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

Sponsor

Merck KGaA, Darmstadt, Germany (Industry)

Overall Status

Completed

CT.gov ID

NCT01263457

Collaborator

Merck Serono Limited, UK (Industry)

191

Enrollment

Locations

Actual Duration (Months)

31.8

Patients Per Site

0.5

Patients Per Site Per Month

Study Details

Study Description

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

Condition or Disease	Intervention/Treatment	Phase
Growth Disorders	Device: Easypod

Detailed 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

Study Design

Study Type:

Observational

Actual Enrollment :

191 participants

Observational Model:

Cohort

Time Perspective:

Other

Official Title:

Easypod Connect: 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

Actual Study Start Date :

Nov 30, 2010

Actual Primary Completion Date :

Dec 31, 2015

Actual Study Completion Date :

Dec 31, 2015

Outcome Measures

Primary Outcome Measures

Mean percent of adherence by subject over a period of time [At least 6 months and up to 5 years]

Secondary Outcome Measures

Correlation of adherence and growth outcome (height velocity (HV), height velocity-standard deviation score (HV-SDS), height, change in height (height SDS) after each year of SAIZEN® treatment with easypod™ [At least 6 months and up to 5 years]
Subject adherence profile based on age, gender, indication, self-injection or not, time on treatment [At least 6 months and up to 5 years]
Impact of patient adherence support programme on adherence and outcomes for subjects using easypod™ [At least 6 months and up to 5 years]

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years to 18 Years

Sexes Eligible for Study:

All

Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Research Site	Sutton	Surrey	United Kingdom	SM2 5PT
2	Research Site	Birmingham	West Midlands	United Kingdom	B4 6NH
3	Research Site	Glasgow		United Kingdom	G3 8SJ
4	Research Site	Hull		United Kingdom	HU3 2JZ
5	Research Site	London		United Kingdom	WC1N 1EH
6	Research Site	Sheffield		United Kingdom	S10 2TH