BELux Children OutcoME During A(@)Dulthood With GHD

NCT06694506 | Not Yet Recruiting

• 1 other identifier: CUSL
• Study Type: interventional
• Target: 200
• Locations: 1 country
• Sites: 1

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).

Conditions

Growth Hormone Deficiency; Adult Growth Hormone Deficiency

Keywords

Growth Hormone; Adult

Outcome Measures

Primary Outcomes (1)

• Determine the proportion of adult CO-GHD patients who continue to receive regular. medical follow-up

  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 Outcomes (4)

• Assessment of the regularity of follow-up and compliance with a questionnaire.

  Baseline

• Assessment of the possible comorbidities and health issues with a questionnaire.

  Baseline

• Assessment of the metabolic profile in adulthood.

  1 year per patient

• Describe those data according to treatment adherence.

  Baseline

Study Arms (2)

Patients with regular follow-up for their GHD (treated with rhGH or not)

NO INTERVENTION

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

Patients who are lost to follow-up or not regularly followed for their GHD

ACTIVE COMPARATOR

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

Interventions

Biological Sample OTHER

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.

Patients who are lost to follow-up or not regularly followed for their GHD

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age \> or = 18 years

You may not qualify if:

• Stopped treatment with rhGH for growth after 2011
• Persistent GHD at the end of growth

Sponsors & Collaborators

• Cliniques universitaires Saint-Luc- Université Catholique de Louvain: lead

Study Sites (1)

Cliniques Universitaires Saint-Luc

Brussels, Woluwe-saint-lambert, 1200, Belgium

Location

MeSH Terms

Conditions

Dwarfism, Pituitary

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 Officials

• Philippe Lysy, MD, PhD

  Cliniques universitaires Saint-Luc- Université Catholique de Louvain

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Philippe Lysy, MD, PhD

CONTACT

02 7641370; philippe.lysy@saintluc.uclouvain.be

Laure Boutsen, MD

CONTACT

02 7641933; laure.boutsen@saintluc.uclouvain.be

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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)

Study Record Dates

• First Submitted: July 25, 2024
• First Posted: November 19, 2024
• Study Start: December 15, 2024
• Primary Completion (Estimated): August 1, 2029
• Study Completion (Estimated): August 1, 2029
• Last Updated: November 19, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1)