E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Idiopathic Short Stature (ISS) |
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E.1.1.1 | Medical condition in easily understood language |
Growth Hormone Deficiency |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the ease of use/convenience as determined by a subject/caregiver dyad self-assessment of ease of use of the new Genotropin Mark VII pen compared to pre-study experience with the Genotropin Pen. |
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E.2.2 | Secondary objectives of the trial |
To evaluate the subject/caregiver dyad preference for the new Genotropin Mark VII pen compared to pre-study experience with the Genotropin Pen. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Age greater than or equal to (>=8) years and less than or equal to (<=) 18 years.
2. Subjects who are currently on treatment with Genotropin Pen >=3 months.
3. Subjects who are compliant with current Genotropin Pen treatment.
4. Written informed consent of the adult member of the subject dyad or legally authorized representative (that is (i.e.), Parent/Legal Guardian) indicating that the subject and the legally acceptable representative have been informed of all pertinent aspects of the study. In addition if possible, assent should be obtained from the child to take part in the study. The adult member of the dyad will be consenting for his/her participation.
5. The adult member of the dyad needs to be able to read English and both members of the dyad need be able to understand English.
6. Subject dyads who are willing and able to comply with scheduled visits, treatment plan, and other study procedures.
7. Female Genotropin users of child bearing potential must have a negative urine pregnancy test at screening.
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E.4 | Principal exclusion criteria |
1. Subjects with Prader-Willi syndrome or chronic renal insufficiency.
2. Subjects and caregivers who have been, in the opinion of the investigator,
3. non-compliant with Genotropin treatment or are not able to adequately participate in the study.
4. Subjects with chronic systemic disorders, such as diabetes and heart disease.
5. Subjects who are not able to understand written and/or verbal instructions on the proper use of grown hormone injection devices.
6. Subjects who are not able to able or willing to complete the questionnaires used for the study assessments.
7. Subjects using other growth hormone injection devices other than the current
8. Genotropin Pen.
9. Subjects who previously participated in any of the Genotropin Mark VII or Injection Pen Assessment Questionnaire (IPAQ) development research and studies.
10. Subjects who are participating in any interventional clinical studies.
11. Subjects who are taking concomitant investigational drugs/treatment.
12. Subjects who, in the opinion of the investigator, are not likely to comply with the study requirements for any reason.
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E.5 End points |
E.5.1 | Primary end point(s) |
Percentage of Dyads (Subjects 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® |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1) 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) Percentage of Dyads Reporting New Genotropin Mark VII Injection Pen Easier to Use Compared to Pre-study Experience With the Genotropin Pen®
3) Percentage of Dyads Reporting New Genotropin Mark VII Injection Pen Preferable Compared to Pre-study Experience With the Genotropin Pen® |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1) 2 months
2) 2 months
3)2 months
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Patient Reported Outcomes |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Historic control of prior injection device |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 4 |