Growth Disorders
Conditions
Keywords
Growth disorders, Saizen, Easypod, Growth hormone, Pediatric subject
Brief summary
This is a National, Multicentre, Observational Registry to study adherence and long term outcomes of therapy in paediatric subjects using Easypod™ electromechanical device for growth hormone treatment from hospitals in France and to assess the level of adherence of subjects receiving SAIZEN® via Easypod™.
Detailed description
Subjects will be enrolled in this observational, multi-center study to assess adherence and treatment outcomes. Parents will provide their written agreement to upload their child's data in the study electronic CRF. Easypod™ is an electromechanical device that delivers growth hormone and also records injections' date and time. Adherence data will be primarily derived from injections recorded in the Easypod™ device combined with physician data entry of outcome measures (i.e. height & weight). This will allow the establishment of adherence profiles and evaluate the link with subsequent clinical outcomes. Since this is an observational study, there will be no study-specific clinical interventions and subjects will be treated according to the clinical and laboratory findings as routinely evaluated by the physician. Auxological and laboratory data will be reported prospectively throughout the duration of the study. At baseline, available data can be recorded retrospectively from the subjects' medical file and Easypod™ device Data will be collected retrospectively and prospectively. This will allow the establishment of adherence profiles 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 asses the Impact of adherence on insulin-like growth factor-1 (IGF1) ranges/levels
Interventions
DEVICEEasypod™
Saizen (Somatotropin) as per Summary of Product Characteristics administered by Easypod™
Sponsors
Merck Serono S.A.S, France
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 Saizen® via the Easypod™ electromechanical device according to pediatric registered indications (Growth Hormone Deficiency, Small for Gestational Age, Turner-Syndrome or prepubertal children with Chronic Renal Failure). * Naïve subjects or already treated with Saizen and Easypod for maximum 1 year * Male and Female between 2 - 18 years of age, or over 18 without fusion of growth plates * Parent's or guardian's (or subject if over 18 without fusion of growth plates) written agreement, given before entering data into the study, with the understanding that the subject or parent/guardian may withdraw agreement at any time without prejudice to future medical care.
Exclusion criteria
* Subjects taking Saizen® in whom growth plates have fused (i.e. taking growth hormone for it's metabolic effects) * Contra-indications to Saizen® as defined in the French Summary of Product Characteristics (SmPC) * Use of an investigational drug or participation in an interventional clinical study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Mean percent of recorded adherence | At least 6 months and up to 5 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Correlation of adherence and growth outcome (change in height velocity (HV), change in HV-Standard Deviation Score (HV-SDS), change in height-Standard Deviation Score) after each year of Saizen® treatment with Easypod™ | At least 6 months and up to 5 years | — |
| Subject adherence profile | At least 6 months and up to 5 years | Subject adherence profile based on age, gender, indication, self-injection or not, time on treatment, administration regimen (6 or 7 days/ week), combination of pituitary hormone deficiencies, previous and/or concomitant treatments, socio-economic data |
| Correlation of adherence with IGF-1 levels/ranges | At least 6 months and up to 5 years | — |
Countries
France
Outcome results
None listed