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

September 12, 2023 updated by: Chunxiu Gong, Beijing Children's Hospital

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Observational

Enrollment (Estimated)

2600

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Beijing
      • Beijing, Beijing, China, 010
        • Recruiting
        • Department of Endocrinology, Genetics, Metabolism
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

The height of the patients was below 2 stadard deviation below average. The patients were diagosesed as endogenous growth hormone deficiency, TS, SGA, ISS, etc. and treated with polyethylene glycol recombinant human growth hormone injection

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
growth hormone deficiency(GHD)
Growth hormone deficiency (GHD) is a rare and treatable condition that causes short height in children and metabolic issues in adults.
Drug: Polyethylene glycol recombinant human growth hormone injection
0.1-0.3mg/kg.w
Idiopathic short stature (ISS)
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.
Drug: Polyethylene glycol recombinant human growth hormone injection
0.1-0.3mg/kg.w
small for gestational age (SGA)
Small for gestational age (SGA) is defined as a birth weight of less than 10th percentile for gestational age.
Drug: Polyethylene glycol recombinant human growth hormone injection
0.1-0.3mg/kg.w
Turner Syndrome (TS)
Turner syndrome, a condition that affects only females, results when one of the X chromosomes (sex chromosomes) is missing or partially missing.
Drug: Polyethylene glycol recombinant human growth hormone injection
0.1-0.3mg/kg.w
others
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.
Drug: Polyethylene glycol recombinant human growth hormone injection
0.1-0.3mg/kg.w

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
the change in height standard deviation score
Time Frame: 3-10years
final adult height standard deviation score- height standard deviation score before treatment
3-10years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Beijing Children's Hospital

Collaborators

Office for Rare Conditions Registries, University of Glasgow

Investigators

  • Principal Investigator: Bingyan Cao, Dr., Beijing Children's Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 1, 2023

Primary Completion (Estimated)

January 1, 2029

Study Completion (Estimated)

August 1, 2029

Study Registration Dates

First Submitted

August 15, 2023

First Submitted That Met QC Criteria

September 12, 2023

First Posted (Actual)

September 14, 2023

Study Record Updates

Last Update Posted (Actual)

September 14, 2023

Last Update Submitted That Met QC Criteria

September 12, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Gensci-LAGH-23003

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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