To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ in the UK (ECOS UK)

Merck KGaA (EMD Serono)

Status: Completed

Conditions: Growth Disorders

Treatments: Device: Easypod

Study type: Observational

Funder types: Industry

Identifiers

NCT01263457

EMR 200104-516

Details and patient eligibility

About

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 the UK and to assess the level of adherence of subject receiving SAIZEN® via Easypod™.

Full description

Subjects will be enrolled in a multicenter longitudinal observational registry. Parents/subjects will provide their Informed Consent/assent to upload their data for populationbased 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)
• To assess the endocrinological profile including Triiodothyronine (T3), Thyroxine (T4), Thyroid-stimulating hormone (TSH), Insulin-like Growth Factor 1 (IGF-1) and Insulin-like Growth Factor-Binding Protein 3(IGFBP-3) levels
• Temporal profile of IGF-1 and IGFBP-3 levels

Enrollment: 191 patients

Sex: All

Ages: 2 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Administered growth hormone via the easypod electromechanical injection 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 (to be confirmed by the Investigator, where relevant)
• Appropriate Informed Consent/Assent provided

Exclusion criteria

• Patients 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 SmPC or any other condition which precludes the use of SAIZEN in a given patient
• Use of an investigational drug or participation in an interventional clinical trial