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
- Conditions
- Growth Disorders
- Registration Number
- NCT01267526
- Lead Sponsor
- Merck KGaA, Darmstadt, Germany
- 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
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 205
- 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.
- 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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Mean percent of adherence by subject over a period of time At least 6 months and up to 5 years Adherence will be defined as 86 percent adherent at one year based on prescribed therapy.
- Secondary Outcome Measures
Name Time Method Correlation of adherence and growth outcome after each year of SAIZEN® treatment with easypod™ At least 6 months and up to 5 years 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™
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
Trial Locations
- Locations (1)
Merck Serono Research Site
🇨🇦Sherbrooke, Quebec, Canada