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

Sponsor

Merck KGaA, Darmstadt, Germany (Industry)

Overall Status

Completed

CT.gov ID

NCT01291394

Collaborator

Merck Serono S.A.S, France (Industry)

223

Enrollment

Location

Actual Duration (Months)

3.8

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 hospitals in France and to assess the level of adherence of subjects receiving SAIZEN® via Easypod™.

Condition or Disease	Intervention/Treatment	Phase
Growth Disorders	Device: 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

Study Design

Study Type:

Observational

Actual Enrollment :

223 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 :

Jan 31, 2011

Actual Primary Completion Date :

Dec 31, 2015

Actual Study Completion Date :

Dec 31, 2015

Outcome Measures

Primary Outcome Measures

Mean percent of recorded adherence [At least 6 months and up to 5 years]

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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