A Real-world Study of Long-acting Growth Hormone Injection for Turner Syndrome

A Comparative Study of the Therapeutic Effect of PEGylated Recombinant Human Growth Hormone(PEG-rhGH) Injection on Turner Syndrome in a Clinical Trial and Short-acting Recombinant Human Growth Hormone in a Real World Study

This study is divided into two parts:

Part A: A multicenter, retrospective, observational study to evaluate the effectiveness of PEG-rhGH in the treatment of Turner syndrome with domestic real-world data on rhGH as external controls. The collection and arrangement of real world data is the content of the real world study (RWS); Part B: Meta analysis (MA) was conducted based on the previous literatures on rhGH in the treatment of Turner syndrome, and MA results were used as external controls to evaluate the effectiveness of PEG-rhGH.

Real-world data on rhGH treatment of Turner syndrome in China were obtained from the HIS system and/or paper medical records of participating domestic centers and/or photocopied/printed medical records of other hospitals. Data on the efficacy of PEG-rhGH injection in the treatment of Turner syndrome were obtained from the 0.2mg /kg/ week group in the Phase II clinical trial of PEG-rhGH injection in the treatment of Turner syndrome (multicenter, randomized, blank control, superior efficacy) conducted by Changchun GeneScience Pharmaceutical Co., Ltd (Protocol number: GenSci032-02, version date: April 27, 2015). The MA study data of rhGH in the treatment of Turner syndrome in foreign countries came from the relevant literature retrieved from the approved drug database of FDA, PubMed and Web of Science database.

Study Overview

• Status: Completed
• Conditions: Turner Syndrome

• Study Type: Observational
• Enrollment (Actual): 743

Contacts and Locations

Study Locations

• China
  • Hubei
    • Wuhan, Hubei, China, 430000
      • Tongji hospital affiliated to tongji medicalcollege of huazhong university of science＆technology

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

The indication in this study is Turner syndrome, which only occurred in females.

Description

Inclusion Criteria:

• Patients diagnosed with Turner syndrome by a clinician;
• The first treatment was short-acting rhGH, and the first administration date was not earlier than July 1, 2008;
• Follow-up data are available at the center before the first dosing and after at least one dosing.

Exclusion Criteria:

● None.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
PEG-rhGH injection; The group of Turner syndrome patients treated with a dose of 0.2 mg/kg/ week in GenSci032-02 clinical trial.
rhGH injection; Part A control group: a group of Turner syndrome patients treated with rhGH who met the inclusion criteria of the RWS study population from participating research centers in China. Part B control group: studies of rhGH treatment for Turner syndrome performed abroad that met all MA inclusion criteria and did not meet any MA exclusion criteria.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in height standard deviation scores (HT SDS) after 1 year (52 weeks) of treatment compared to before (i.e., baseline) (ΔHT SDS).	Change in height standard deviation scores (HT SDS) after 1 year (52 weeks) of treatment compared to before (i.e., baseline) (ΔHT SDS).	Week 52

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The annualized height velocity (AHV, cm/ year) after 1 year (52 weeks) of treatment.	The annualized height velocity (AHV, cm/ year) after 1 year (52 weeks) of treatment.	Week 52

Collaborators and Investigators

• Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.
• Collaborators: Tongji Hospital
• Investigators: Study Chair: xiaoping Luo, Doctor, Tongji hospital affiliated to tongji medicalcollege of huazhong university of science＆technology

Study record dates

Study Major Dates

• Study Start (Actual): November 8, 2022
• Primary Completion (Actual): June 5, 2023
• Study Completion (Actual): June 5, 2023

Study Registration Dates

• First Submitted: December 3, 2024
• First Submitted That Met QC Criteria: December 5, 2024
• First Posted (Estimated): December 9, 2024

Study Record Updates

• Last Update Posted (Estimated): December 9, 2024
• Last Update Submitted That Met QC Criteria: December 5, 2024
• Last Verified: December 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urogenital Diseases; Endocrine System Diseases; Cardiovascular Diseases; Pathologic Processes; Male Urogenital Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Heart Diseases; Genetic Diseases, Inborn; Disease; Gonadal Disorders; Congenital Abnormalities; Cardiovascular Abnormalities; Heart Defects, Congenital; Disorders of Sex Development; Urogenital Abnormalities; Sex Chromosome Disorders; Chromosome Disorders; Sex Chromosome Disorders of Sex Development; Syndrome; Turner Syndrome; Gonadal Dysgenesis
• Other Study ID Numbers: Gensci032-401

Drug and device information, study documents

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