Clinical trial NCT01267526: A Canadian, Multi-centre, Observational Registry to Study Adherence and Long Term Outcomes of Therapy in Pediatric Subjects Using SAIZEN® Via Easypod™ Auto-injector for Growth Hormone Treatment
Official title: "Easypod™ Connect Registry": A Canadian, Multi-centre, Observational Registry to Study Adherence and Long Term Outcomes of SAIZEN® in Pediatric Subjects Using Easypod™ Auto-injector
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: EMD Serono, a division of EMD Inc., Canada
First posted: 2010-12-28
Enrollment: 205
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Pediatric subjects 18 and under who are prescribed SAIZEN® (Health Canada approved indications) using the easypod™ auto-injector.

Brief summary: This is a Canadian, 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 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 prescribed SAIZEN® via easypod™

Secondary Objectives:

* To describe the impact of adherence on clinical outcomes for subject prescribed SAIZEN via easypod™
* To identify adherence subject profiling

Eligibility criteria: Inclusion Criteria:

* Prescribed SAIZEN® via the easypod™ auto-injector (SAIZEN® Health Canadian approved indications: Growth Hormone Insufficiency or Deficiency, Turner's Syndrome, Chronic Renal Failure, Small for Gestational Age
* Under 18 years of age, or over 18 without fusion of growth plates
* Parent's or legal guardian's written informed consent, given before entering data into the registry, with the understanding that the subject or parent/legal 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 by Health Canada regulations. Adult consent is defined at 18+ in the province of Quebec, 16+ in the remainder of Canada.

Exclusion Criteria:

* Subjects taking growth hormone in whom growth plates have fused (i.e. for taking growth hormone for its metabolic effects)
* Contra-indications to SAIZEN® as defined in SAIZEN® Canadian Product Monograph.
* Use of an investigational drug or participation in another interventional clinical study

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 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: Calgary
country: Canada
facility: Merck Serono Research Site
state: Alberta
city: Edmonton
country: Canada
facility: Merck Serono Research Site
state: Alberta
city: North Vancouver
country: Canada
facility: Merck Serono Research Site
state: British Columbia
city: Vancouver
country: Canada
facility: Merck Serono Research Site
state: British Columbia
city: Hamilton
country: Canada
facility: Merck Serono Research Site
state: Ontario
city: London
country: Canada
facility: Merck Serono Research Site
state: Ontario
city: Toronto
country: Canada
facility: Merck Serono Research Site
state: Ontario
city: Montréal
country: Canada
facility: Merck Serono Research Site
state: Quebec
city: Sherbrooke
country: Canada
facility: Merck Serono Research Site
state: Quebec
Study officials:
affiliation: EMD Serono, a division of EMD Inc., Canada
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.