Long-term Efficacy and Safety Evaluation of Growth Hormone in Children in China(CGLS)

Официальное название

Long-term Efficacy and Safety Evaluation of Growth Hormone in Children in China(CGLS)-an Open-label, Multicenter, Prospective and Retrospective Observational Study

Обзор

Short stature is a relatively common pediatric condition, referring to individuals whose height is more than 2 (-2 SD) standard deviations below the average height of a similar age, gender, and ethnicity population in similar living conditions, or those below the third percentile (-1.88 SD). This study is an open-label, multicenter, prospective and retrospective, observational, cohort study aimed at assessing the long-term safety and efficacy of PEG-rhGH or rhGH treatment for Chinese children with short stature. The study is divided into retrospective cohorts, retrospective prospective cohorts, and prospective cohorts. It is expected to include approximately 10000 patients (including around 3000 in the retrospective cohorts and around 7000 in the retrospective prospective and prospective cohorts). The total duration is expected to be 16 years, including 2 years for study center initiation and patient recruitment and follow-up of patients in the retrospective prospective and prospective cohorts until near-adult height (NAH). The primary objective is to evaluate the long-term safety of PEG-rhGH or rhGH for the treatment of children with short stature (including GHD, ISS, SGA, TS, PWS, NS, SHOX gene deletion, and other etiologies); the secondary objective is to assess the effectiveness of PEG-rhGH or rhGH treatment for children with short stature (including GHD, ISS, SGA, TS, PWS, NS, SHOX gene deletion, and other etiologies).

Вмешательства

• Препарат Polvethylene glycol recombinant human growth hormone injection (PEG-rhGH) or recombinanthuman growth hormone injection (rhGH)
  Polvethylene glycol recombinant human growth hormone injection (PEG-rhGH) and recombinanthuman growth hormone injection (rhGH) were used.

Первичные конечные точки

• The AE (Adverse Event) and SAE (Serious Adverse Event) occurrence rates [Срок оценки: From the time of signing the informed consent form to the near adult height(NAH), the overall assessment time was approximately 192 months.]

Критерии участия

Критерии включения

• Children aged 2 years and older with various causes of short stature.
• Height below the 3rd percentile (-1.88 SD) compared to the height of normal, healthy children of the same age and gender.
• Belonging to one of the following indications: GHD, ISS, SGA, TS, PWS, NS, SHOX gene deletion, or other causes of short stature.

Note: In this study, the inclusion criteria for the PWS cohort do not impose restrictions on height and age at the start of treatment.

Критерии исключения

• Patients with fully closed growth plates.
• Patients with signs of potential tumor progression or those already diagnosed with tumors.
• Allergic reactions to growth hormone or its adjuvants.
• Patients who have received any growth hormone treatment other than the investigational drug in the six months prior to screening.
• Any other circumstances deemed unsuitable for study inclusion by the investigator.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Китай · 1 центр

• TongjiHospital — Ухань

Публикации

• Wu W, Luo X. Long-Term Efficacy and Safety of Growth Hormone in Children Suffering from Short Stature in China (CGLS): An Open-Label, Multicenter, Prospective and Retrospective, Observational Study. Adv Ther. 2025 Jun;42(6):2957-2969. doi: 10.1007/s12325-025-03146-2. Epub 2025 Apr 8. PMID 40198521

Идентификаторы

NCT: NCT06110910 · TJ-IRB202410048

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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