Clinical trial NCT06542809: Evaluation of the Propensity of Patients Under rhGH to Envision a Modification of Their Treatment Regimen Toward LAGH
Official title: Evaluation of the Propensity of Patients Under rhGH to Envision a Modification of Their Treatment Regimen Toward LAGH
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Growth Hormone Deficiency
Keywords: Growth Hormone
New treatment
Propensity
Pediatric
Lead sponsor: Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Sponsor class: OTHER
Collaborators: name: The Belux Society for Pediatric Endocrinology and Diabetology (BELSPEED)
First posted: 2024-08-07
Enrollment: 500
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SCREENING

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.


Eligibility criteria: Inclusion Criteria:

* 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
* 0-18 years
* Free written or e-consent and oral consent

Exclusion Criteria:

* No exclusion criteria

Primary outcomes:
measure: Evaluate families and patients propensity with a questionnaire to envision a switch from rhGH treatment to LAGH
timeFrame: Baseline
Secondary outcomes:
measure: Evaluate if families/patients response varies according to age, indication of rhGH treatment, any underlying pathology, and/or duration of rhGH treatment.
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: