跳至主要内容
临床试验/NCT06542809
NCT06542809招募中不适用

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

Cliniques universitaires Saint-Luc- Université Catholique de Louvain2 个研究点 分布在 1 个国家目标入组 500 人开始时间: 2024年4月15日最近更新:
适应症

试验速览

阶段
不适用
状态
招募中
发起方
入组人数
500
试验地点
2
主要终点
Evaluate families and patients propensity with a questionnaire to envision a switch from rhGH treatment to LAGH

研究概览

简要总结

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.

研究设计

研究类型
Interventional
分配方式
Na
干预模型
Single Group
主要目的
Screening
盲法
None

入排标准

年龄范围
1 Day 至 18 Years(Child, Adult)
性别
All
接受健康志愿者

入选标准

  • 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

排除标准

  • No exclusion criteria

结局指标

主要结局

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

时间窗: 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.

次要结局

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

研究者

发起方
Cliniques universitaires Saint-Luc- Université Catholique de Louvain
申办方类型
Other
责任方
Sponsor

研究点 (2)

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