Clinical trial NCT01263457: To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ in the UK
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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Growth Disorders
Keywords: Growth disorders
Saizen
EasypodTM
Growth hormone
Pediatric subject
Lead sponsor: Merck KGaA, Darmstadt, Germany
Sponsor class: INDUSTRY
Collaborators: name: Merck Serono Limited, UK
First posted: 2010-12-20
Enrollment: 191
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Paediatric subjects (aged 2-18) who are taking SAIZEN® for registered indications using the easypod™ electromechanical device.

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™.

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

Eligibility criteria: 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

Primary outcomes:
measure: Mean percent of adherence by subject over a period of time
timeFrame: At least 6 months and up to 5 years
Secondary outcomes:
measure: 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™
timeFrame: At least 6 months and up to 5 years
measure: Subject adherence profile based on age, gender, indication, self-injection or not, time on treatment
timeFrame: At least 6 months and up to 5 years
measure: Impact of patient adherence support programme on adherence and outcomes for subjects using easypod™
timeFrame: At least 6 months and up to 5 years
Other outcomes:
Locations:
city: Sutton
country: United Kingdom
facility: Research Site
state: Surrey
city: Birmingham
country: United Kingdom
facility: Research Site
state: West Midlands
city: Glasgow
country: United Kingdom
facility: Research Site
city: Hull
country: United Kingdom
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
city: Sheffield
country: United Kingdom
facility: Research Site
Study officials:
affiliation: Merck Serono Limited, UK
name: Medical Responsible
role: STUDY_DIRECTOR
References:
citation: Koledova, E. et al. (2017) Analysis of results from the global, 5-year Easypod™ Connect Observational Study (ECOS) study in children with growth disorders. 10th International Meeting for Pediatric Endocrinology; 2017 Sep 14-17; Washington, DC.
citation: Wit, JM. et al. (2017) Effect of adherence on the 2-year growth response to growth hormone treatment in prepubertal children with idiopathic isolated growth hormone deficiency participating in the EasypodTM Connect Observational Study (ECOS). 10th International Meeting of Paediatric Endocrinology; 2017 Sep 14-17; Washington, DC.