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

Xiaoping Luo

Status: Enrolling

Conditions: Childhood Short Stature

Treatments: Drug: Polvethylene glycol recombinant human growth hormone injection (PEG-rhGH) or recombinanthuman growth hormone injection (rhGH)

Study type: Observational

Funder types: Other

Identifiers

NCT06110910

TJ-IRB202410048

Details and patient eligibility

About

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).

Enrollment: 10,000 estimated patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

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.

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

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. Any other circumstances deemed unsuitable for study inclusion by the investigator.

Trial design

10,000 participants in 3 patient groups

prospective cohort

Description:

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

Treatment:

Drug: Polvethylene glycol recombinant human growth hormone injection (PEG-rhGH) or recombinanthuman growth hormone injection (rhGH)

retrospective cohort

Description:

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

Treatment:

Drug: Polvethylene glycol recombinant human growth hormone injection (PEG-rhGH) or recombinanthuman growth hormone injection (rhGH)

ambispective cohort

Description:

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

Treatment:

Drug: Polvethylene glycol recombinant human growth hormone injection (PEG-rhGH) or recombinanthuman growth hormone injection (rhGH)