Growth Disorders
Conditions
Keywords
Growth disorders
Brief summary
The aim of the study is to evaluate the E-Device performances and handling on the use in common practice, by collecting the impressions of patients, nurses and the investigator on the graphic interface, the instructions manual, the E-Device training and the material itself.
Interventions
DEVICESaizen® E-Device
Saizen® (recombinant human growth hormone \[r-hGH\]) injection 8 milligram (mg) per 137 milliliter (mL) will be administered using an electronic auto-injector (E-Device) subcutaneously (SC) in the evening as per local Summary of Product Characteristics (SmPC) for 60 days.
Sponsors
Merck KGaA, Darmstadt, Germany
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Inclusion criteria
1. Patients naïve to, or experienced with, Saizen® with growth disorders in registered indications (GHD, Turner's Syndrome, Chronic Renal Failure, patient born Small Gestational Age \[SGA\] according to the local SmPC) 2. Written informed consent must be obtained from the parent(s)/legal guardian(s) at the beginning of the study. Children able to understand the trial should personally sign and date the written informed consent
Exclusion criteria
1. Known hypersensitivity to somatropin or any of the excipients 2. Epiphyseal fusion 3. Active neoplasia (either newly diagnosed or recurrent) 4. History of intracranial hypertension with papilledema 5. Diabetes mellitus or history of significant glucose intolerance as defined by a fasting blood glucose \> 116 mg/dL 6. Severe congenital malformations 7. Severe psychomotor retardation 8. Known hepatic disease as defined by elevated liver enzymes or total bilirubin (x 2 N) 9. Current congestive heart failure, untreated hypertension, serious chronic oedema of any cause 10. Chronic infectious disease 11. Previous or ongoing treatment with sex steroid therapy such as estrogens and testosterone 12. Previous or ongoing treatment with any therapy that may directly influence growth, including GH, GHRF and long duration corticosteroids therapy 13. Proliferative or preproliferative diabetic retinopathy 14. Evidence of any progression or recurrence of an underlying intra-cranial space occupying lesion 15. Precocious puberty 16. Severe associated pathology affecting growth such as malnutrition, malabsorption or bone dysplasia 17. Concomitant corticoid treatment or levothyroxine treatment other than substitutive treatment, topical or inhaled treatment 18. Participation to any clinical study within the 30 days preceding study entry 19. Pregnancy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Subjects' Overall Impression After Using E-Device | 2 Weeks | Subjects' overall impression after using E-Device was measured on a scale ranging from 1 to 3, where 1 = bad, 2 = good, and 3 = very good. Number of subjects with response based on overall impression scale were presented. |
| Usefulness and Reliability of E-Device Functions | 2 Weeks | Following functions were assessed: Display of remaining dose in cartridge, Display of last injection date and time, Automatic needle attachment, Audible and visual signals, Dose injected confirmation, Dose history, Customizable needle insertion speed, Customizable drug insertion speed, Customizable insertion depth, Teach me menu, On screen instructions, Customizable name and picture, Pre-programmed dose and Skin sensor. Usefulness and reliability of each of the E-Device functions was measured on a scale ranging from 1 to 3, where 1 = not useful, 2 = useful, and 3 = very useful. Number of subjects with response based on usefulness and reliability scale were presented. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Inclusion visit (Day 1) | Subjects' feedback immediately after initial training on the handling and use of E-Device was assessed on scale ranging from 1 to 3, where 1 = difficult, 2 = easy, and 3 = very easy. Subjects were provided training on the following aspects: Cartridge loading, Needle attachment, Needle detachment, Injection process, Navigation in the menu, and Handling of the device. Number of subjects with response based on their feedback were presented. |
| Nurse/Physician's Feedback After E-Device Set up During Inclusion Visit | Inclusion visit (Day 1) | Nurse/physician's feedback was assessed for E-Device setting up and Dose programming on inclusion visit using a scale ranging from 1 to 3, where 1 = difficult, 2 = easy, and 3 = very easy. Nurse/Physician's response for the number of subjects were presented. |
| Number of Subjects With Adverse Events (AEs) or Serious Adverse Events (SAEs) | Day 1 up to Day 90 | An Adverse Event (AE) was defined as any new untoward medical occurrences/worsening of pre-existing medical condition without regard to possibility of causal relationship. A Serious AE was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect, AEs leading to discontinuation and AEs leading to death. |
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Saizen® E-Device Saizen® (recombinant human growth hormone \[r-hGH\]) injection 8 milligram (mg) per 137 milliliter (mL) was administered using an electronic auto-injector (E-Device) subcutaneously (SC) in the evening as per local Summary of Product Characteristics (SmPC) for 60 days. | 20 |
| Total | 20 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Withdrawal by Subject | 2 |
Baseline characteristics
| Characteristic | Saizen® E-Device |
|---|---|
| Age, Continuous | 11.30 years STANDARD_DEVIATION 2.66 |
| Sex: Female, Male Female | 10 Participants |
| Sex: Female, Male Male | 10 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 20 |
| serious Total, serious adverse events | 0 / 20 |
Outcome results
Primary
Subjects' Overall Impression After Using E-Device
Subjects' overall impression after using E-Device was measured on a scale ranging from 1 to 3, where 1 = bad, 2 = good, and 3 = very good. Number of subjects with response based on overall impression scale were presented.
