To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ (ECOS Taiwan, Indonesia, Singapore)

Completed

• Conditions: Growth Disorders
• Interventions: Device: easypod™

• Registration Number: NCT02015273
• Lead Sponsor: Merck KGaA, Darmstadt, Germany

Brief Summary

This is an Multinational, Multicenter, Observational Study to evaluate Adherence and Long Term Outcomes of Therapy in pediatric subjects using easypod™ electromechanical device for growth hormone treatment and to assess the level of adherence of subject receiving SAIZEN® via easypod™.

Detailed Description

Subjects will be enrolled in a multicenter longitudinal observational study. Parents/subjects will provide their Informed Consent/assent to upload their data for population-based analyses 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 both 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 (IGF1) (that is, above, below or within normal ranges)

Study Timeline

• Study First Submitted: 2013-12-18
• Study First Submitted QC: 2013-12-18
• Study First Posted: 2013-12-19
• Study Start: 2014-02-28
• Primary Completion: 2015-12-31
• Study Completion: 2015-12-31
• Status Verified: 2020-07
• Last Update Submitted: 2020-07-03
• Last Update Posted: 2020-07-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 82

Inclusion Criteria

• Administered growth hormone via the easypod™ electromechanical device according to Summary of Product Characteristic (SmPC)
• Over the age of 2 years
• Under 18 years of age, or over 18 without fusion of growth plates
• Parent's or guardian's written informed consent, given before entering data into the observational study, 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

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Exclusion Criteria

• Subjects taking growth hormone in whom growth plates have fused (that is, 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 another interventional clinical study

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Growth Disorders	easypod™	-

Primary Outcome Measures

Name	Time	Method
Mean percent of daily recorded adherence	At least 6 months and up to 2.5 years

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 2.5 years	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™
Subject adherence profile based on age, gender, indication, self-injection or not, time on treatment	At least 6 months and up to 2.5 years
Correlation of adherence with current IGF-1 status (that is, above, below or within normal ranges)	At least 6 months and up to 2.5 years

Trial Locations

Locations (6)

KK Women's & Children's Hospital; 🇸🇬 Singapore, Singapore

National University Hospital; 🇸🇬 Singapore, Singapore

Anakku Clinic Pondok Pinang; 🇮🇩 Jakarta, Indonesia

Taipei Medical University Hospital; 🇨🇳 Taipei, Taiwan

Kaohsiung Medical University Hospital; 🇨🇳 Kaohsiung, Taiwan

Chung Shan Medical University Hospital; 🇨🇳 Taichung, Taiwan