Clinical trial NCT01307059: To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ in Sweden
Official title: Easypod Connect: A Swedish, 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
Easypod
Growth hormone
Pediatric subject
Lead sponsor: Merck KGaA, Darmstadt, Germany
Sponsor class: INDUSTRY
Collaborators: name: Merck A.B., Sweden
First posted: 2011-03-02
Enrollment: 152
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 Swedish, Multicentre, Observational Registry to study Adherence and Long Term Outcomes of Therapy in paediatric subjects using Easypod™ electromechanical device for growth hormone treatment 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 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 based on age, gender, self injection or not, time on treatment, medical history at time of enrollment
* To assess the impact of adherence on Insulin-like Growth Factor 1 (IGF1) (i.e. above, below or within normal ranges)

Other Objectives:

• To assess the impact of adherence on average blood glucose (HbA1C)

Eligibility criteria: Inclusion Criteria:

* Administered growth hormone via the EasypodTM electromechanical device according to Summary of Product Characteristics (SmPC)
* Over the age of \> 2 years old
* Under \< 18 years of age, or over 18 years of age 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 SmPC
* Use of an investigational drug or participation in an interventional clinical study

Primary outcomes:
measure: Mean percent of daily recorded adherence
timeFrame: At least 6 months and up to 5 years
Secondary outcomes:
measure: Correlation of adherence and growth outcome (change in height velocity (HV), height velocity-standard deviation score (HV-SDS), height, height-standard deviation score(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: Correlation of adherence with current IGF-1 status (i.e. above, below or within normal ranges)
timeFrame: At least 6 months and up to 5 years
measure: Correlation of adherence and HbA1C
timeFrame: At least 6 months and up to 5 years
Other outcomes:
Locations:
city: Göteborg
country: Sweden
facility: Merck Serono Research Site
city: Solna
country: Sweden
facility: Merck Serono Research Site
city: Uppsala
country: Sweden
facility: Merck Serono Research Site
Study officials:
affiliation: Merck A.B., Sweden
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.