Evaluation of the Propensity of Patients Under rhGH to Envision a Modification of Their Treatment Regimen Toward LAGH

NCT06542809 | Recruiting

• 1 other identifier: 2024/21FEV/083
• Study Type: interventional
• Target: 500
• Locations: 1 country
• Sites: 1

Brief Summary

Daily subcutaneous injections of rhGH can be burdensome for patients, leading to poor adherence and reduced growth outcomes. This has spurred the development of long-acting GH (LAGH) analogues that allow for weekly, biweekly, or monthly injections. Previous studies on LAGH analogues have demonstrated their non-inferiority compared to daily rhGH in terms of increasing growth velocity and improving body composition in children and adults with growth hormone deficiency (GHD), respectively, without significant and unexpected adverse events. Since 2020, three molecules have received approval from the Food and Drug Administration (FDA) for the treatment of pediatric GHD: lonapegsomatropin, somatrogon, and somapacitan. These LAGH analogues may offer better patient acceptance, improved tolerance, and greater therapeutic flexibility. However, these LAGH analogues could also be associated with potential clinical issues in terms of therapeutic monitoring, incidence and duration of side effects, and long-term safety due to a non-physiological GH profile. The introduction of these new LAGH products will require clinicians to identify optimal candidates for LAGH therapy and gain knowledge on monitoring and adjusting treatment.

Conditions

Growth Hormone Deficiency

Keywords

Growth Hormone; New treatment; Propensity; Pediatric

Outcome Measures

Primary Outcomes (1)

• Evaluate families and patients propensity with a questionnaire to envision a switch from rhGH treatment to LAGH

  By distributing a questionnaire designed by the investigators, they are looking to know if patients and families are already aware of these new treatments, likelihood to improve adherence with LAGH or other benefits they might expect from it, potential to increase/decrease risk of treatment-related side effects, concerns they might have in changing the current treatment, what information would they want to receive from their practitioner.

  Baseline

Secondary Outcomes (1)

• Evaluate if families/patients response varies according to age, indication of rhGH treatment, any underlying pathology, and/or duration of rhGH treatment.

  Baseline

Study Arms (1)

Belgian and Luxembourgish patients currently under rhGh treatment

EXPERIMENTAL

Patient population studied are Belgian and Luxembourgish patients currently under rhGh treatment in its various official indications (as per the Belgian RIZIV/INAMI monitor : growth hormone deficiency, Turner Syndrome, chronic renal insufficiency, Prader-Willi syndrome, Small for gestational age, Noonan Syndrome, SHOX gene deficiency) and included in the BELGROW Registry Number.

Other: Questionnaire

Interventions

Questionnaire OTHER

Recruitment will take place through routine paediatric endocrinology consultations with physicians registered with BELSPEED. These doctors will take the time to explain the questionnaire. And allow the patient to complete it.

Belgian and Luxembourgish patients currently under rhGh treatment

Eligibility Criteria

• Age: 1 Day - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Belgian and Luxembourgish patients currently under rhGh treatment in its various official indications (as per the Belgian RIZIV/INAMI monitor : growth hormone deficiency, Turner Syndrome, chronic renal insufficiency, Prader-Willi syndrome, Small for gestational age, Noonan Syndrome, SHOX gene deficiency) and included in the BELGROW Registry
• Male -female
• years
• Free written or e-consent and oral consent

Sponsors & Collaborators

• Cliniques universitaires Saint-Luc- Université Catholique de Louvain: lead
• The Belux Society for Pediatric Endocrinology and Diabetology (BELSPEED): collaborator

Study Sites (1)

Cliniques Universitaires Saint-Luc

Brussels, Woluwe-saint-lambert, 1200, Belgium

RECRUITING

MeSH Terms

Conditions

Dwarfism, Pituitary

Interventions

Surveys and Questionnaires

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

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Philippe Lysy, MD, PhD

  Cliniques universitaires Saint-Luc- Université Catholique de Louvain

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Philippe Lysy, MD, PhD

CONTACT

027641370; philippe.lysy@saintluc.uclouvain.be

Laure Boutsen, MD

CONTACT

+3227641933; laure.boutsen@saintluc.uclouvain.be

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: SCREENING
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 25, 2024
• First Posted: August 7, 2024
• Study Start: April 15, 2024
• Primary Completion: March 1, 2026
• Study Completion: March 1, 2026
• Last Updated: August 7, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1)