Clinical trial NCT06694506: BELux Children OutcoME During A(@)Dulthood With GHD
Official title: BELux Children OutcoME During A(@)Dulthood With Growth Hormone Deficiency
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: NA
Conditions: Growth Hormone Deficiency
Adult Growth Hormone Deficiency
Keywords: Growth Hormone
Adult
Lead sponsor: Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Sponsor class: OTHER
First posted: 2024-11-19
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: We aim to include both male and female adult patients with a diagnosis of childhood onset-GHD, registered in BELGROW, that stopped the GH therapy for growth after 2011 and have a persistent GHD in adolescence/transition period. After completing a questionnaire, each participant will be assigned an analysis group:

* Group 1: 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
* Group 2: Patients who are lost to follow-up or not regularly followed for their GHD (or only followed by their GP with no endocrine assessment)
Primary purpose: DIAGNOSTIC

Brief summary: 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).


Eligibility criteria: 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

Primary outcomes:
measure: Determine the proportion of adult CO-GHD patients who continue to receive regular. medical follow-up
timeFrame: Baseline
Secondary outcomes:
measure: Assessment of the regularity of follow-up and compliance with a questionnaire.
timeFrame: Baseline
measure: Assessment of the possible comorbidities and health issues with a questionnaire.
timeFrame: Baseline
measure: Assessment of the metabolic profile in adulthood.
timeFrame: 1 year per patient
measure: Describe those data according to treatment adherence.
timeFrame: Baseline
Other outcomes:
Locations:
city: Bruxelles
country: Belgium
facility: Cliniques Universitaires Saint-Luc
state: Woluwe-saint-lambert
Study officials:
affiliation: Cliniques universitaires Saint-Luc- Université Catholique de Louvain
name: Philippe Lysy, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: