Development of a Personalized Growth Hormone Treatment Profile (PGTP) Report for Pediatric Endocrinology (PGTP)

The goal of this observational study is to learn how to make growth hormone treatment more personalized for children. The main questions it aims to answer are:

• How can information about treatment adherence and patient experiences help improve care?
• What insights can help doctors create treatment plans that are more tailored to each child?

The study will include 40 families of children who are using a connected injector device for their growth hormone treatment. These devices track how often the treatment is taken, and families will also share their experiences through surveys. The information will be used to develop a new tool, similar to tools used in diabetes care, to help doctors create better, personalized treatment plans for children with growth hormone needs.

Study Overview

• Status: Completed
• Conditions: Growth Hormone Deficiency (GHD)
• Intervention / Treatment: Combination product: ACDP® and growth hormone treatment

• Study Type: Observational
• Enrollment (Actual): 40

Contacts and Locations

Study Locations

• Spain
  • Aragon
    • Zaragoza, Aragon, Spain
      • Hospital Universitario Miguel Servet

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

The sample of the study (children and their caregivers) will be recruited from the Pediatric Endocrinology unit at the Miguel Servet Children's University Hospital. This public hospital provides pediatric health attention to the regional area of Zaragoza (Spain), which covers a total of 367.110 inhabitants.

Families of children undergoing growth hormone therapy and are willing to join a combined intervention.

Description

Inclusion Criteria:

• Pediatric patients (up to 18 years old) diagnosed with growth hormone deficiency or disorders requiring growth hormone treatment.
• Patients are currently undergoing growth hormone therapy. Includes both naive patients (those newly starting weekly treatment) and those already on treatment.
• Families willing to use connected injector devices and participate in mobile-based psychometrics for the study duration.
• Families are able and willing to regularly provide patient-reported outcomes (PROs) and adherence data through the Adhera®️ Caring Digital Program.
• Participants willing to sign the informed consent form, confirming their understanding of the essential aspects of the study, including the potential risks, benefits and rights as participants.

Exclusion Criteria:

