Saizen® E-Device User Trial

Phase 3

Completed

• Conditions: Growth Disorders
• Interventions: Device: Saizen® E-Device

• Registration Number: NCT00450190
• Lead Sponsor: Merck KGaA, Darmstadt, Germany

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2006-02-28
• Primary Completion: 2006-09-30
• Study Completion: 2006-09-30
• Study First Submitted: 2007-03-21
• Study First Submitted QC: 2007-03-21
• Study First Posted: 2007-03-22
• Status Verified: 2017-09
• Results First Submitted: 2017-09-29
• Results First Submitted QC: 2017-09-29
• Last Update Submitted: 2017-09-29
• Results First Posted: 2018-07-23
• Last Update Posted: 2018-07-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Saizen® E-Device	Saizen® E-Device	-

Primary Outcome Measures

Name	Time	Method
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.
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.

Secondary Outcome Measures

Name	Time	Method
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.
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.