Growth Disorders
Conditions
Keywords
easypod™, SAIZEN®
Brief summary
This is an Multinational, Multicenter, Observational Study to evaluate Adherence and Long Term Outcomes of Therapy in pediatric subjects using easypod™ electromechanical device for growth hormone treatment and to assess the level of adherence of subject receiving SAIZEN® via easypod™.
Detailed description
Subjects will be enrolled in a multicenter longitudinal observational study. Parents/subjects will provide their Informed Consent/assent to upload their data for population-based analyses and, ultimately, clinical outcomes. Adherence data will be primarily derived from the easypod™ device combined with physician data entry of outcome measures. Data will be collected both retrospectively and prospectively. This will allow the establishment of adherence profiles and explore the hypothesis that patient adherence support programs improve adherence and subsequent clinical outcomes. Collected data will be also analyzed in a multinational pooled analysis of comparable national studies. Primary Objective: • To assess the level of adherence of subjects receiving SAIZEN® via easypod™ Secondary Objectives: * To describe the impact of adherence on clinical outcomes for subject receiving SAIZEN® via easypod™ * To identify adherence subject profiling * To assess the impact of adherence on Insulin-like Growth Factor 1 (IGF1) (that is, above, below or within normal ranges)
Interventions
DEVICEeasypod™
Saizen® (Somatropin) as per Summary of Product Characteristics administered by easypod™
Sponsors
Merck Ltd., Taiwan
PT Merck Tbk., Indonesia, an affiliate of Merck KGaA, Darmstadt, Germany
Merck Pte. Ltd., Singapore
Merck KGaA, Darmstadt, Germany
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
2 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Administered growth hormone via the easypod™ electromechanical device according to Summary of Product Characteristic (SmPC) * Over the age of 2 years * Under 18 years of age, or over 18 without fusion of growth plates * Parent's or guardian's written informed consent, given before entering data into the observational study, with the understanding that the subject or parent/guardian may withdraw consent at any time without prejudice to future medical care. If the child is old enough to read and write, a separate assent form will be given as defined in the appropriate jurisdiction of each country
Exclusion criteria
* Subjects taking growth hormone in whom growth plates have fused (that is, for taking growth hormone for it's metabolic effects) * Contra-indications to SAIZEN® as defined in the SmPC * Use of an investigational drug or participation in another interventional clinical study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Mean percent of daily recorded adherence | At least 6 months and up to 2.5 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Correlation of adherence and growth outcome after each year of SAIZEN® treatment with easypod™ | At least 6 months and up to 2.5 years | Correlation of adherence and growth outcome (change in: height velocity \[HV\], height velocity-standard deviation score \[HV-SDS\], height, height-standard deviation score\[height SDS\]) after each year of SAIZEN® treatment with easypod™ |
| Subject adherence profile based on age, gender, indication, self-injection or not, time on treatment | At least 6 months and up to 2.5 years | — |
| Correlation of adherence with current IGF-1 status (that is, above, below or within normal ranges) | At least 6 months and up to 2.5 years | — |
Countries
Indonesia, Singapore, Taiwan
Outcome results
None listed