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

NCT06830525 | Completed

• 1 other identifier: PGTP
• Study Type: observational
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Growth Hormone Deficiency (GHD)

Keywords

Growth Hormone Disorder; Digital health; pediatric endocrinology; Personalized Growth Hormone Treatment Profile

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (13)

• Adherence to Treatment Using Connected Injector Devices

  From enrollment to the end of the study at Month 12

• Physiological Monitoring via Wearable Devices - Sleep Quality

  From enrollment to the end of the study at Month 12

• Physiological Monitoring via Wearable Devices - Heart Rate

  From enrollment to the end of the study at Month 12

• Physiological Monitoring via Wearable Devices - Physical Activity Metrics

  From enrollment to the end of the study at Month 12

• Physiological Monitoring via Wearable Devices - Blood Oxygen Levels

  From enrollment to the end of the study at Month 12

Study Arms (1)

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

Interventions

ACDP® and growth hormone treatment COMBINATION_PRODUCT

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.

Families with children undergoing growth hormone treatment

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Luis Fernandez Luque: lead
• Hospital Miguel Servet: collaborator

Study Sites (1)

Hospital Universitario Miguel Servet

Zaragoza, Aragon, Spain

Location

MeSH Terms

Conditions

Dwarfism, Pituitary

Interventions

2-amino-3-cyano-6-methyl-5-(3,4-dimethoxyphenyl)pyridine

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief Scientific Officer

Study Record Dates

• First Submitted: January 23, 2025
• First Posted: February 17, 2025
• Study Start: May 19, 2025
• Primary Completion: November 12, 2025
• Study Completion: November 12, 2025
• Last Updated: December 1, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1)