Time frame: 2 Weeks
Population: All subjects who were administered with at least 1 dose of study medication with E-Device.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Saizen® E-Device | Subjects' Overall Impression After Using E-Device | Bad | 0 subjects |
| Saizen® E-Device | Subjects' Overall Impression After Using E-Device | Good | 7 subjects |
| Saizen® E-Device | Subjects' Overall Impression After Using E-Device | Very Good | 13 subjects |
Primary
Usefulness and Reliability of E-Device Functions
Following functions were assessed: Display of remaining dose in cartridge, Display of last injection date and time, Automatic needle attachment, Audible and visual signals, Dose injected confirmation, Dose history, Customizable needle insertion speed, Customizable drug insertion speed, Customizable insertion depth, Teach me menu, On screen instructions, Customizable name and picture, Pre-programmed dose and Skin sensor. Usefulness and reliability of each of the E-Device functions was measured on a scale ranging from 1 to 3, where 1 = not useful, 2 = useful, and 3 = very useful. Number of subjects with response based on usefulness and reliability scale were presented.
Time frame: 2 Weeks
Population: All subjects who were administered with at least 1 dose of study medication with E-Device.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Display of remaining dose in cartridge:Very Useful | 12 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Automatic needle attachment: Useful | 9 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Display of remaining dose in cartridge: Not Useful | 0 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Display of remaining dose in cartridge: Useful | 8 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Display of last injection date/time: Not Useful | 3 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Display of last injection date/time: Useful | 11 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Display of last injection date/time: Very Useful | 6 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Automatic needle attachment: Not Useful | 0 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Automatic needle attachment: Very Useful | 11 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Audible and visual signals: Not Useful | 1 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Audible and visual signals: Useful | 9 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Audible and visual signals: Very Useful | 10 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Dose injected confirmation: Not Useful | 2 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Dose injected confirmation: Useful | 10 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Dose injected confirmation: Very Useful | 8 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Customizable name and picture: Not Useful | 13 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Dose history: Not Useful | 5 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Dose history: Useful | 10 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Dose history: Very Useful | 5 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Customizable needle insertion speed: Not Useful | 2 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Customizable needle insertion speed: Useful | 9 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Customizable needle insertion speed: Very Useful | 9 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Customizable drug insertion speed: Not Useful | 1 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Customizable drug insertion speed: Useful | 11 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Customizable drug insertion speed: Very Useful | 8 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Customizable insertion depth: Not Useful | 1 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Customizable insertion depth: Useful | 9 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Customizable insertion depth: Very Useful | 10 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Teach me menu: Not Useful | 8 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Teach me menu: Useful | 7 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Teach me menu: Very Useful | 5 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | On screen instructions: Not Useful | 0 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | On screen instructions: Useful | 6 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | On screen instructions: Very Useful | 14 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Customizable name and picture: Useful | 7 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Customizable name and picture: Very Useful | 0 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Pre-programmed dose: Not Useful | 0 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Pre-programmed dose: Useful | 6 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Pre-programmed dose: Very Useful | 14 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Skin sensor: Not Useful | 2 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Skin sensor: Useful | 8 subjects |
| Saizen® E-Device | Usefulness and Reliability of E-Device Functions | Skin sensor: Very Useful | 10 subjects |
Secondary
Number of Subjects With Adverse Events (AEs) or Serious Adverse Events (SAEs)
An Adverse Event (AE) was defined as any new untoward medical occurrences/worsening of pre-existing medical condition without regard to possibility of causal relationship. A Serious AE was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect, AEs leading to discontinuation and AEs leading to death.
