To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ in South Korea

Merck KGaA, Darmstadt, Germany224 enrolled

Overview

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 hospital in Korea in order to assess the level of adherence of subjects receiving SAIZEN® via easypod™.

Subjects will be enrolled in a multicenter, longitudinal, observational registry. Parents/subjects will provide their Informed Consent/assent to upload their data for population-based analyses and optionally to adhere to a patient adherence support program designed to improve their adherence 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 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 (IGF-1) (i.e. above, below or within normal ranges) * Temporal profile of IGF-1 and IGFBP-3 levels

Study Type

OBSERVATIONAL

Enrollment

224

Conditions

Growth Disorders

Eligibility

Sex: ALLMin age: 2 YearsMax age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Administered growth hormone via the easypod™ electromechanical device according to Summary of Product Characteristics (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 registry, 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 (i.e. for taking growth hormone for it's metabolic effects) * Contra-indications to SAIZEN® as defined in the Summary of Product Characteristics (SmPC) * Use of an investigational drug or participation in an interventional clinical study

Locations (1)

Yonsei University Colleage of Medicine, Severance Hospital

Seoul, South Korea

Outcomes

Primary Outcomes

Mean percent of adherence by subject over a period of time

Time frame: At least 6 months and up to 5 years

Secondary Outcomes

Correlation of adherence and growth outcome (height velocity (HV), height velocity-standard deviation score (HV-SDS), height, height standard deviation score) after each year of SAIZEN® treatment with easypod™

Time frame: At least 6 months and up to 5 years

Subject adherence profile based on age, gender, indication, self-injection or not, time on treatment

Time frame: At least 6 months and up to 5 years

Impact of patient adherence support programme on adherence and outcomes for subjects using easypod™

Time frame: At least 6 months and up to 5 years

Data from ClinicalTrials.gov

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