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NCT Number: NCT06542809

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

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.

Recruiting

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Key information

Age range

1 day–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Cliniques Universitaires Saint-Luc

Brussels, Woluwe-saint-lambert, 1200, Belgium

Location status: Recruiting

Location contact

Philippe Lysy, Pr

CONTACT

[email protected]

02764130 ext. +32

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

Primary outcomes

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

    Time frame: Baseline

    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.

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.

    Time frame: Baseline

    By distributing a questionnaire designed by the investigators.

Study contacts

Contact information is provided by the study sponsor or research team.

Laure Boutsen, MD

CONTACT

[email protected]

+3227641933

Philippe Lysy, MD, PhD

CONTACT

[email protected]

027641370 ext. +32

Sponsors and collaborators

Lead sponsor

Cliniques universitaires Saint-Luc- Université Catholique de Louvain

Other

Collaborators

  • The Belux Society for Pediatric Endocrinology and Diabetology (BELSPEED)

Registry information

Acronym: TOWARD-LAGH

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Aug 7, 2024
Registry last updated
Aug 7, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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