Time frame: Day 1 up to Day 90
Population: All subjects who were administered with at least 1 dose of study medication with E-Device.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Saizen® E-Device | Number of Subjects With Adverse Events (AEs) or Serious Adverse Events (SAEs) | AEs | 0 subjects |
| Saizen® E-Device | Number of Subjects With Adverse Events (AEs) or Serious Adverse Events (SAEs) | SAEs | 0 subjects |
Secondary
Nurse/Physician's Feedback After E-Device Set up During Inclusion Visit
Nurse/physician's feedback was assessed for E-Device setting up and Dose programming on inclusion visit using a scale ranging from 1 to 3, where 1 = difficult, 2 = easy, and 3 = very easy. Nurse/Physician's response for the number of subjects were presented.
Time frame: Inclusion visit (Day 1)
Population: All included subjects were analyzed who used at least 1 dose of study medication with E-Device.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Saizen® E-Device | Nurse/Physician's Feedback After E-Device Set up During Inclusion Visit | Device setting up: Difficult | 1 subjects |
| Saizen® E-Device | Nurse/Physician's Feedback After E-Device Set up During Inclusion Visit | Device setting up: Easy | 19 subjects |
| Saizen® E-Device | Nurse/Physician's Feedback After E-Device Set up During Inclusion Visit | Device setting up: Very Easy | 0 subjects |
| Saizen® E-Device | Nurse/Physician's Feedback After E-Device Set up During Inclusion Visit | Dose programming: Difficult | 0 subjects |
| Saizen® E-Device | Nurse/Physician's Feedback After E-Device Set up During Inclusion Visit | Dose programming: Easy | 19 subjects |
| Saizen® E-Device | Nurse/Physician's Feedback After E-Device Set up During Inclusion Visit | Dose programming: Very Easy | 0 subjects |
| Saizen® E-Device | Nurse/Physician's Feedback After E-Device Set up During Inclusion Visit | Dose programming: Missing data | 1 subjects |
Secondary
Subjects' Feedback Immediately After Initial Training During Inclusion Visit
Subjects' feedback immediately after initial training on the handling and use of E-Device was assessed on scale ranging from 1 to 3, where 1 = difficult, 2 = easy, and 3 = very easy. Subjects were provided training on the following aspects: Cartridge loading, Needle attachment, Needle detachment, Injection process, Navigation in the menu, and Handling of the device. Number of subjects with response based on their feedback were presented.
Time frame: Inclusion visit (Day 1)
Population: All subjects who were administered with at least 1 dose of study medication with E-Device.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Cartridge loading: Difficult | 1 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Cartridge loading: Easy | 17 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Cartridge loading: Very Easy | 2 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Needle attachment: Difficult | 2 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Needle attachment: Easy | 15 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Needle attachment: Very Easy | 3 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Needle detachment: Difficult | 7 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Needle detachment: Easy | 12 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Needle detachment: Very Easy | 1 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Injection process: Difficult | 1 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Injection process: Easy | 16 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Injection process: Very Easy | 1 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Injection process: Missing data | 2 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Navigation in the menu: Difficult | 2 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Navigation in the menu: Easy | 17 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Navigation in the menu: Very Easy | 1 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Handling of the device: Difficult | 1 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Handling of the device: Easy | 15 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Handling of the device: Very Easy | 0 subjects |
| Saizen® E-Device | Subjects' Feedback Immediately After Initial Training During Inclusion Visit | Handling of the device: Missing data | 4 subjects |