• Families/patients already enrolled in other study protocols, including the use of connected injector devices and participation in mobile-based psychometrics.
• Patients with other significant medical conditions that could interfere with growth hormone treatment or the study's data collection processes.
• Inability to understand or communicate in the language used for data collection and study participation, unless appropriate translation services are available and can be consistently utilized.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Families with children undergoing growth hormone treatment; The study targets families of children with growth hormone deficiency (GHD) under growth hormone treatment, using a connected injector device that quantifies treatment adherence. We will recruit 40 families, divided into two groups: one group with adherence rates of <85%, and the other group with adherence rates of >85%.	Combination product: ACDP® and growth hormone treatment; A cohort of 40 families of children undergoing growth hormone treatment using a connected injector devices that quantifies treatment adherence will be recruited via the ACDP®, to capture additional data that combine Patient Reported Outcomes and Adherence Data.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Develop a Personalized Growth Hormone Treatment Profile (PGTP)	To develop a Personalized Growth Hormone Treatment Profile (PGTP) standard - analogous to the Ambulatory Glucose Profile (AGP) for diabetes care-, integrating diverse data sources to support personalized treatment strategies in pediatric endocrinology.	From enrollment to the end of the study at Month 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adherence to Treatment Using Connected Injector Devices	Automated tracking of injection times and adherence. Adherence will be quantified through connected injector device data, capturing timestamps of each injection and calculating adherence rates.	From enrollment to the end of the study at Month 12
Physiological Monitoring via Wearable Devices - Sleep Quality	Unit of Measure: - Total sleep duration (hours per night). Description: Sleep parameters will be tracked using wearable devices.	From enrollment to the end of the study at Month 12
Physiological Monitoring via Wearable Devices - Heart Rate	Unit of Measure: Average daily heart rate (beats per minute, bpm). Description: Daily average heart rate recorded through wearable devices.	From enrollment to the end of the study at Month 12
Physiological Monitoring via Wearable Devices - Physical Activity Metrics	Unit of Measure: Total calories burned per day (kilocalories, kcal).; Total steps per day (steps).; Total activity time per day (minutes). Description: Data will be collected from wearable devices to assess daily physical activity.	From enrollment to the end of the study at Month 12
Physiological Monitoring via Wearable Devices - Blood Oxygen Levels	Unit of Measure: Daily oxygen saturation percentage (%). Description: Measured through wearable device sensors.	From enrollment to the end of the study at Month 12
Evaluating Usability of Connected Devices	Outcome Measure: System Usability Scale (SUS) score. Unit of Measure: SUS score (0-100, higher scores indicate better usability). Description: The System Usability Scale (Bangor et al., 2008) is a 10-item scale based on a 5-point Likert scale assessing perceived usability.	From enrollment to the end of the study at Month 12
Mobile Application Engagement	Outcome Measure: User engagement with the mobile application. Unit of Measure: Frequency of interactions (e.g., number of logins per day/week). Description: Engagement will be assessed using mobile user logs, including user profiling metrics based on Hors-Fraile et al. (2018).	From enrollment to the end of the study at Month 12
Distress Assessment 2	Distress Thermometer Unit of Measure: Score on a 0-10 scale (higher scores indicate higher distress). Description: The Distress Thermometer is a single-item, self-report tool measuring psychological distress on a 0-10 scale.	From enrollment to the end of the study at Month 12
Distress Assessment 2	Depression, Anxiety, and Stress Scale - 21 Items (DASS-21) Unit of Measure: Score range 0-63 (higher scores indicate greater distress). Description: The DASS-21 is a 21-item scale measuring general affective distress, including depression, anxiety, and stress, over the past week.	From enrollment to the end of the study at Month 12
Self-Efficacy Assessment	General Self-Efficacy Scale (GSE) Unit of Measure: Score range 10-40 (higher scores indicate greater self-efficacy). Description: The GSE scale measures an individual's belief in their ability to cope with adversity.	From enrollment to the end of the study at Month 12
Mood Assessment	Positive and Negative Affect Schedule (PANAS) Unit of Measure: Positive Affect (PA) score range: 10-50 (higher scores indicate greater positive affect).; Negative Affect (NA) score range: 10-50 (higher scores indicate greater negative affect). Description: PANAS is a self-report questionnaire measuring two dimensions of mood: positive and negative affect.	From enrollment to the end of the study at Month 12
Mental Health and Well-Being Assessment	Mental Health Continuum-Short Form (MHC-SF) Unit of Measure: Score range 0-70 (higher scores indicate better mental health). Description: The MHC-SF is a 14-item scale assessing emotional, social, and psychological well-being as indicators of positive mental health.	From enrollment to the end of the study at Month 12
Health-Related Quality of Life (HRQoL)	KIDSCREEN-10 Unit of Measure: Score range 10-50 (higher scores indicate better health-related quality of life). Description: The KIDSCREEN-10 is a health-related quality of life measure for children and adolescents, assessing physical, psychological, and social well-being, autonomy, school environment, and peer and family support.	From enrollment to the end of the study at Month 12

Collaborators and Investigators

• Sponsor: Luis Fernandez Luque
• Collaborators: Hospital Miguel Servet

Study record dates

Study Major Dates

• Study Start (Actual): May 19, 2025
• Primary Completion (Actual): November 12, 2025
• Study Completion (Actual): November 12, 2025

Study Registration Dates

• First Submitted: January 23, 2025
• First Submitted That Met QC Criteria: February 14, 2025
• First Posted (Actual): February 17, 2025

Study Record Updates

• Last Update Posted (Actual): December 1, 2025
• Last Update Submitted That Met QC Criteria: November 27, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Keywords: Digital health; Growth Hormone Disorder; pediatric endocrinology; Personalized Growth Hormone Treatment Profile
• Additional Relevant MeSH Terms: Endocrine System Diseases; Bone Diseases; Musculoskeletal Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hypothalamic Diseases; Pituitary Diseases; Bone Diseases, Endocrine; Bone Diseases, Developmental; Dwarfism; Hypopituitarism; Dwarfism, Pituitary; Physiological Effects of Drugs; Hormones, Hormone Substitutes, and Hormone Antagonists; 2-amino-3-cyano-6-methyl-5-(3,4-dimethoxyphenyl)pyridine
• Other Study ID Numbers: PGTP

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No