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

TJ-IRB202303156

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 5000 patients (including around 3000 in the retrospective cohorts and around 2000 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).

2023-03-01

2027-12-31

2027-12-31

Recruiting

5000

Inclusion Criteria: - 1. Children aged 2 years and older with various causes of short stature. 2. Height below the 3rd percentile (-1.88 SD) compared to the height of normal, healthy children of the same age and gender. 3. Belonging to one of the following indications: GHD, ISS, SGA, TS, PWS, NS, SHOX gene deletion, or other causes of short stature. Exclusion Criteria: * 1. Patients with fully closed growth plates. 2. Patients with signs of potential tumor progression or those already diagnosed with tumors. 3. Allergic reactions to growth hormone or its adjuvants. 4. Patients who have received any growth hormone treatment other than the investigational drug in the six months prior to screening. 5. Unable to provide complete medical records or expected to be unable to complete follow-up or collect complete medical records. 6. Any other circumstances deemed unsuitable for study inclusion by the investigator.

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'OTHER'}, 'enrollmentInfo': {'count': 5000, 'type': 'ESTIMATED'}}

2023-11-01