
Outcome results
Primary
Percentage of Dyads (Participant and Caregiver or Parent) and Adult Participants Reporting no Difference or Easier to Use for the New Genotropin Mark VII Injection Pen Compared to the Genotropin Pen®
Participants were asked the following question from Section II of the Injection Pen Assessment Questionnaire (IPAQ) patient-reported outcome (PRO) tool, Thinking about the Genotropin pen and the new injection pen you used over the past few months, please compare both injection pens and choose which one is easier to use overall? Choices included: Genotropin Pen® easier to use, new injection pen easier to use, or no difference.
Time frame: Month 4
Population: Full Analysis Set (FAS): randomized participants who used a study pen at least once to administer somatropin; Number of participants analyzed (N)= participants with evaluable data. Dyad defined as the participant (child being treated) and adult partner (parent or caregiver).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Entire Study Population | Percentage of Dyads (Participant and Caregiver or Parent) and Adult Participants Reporting no Difference or Easier to Use for the New Genotropin Mark VII Injection Pen Compared to the Genotropin Pen® | 67.23 percentage of dyads, adult participants |
Secondary
Ease of Use of Each Injection Pen
Participants were asked the following question from Section I of the IPAQ PRO tool, Thinking about the injection pen you have been using for the past few months, how easy or difficult it is for you to use the injection pen overall? Responses were provided using a 5 point scale which ranged from very easy (5), somewhat easy (4), neither easy nor difficult (3), somewhat difficult (2), or very difficult (1).
Time frame: Month 2 and Month 4
Population: FAS; Number of participants analyzed (N) = participants with evaluable data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Entire Study Population | Ease of Use of Each Injection Pen | 4.5 scores on a scale | Standard Deviation 0.64 |
| Genotropin® Pen | Ease of Use of Each Injection Pen | 4.2 scores on a scale | Standard Deviation 0.71 |
Secondary
Percentage of Dyads and Adult Participants Reporting no Preference or Preference for the New Genotropin Mark VII Injection Pen Compared to the Genotropin Pen®
Participants were asked the following question from Section II of the IPAQ PRO tool, Thinking about both injection pens over the past few months, please choose which injection pen you prefer overall. Choices included: prefer Genotropin Pen®, prefer new injection pen, or no preference.
Time frame: Month 4
Population: FAS
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Entire Study Population | Percentage of Dyads and Adult Participants Reporting no Preference or Preference for the New Genotropin Mark VII Injection Pen Compared to the Genotropin Pen® | 64.17 percentage of dyads, adult participants |
Secondary
Percentage of Dyads and Adult Participants Reporting the New Genotropin Mark VII Injection Pen Easier to Use Compared to the Genotropin Pen®
Participants were asked the following question from Section II of the IPAQ PRO tool, Thinking about the Genotropin pen and the new injection pen you used over the past few months, please compare both injection pens and choose which one is easier to use overall? Choices included: Genotropin Pen® easier to use, new injection pen easier to use, or no difference.
Time frame: Month 4
Population: FAS; Number of participants analyzed (N)= participants with evaluable data
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Entire Study Population | Percentage of Dyads and Adult Participants Reporting the New Genotropin Mark VII Injection Pen Easier to Use Compared to the Genotropin Pen® | 51.26 percentage of dyads, adult participants |
Secondary
Percentage of Dyads and Adult Participants Reporting the New Genotropin Mark VII Injection Pen Preferable Compared to the Genotropin Pen®
Participants were asked the following question from Section II of the IPAQ PRO tool, Thinking about both injection pens over the past few months, please choose which injection pen you prefer overall. Choices included: prefer Genotropin Pen®, prefer new injection pen, or no preference.
Time frame: Month 4
Population: FAS
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Entire Study Population | Percentage of Dyads and Adult Participants Reporting the New Genotropin Mark VII Injection Pen Preferable Compared to the Genotropin Pen® | 54.17 percentage of dyads, adult participants |
Secondary
Percentage of Dyads and Adult Participants Who Would Choose the New Genotropin Mark VII Injection Pen in Preference to the Genotropin® Pen
Investigators were asked the following study treatment continuation question, Which device did the participant choose for continued treatment? Choices included the Genotropin® Pen or the new injection pen.
Time frame: Month 4
Population: FAS subset of participants located in areas where the new device was available.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Entire Study Population | Percentage of Dyads and Adult Participants Who Would Choose the New Genotropin Mark VII Injection Pen in Preference to the Genotropin® Pen | 47.27 percentage of dyads, adult participants |
Comparison: Null hypothesis: percentage of participants preferring Genotropin Mark VII injection pen = 50%p-value: 0.6858binomial test