UMIN-CTR Clinical Trial

Basic information
Public title	Effectiveness of smartphone-based mobile application on medication adherence in patients receiving growth hormone therapy-An exploratory observational study. (GTL-App)
Acronym	Effectiveness of smartphone-based mobile application on medication adherence in patients receiving growth hormone therapy-An exploratory observational study. (GTL-App)
Scientific Title	Effectiveness of smartphone-based mobile application on medication adherence in patients receiving growth hormone therapy-An exploratory observational study. (GTL-App)
Scientific Title:Acronym	Effectiveness of smartphone-based mobile application on medication adherence in patients receiving growth hormone therapy-An exploratory observational study. (GTL-App)
Region	Japan

Condition
Condition	Short statue caused by -Growth hormone (GH) deficiency -Turner syndrome -Small for gestational age
Classification by specialty	Endocrinology and Metabolism Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	-To prospectively investigate medication adherence of pediatric patients who initiate GH therapy using a smartphone-based mobile application. -To explore factors associated with low adherence to GH therapy.
Basic objectives2	Others
Basic objectives -Others	Effectiveness of a mobile application not classified as a medical device
Trial characteristics_1	Exploratory
Trial characteristics_2	Pragmatic
Developmental phase	Not applicable

Assessment
Primary outcomes	Medication adherence of patients during 24 weeks of observation period.
Key secondary outcomes	-Patients characteristics at registration. -Time course change in medication adherence. -Nonadherence-free survival. -Questionnaire on GH therapy. -Questionnaire on mobile application. -Factors affecting adherence to GH therapy. -Exploratory analysis of application log data.

Base
Study type	Observational

Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	3 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	-Never received growth hormone therapy before. -Aged 3 years or older. -Planning to: -Use GROWJECTOR-L. -Receive seven-days-a-week administration. -Use type A needles. -Written informed consent from a legally acceptable representative of the patient, along with informed assent if applicable.
Key exclusion criteria	-Contraindications for GH therapy. eg, Diabetes, malignancy, (possible) pregnancy. -Not supposed to be able to answer the questionnaire properly, eg, unable to read. -Judgement of physicians.
Target sample size	50

Research contact person
Name of lead principal investigator	1st name Tatsuhiko Middle name Last name Urakami
Organization	Nihon University Hospital
Division name	Department of Pediatrics
Zip code	101-8309
Address	1-6, Kanda-Surugadai, Chiyoda-ku, Tokyo 101-0062, Japan
TEL	81-3-3293-1711
Email	urakami.tatsuhiko@nihon-u.ac.jp

Public contact
Name of contact person	1st name Tatsuhiko Middle name Last name Urakami
Organization	Nihon University Hospital
Division name	Department of Pediatrics
Zip code	101-8309
Address	1-6, Kanda-Surugadai, Chiyoda-ku, Tokyo 101-0062, Japan
TEL	81-3-3293-1711
Homepage URL
Email	urakami.tatsuhiko@nihon-u.ac.jp

Sponsor
Institute	Department of Pediatrics, Nihon University Hospital
Institute
Department

Funding Source
Organization	JCR pharmaceuticals Co., Ltd.
Organization
Division
Category of Funding Organization	Profit organization
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization	Ethics committee of Nihon university hospital
Address	1-6, Kanda-Surugadai, Chiyoda-ku, Tokyo 101-0062, Japan
Tel	81-3-3293-1711
Email	suzuki.ikumi@nihon-u.ac.jp

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions	日本大学病院をはじめとする全国約40施設を予定

Other administrative information
Date of disclosure of the study information	2019 Year 03 Month 01 Day

Related information
URL releasing protocol
Publication of results	Published

Result
URL related to results and publications	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8022037/
Number of participants that the trial has enrolled	60
Results	This study enrolled 60 children with short stature from 28 Japanese medical institutions and analyzed 57 of them. The median and mean adherence rates after 24 wk of observation were 96% and 93%, respectively. Although adherence rates were significantly lower from wk 16 to wk 20 than from wk 1 to wk 4, cumulative adherence rates remained high throughout the observation period. The questionnaire analysis revealed that most patients actively used the application.
Results date posted	2021 Year 04 Month 25 Day
Results Delayed
Results Delay Reason
Date of the first journal publication of results	2021 Year 04 Month 03 Day
Baseline Characteristics	The mean age (SD) of the patients was 7.2 (3.84) yr, and the proportions of male and female patients was almost similar. A total of 54.4%, 7.0%, and 38.6% of the included patients were diagnosed with GHD, TS, and SGA, respectively. A relatively high proportion of patients were diagnosed with SGA. Among all patients, patients with SGA started GH therapy at the lowest age (P = 0.002). The baseline mean (SD) height SD score was -2.71 (0.57).
Participant flow	This study enrolled 60 patients with short stature from 28 Japanese medical institutions. Three patients discontinued shortly after GH therapy initiation due to medication change, unbearable pain, and failure to return to the hospital and were subsequently excluded from the statistical analysis.
Adverse events	N/A
Outcome measures	Good adherence rates to GH therapy were observed with the use of the treatment-supporting application. Analyses of questionnaire answers revealed that most patients actively used the application.
Plan to share IPD	N/A
IPD sharing Plan description	N/A

Progress
Recruitment status	Completed
Date of protocol fixation	2019 Year 01 Month 15 Day
Date of IRB	2019 Year 01 Month 10 Day
Anticipated trial start date	2019 Year 03 Month 01 Day
Last follow-up date	2020 Year 05 Month 31 Day
Date of closure to data entry	2020 Year 07 Month 31 Day
Date trial data considered complete	2020 Year 09 Month 30 Day
Date analysis concluded	2020 Year 12 Month 31 Day

Other
Other related information	-To prospectively investigate medication adherence of pediatric patients who initiate GH therapy using a smartphone-based mobile application.

Management information
Registered date	2019 Year 02 Month 25 Day
Last modified on	2021 Year 04 Month 26 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000040956