BELux Children OutcoME During A(@)Dulthood With GHD (BELCOME@GHD)

November 15, 2024 updated by: Cliniques universitaires Saint-Luc- Université Catholique de Louvain

BELux Children OutcoME During A(@)Dulthood With Growth Hormone Deficiency

Our objective is to evaluate the outcome in adulthood of Belgian and Luxembourgish patients treated with rhGH during childhood for CO-GHD. The primary goal is to determine the proportion of adult CO-GHD patients who continue to receive regular medical follow-up and those still undergoing rhGH therapy using a questionnaire. Secondary objectives include assessing the regularity of follow-up and compliance with treatment in adulthood, evaluating possible comorbidities, health issues, lifestyle, living environment, and quality of life. Additionally, we aim to assess the metabolic profile in adulthood, particularly focusing on BMI, glycemic, and lipid data, for patients who consent to share their current clinical and biological data. Data will be described according to treatment adherence and CO-GHD etiology/phenotypes (e.g., idiopathic vs. organic GHD, isolated vs. combined GHD, partial vs. severe GHD).

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

200

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Age > or = 18 years
  • Diagnosed with child-hood onset growth hormone deficiency (CO-GHD) (no exclusion criteria: isolated or multiple GHD, idiopathic or organic, severe or partial)
  • Registered in the BELGROW registry (informed consent at the time of inclusion in registry)
  • Stopped treatment with rhGH for growth after 2011
  • Persistent GHD at the end of growth

Exclusion Criteria:

  • No exclusion criteria

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Diagnostic
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
No Intervention: Patients with regular follow-up for their GHD (treated with rhGH or not)
Patients with regular follow-up for their GHD (treated with rhGH or not) in a adult endocrinology department including a clinical examination and endocrine blood work-up at least once a year
Active Comparator: Patients who are lost to follow-up or not regularly followed for their GHD
Patients who are lost to follow-up or not regularly followed for their GHD (or only followed by their GP with no endocrine assessment)
Other: Biological Sample
Clinical examination and blood test. The clinical examination and blood test are considered standard-of-care as they could be performed annually or bi-annually as part of an adult GHD follow-up. Subsequently, the data will be collected directly from the patient's chosen physician.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Determine the proportion of adult CO-GHD patients who continue to receive regular. medical follow-up
Time Frame: Baseline
Using our questionnaire, the primary objective is to determine the proportion of adult CO-GHD patients who continue to receive regular medical follow-up (and where/by who) and the proportion of patients still undergoing rhGH therapy.
Baseline

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assessment of the regularity of follow-up and compliance with a questionnaire.
Time Frame: Baseline
Assessment of the regularity of follow-up and compliance to treatment in adulthood (in patients that are still followed and/or treated with rhGH therapy), with the help of a questionnaire created by the research team.
Baseline
Assessment of the possible comorbidities and health issues with a questionnaire.
Time Frame: Baseline
Assessment of the possible comorbidities and health issues, lifestyle, living environment and quality of live (all patients) in adulthood. with the help of a questionnaire created by the research team.
Baseline
Assessment of the metabolic profile in adulthood.
Time Frame: 1 year per patient
Assessment of the metabolic profile in adulthood - particularly in relation to BMI, glycemic and lipid data. (fasting glycemia, fasting insulin, hemoglobin A1c, transaminase levels, lipid profile (total cholesterol, high-density lipoprotein (HDL), low-density lipoprotein (LDL), and triglycerides), insulin-like growth factor 1 (IGF-1) levels, and other hormone levels (thyroid-stimulating hormone (TSH), thyroxine (T4), prolactin (PRL), luteinizing hormone (LH), follicle-stimulating hormone (FSH), estradiol/testosterone, cortisol).). Each of these data will be compared with the clinical data in the same way.
1 year per patient
Describe those data according to treatment adherence.
Time Frame: Baseline
Describe those data according to treatment adherence and CO- GHD etiology/phenotypes (e.g. idiopathic vs organic GHD, isolated vs combined GHD, partial vs severe GHD) The questionnaire will provide us with information on patients' adherence to treatment.
Baseline

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cliniques universitaires Saint-Luc- Université Catholique de Louvain

Investigators

  • Principal Investigator: Philippe Lysy, MD, PhD, Cliniques universitaires Saint-Luc

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

December 15, 2024

Primary Completion (Estimated)

August 1, 2029

Study Completion (Estimated)

August 1, 2029

Study Registration Dates

First Submitted

July 25, 2024

First Submitted That Met QC Criteria

November 15, 2024

First Posted (Estimated)

November 19, 2024

Study Record Updates

Last Update Posted (Estimated)

November 19, 2024

Last Update Submitted That Met QC Criteria

November 15, 2024

Last Verified

November 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CUSL

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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