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

Growth Disorders Clinical Trial

Official title:

A Real-world Study of the Efficacy and Safety of PEGylated Recombinant Human Growth Hormone Injection in the Treatment of Short Stature in Chinese Children From the Global Registry for Novel Therapies For Rare Bone or Endocrine Conditions

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06037473
• Other study ID #: Gensci-LAGH-23003
• Status: Recruiting
• Start date: August 1, 2023
• Est. completion date: August 1, 2029

Study information

• Verified date: September 2023
• Source: Beijing Children's Hospital
• Contact: Chunxiu Gong, Dr.
• Phone: 13370115001
• Email: chunxiugong[@]163.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 2600
• Est. completion date: August 1, 2029
• Est. primary completion date: January 1, 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years to 18 Years

Eligibility

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;

Related Conditions & MeSH terms

• Dwarfism
• Growth Disorders
• Growth Hormone Treatment

Intervention

Drug:
• Polyethylene glycol recombinant human growth hormone injection
0.1-0.3mg/kg.w

Locations

Country	Name	City	State
China	Department of Endocrinology, Genetics, Metabolism	Beijing	Beijing

Sponsors (2)

Lead Sponsor	Collaborator
Beijing Children's Hospital	Office for Rare Conditions Registries, University of Glasgow

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	the change in height standard deviation score	final adult height standard deviation score- height standard deviation score before treatment	3-10years