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

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

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).

Study Overview

• Status: Recruiting
• Conditions: Childhood Short Stature
• Intervention / Treatment: Drug: Polvethylene glycol recombinant human growth hormone injection (PEG-rhGH) or recombinanthuman growth hormone injection (rhGH)

• Study Type: Observational
• Enrollment (Estimated): 10000

Contacts and Locations

• Study Contact: Name: xiaoping Luo, doctor; Phone Number: 86 13387522645; Email: xpluo@tjh.tjmu.edu.cn

Study Locations

• China
  • Hubei
    • Wuhan, Hubei, China, 430030
      • Recruiting
      • TongjiHospital
      • Contact: xiaoping Luo, doctor; Phone Number: 86 13387522645; Email: xpluo@tjh.tjmu.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

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.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
prospective cohort; 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.; Other Names: PEG-rhGH or rhGH
retrospective cohort; 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.; Other Names: PEG-rhGH or rhGH
ambispective cohort; 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.; Other Names: PEG-rhGH or rhGH

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	From the time of signing the informed consent form to the near adult height(NAH), the overall assessment time was approximately 192 months.

Collaborators and Investigators

• Sponsor: Xiaoping Luo
• Investigators: Principal Investigator: xiaoping Luo, doctor, Tongji Hospital

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2023
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027

Study Registration Dates

• First Submitted: October 7, 2023
• First Submitted That Met QC Criteria: October 31, 2023
• First Posted (Actual): November 1, 2023

Study Record Updates

• Last Update Posted (Estimated): November 5, 2024
• Last Update Submitted That Met QC Criteria: November 4, 2024
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Endocrine System Diseases; Bone Diseases; Musculoskeletal Diseases; Genetic Diseases, Inborn; Bone Diseases, Developmental; Dwarfism; Physiological Effects of Drugs; Hormones, Hormone Substitutes, and Hormone Antagonists; Hormones
• Other Study ID Numbers: TJ-IRB202410048

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No