The Efficacy and Safety of PEGylated GH for the Treatment of Short Stature in Chinese Children-GLOBE Reg

Recruiting

• Conditions: Growth Hormone Treatment; Growth Disorders
• Interventions: Drug: Polyethylene glycol recombinant human growth hormone injection

• Registration Number: NCT06037473
• Lead Sponsor: Beijing Children's Hospital

Brief Summary

In order to further observe the long-term safety and effectiveness of real-world polyethylene glycol-recombinant human growth hormone(PEG-rhGH) treatment of GHD, idiopathic short stature, and SGA in children, explore and analyze the factors affecting the efficacy of PEG-rhGH and the height prediction model after treatment, etc., collect and analyze more scientifically and rationally, and understand the situation of real-world PEG-GH treatment. A database registration study was developed.

Detailed Description

A database registration study was developed to retrospectively analyze the efficacy and safety of polyethylene glycol recombinant human growth hormone injection in the treatment of slow growth in children caused by endogenous growth hormone deficiency, idiopathic short stature, Turner syndrome, etc., and to further prospectively follow up and monitor. In order to participate in the global real-world use of long-acting growth hormone, all data from this study were uploaded to the Global Registry of New Therapies for Rare Endocrine Diseases and Bone Disorders (GloBE-Reg) platform.

Study Timeline

• Study Start: 2023-08-01
• Study First Submitted: 2023-08-15
• Status Verified: 2023-09
• Study First Submitted QC: 2023-09-12
• Last Update Submitted: 2023-09-12
• Study First Posted: 2023-09-14
• Last Update Posted: 2023-09-14
• Primary Completion: 2029-01-01
• Study Completion: 2029-08-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 2600

Inclusion Criteria

Patients with endogenous growth hormone deficiency, Turner Syndrome, SGA, idiopathic short stature, etc. treated with polyethylene glycol recombinant human growth hormone injection

Exclusion Criteria

Patients with serious heart and lung, blood system, malignant tumors and other diseases or systemic infections, immune function is low and Persons with mental illness;

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
growth hormone deficiency(GHD)	Polyethylene glycol recombinant human growth hormone injection	Growth hormone deficiency (GHD) is a rare and treatable condition that causes short height in children and metabolic issues in adults.
others	Polyethylene glycol recombinant human growth hormone injection	others who with the height of the individual is more than 2 SD below the corresponding mean height for a given age, sex and population.
Idiopathic short stature (ISS)	Polyethylene glycol recombinant human growth hormone injection	Idiopathic short stature is a condition in which the height of the individual is more than 2 SD below the corresponding mean height for a given age, sex and population, in whom no identifiable disorder is present.
small for gestational age (SGA)	Polyethylene glycol recombinant human growth hormone injection	Small for gestational age (SGA) is defined as a birth weight of less than 10th percentile for gestational age.
Turner Syndrome (TS)	Polyethylene glycol recombinant human growth hormone injection	Turner syndrome, a condition that affects only females, results when one of the X chromosomes (sex chromosomes) is missing or partially missing.

Primary Outcome Measures

Name	Time	Method
the change in height standard deviation score	3-10years	final adult height standard deviation score- height standard deviation score before treatment

Trial Locations

Locations (1)

Department of Endocrinology, Genetics, Metabolism; 🇨🇳 Beijing, Beijing, China