An observational study in Childhood Short Stature, sponsored by Xiaoping Luo. Recruiting at 1 site in China. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2024-11-05.
Sponsored by Xiaoping Luo · Observational
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).
Childhood short stature includes various conditions such as Growth Hormone Deficiency (GHD), Idiopathic Short Stature (ISS), Small for Gestational Age (SGA), Turner Syndrome (TS), Prader-Willi Syndrome (PWS), Noonan Syndrome (NS), SHOX gene deletion, and 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.
Exclusion Criteria:
Polvethylene glycol recombinant human growth hormone injection (PEG-rhGH) and recombinanthuman growth hormone injection (rhGH) were used.
Drug: 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.
Drug: 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.
Drug: 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.
Also known as: PEG-rhGH or rhGH
The AE (Adverse Event) and SAE (Serious Adverse Event) occurrence rates
An adverse event refers to any unfavorable medical occurrence in a patient after receiving the investigational drug, which can manifest as symptoms, signs, diseases, or abnormalities in laboratory tests, but may not necessarily be causally related to the investigational drug. SAE, on the other hand, stands for serious adverse events, which encompass events such as patient death, life-threatening situations, permanent or severe disabilities or functional loss, hospitalization or extended hospital stays, as well as congenital anomalies or birth defects, among other adverse medical occurrences, following the administration of the investigational drug.
Time frame: From the time of signing the informed consent form to the near adult height(NAH), the overall assessment time was approximately 192 months.
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