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

NCT06722079 | Completed

• 1 other identifier: Gensci032-401
• Study Type: observational
• Target: 743
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Turner Syndrome

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• The annualized height velocity (AHV, cm/ year) after 1 year (52 weeks) of treatment.

  Week 52

Study Arms (2)

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.

Eligibility Criteria

• Age: 0 Years - 18 Years
• Gender Eligibility Details: The indication in this study is Turner syndrome, which only occurred in females.
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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.

You may not qualify if:

• ● None.

Sponsors & Collaborators

• Changchun GeneScience Pharmaceutical Co., Ltd.: lead
• Tongji Hospital: collaborator

Study Sites (1)

Tongji hospital affiliated to tongji medicalcollege of huazhong university of science＆technology

Wuhan, Hubei, 430000, China

Location

MeSH Terms

Conditions

Turner Syndrome

Condition Hierarchy (Ancestors)

Gonadal Dysgenesis; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Sex Chromosome Disorders of Sex Development; Male Urogenital Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Sex Chromosome Disorders; Chromosome Disorders; Genetic Diseases, Inborn; Gonadal Disorders; Endocrine System Diseases

Study Officials

• xiaoping Luo, Doctor

  Tongji hospital affiliated to tongji medicalcollege of huazhong university of science＆technology

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 3, 2024
• First Posted: December 9, 2024
• Study Start: November 8, 2022
• Primary Completion: June 5, 2023
• Study Completion: June 5, 2023
• Last Updated: December 9, 2024

Record last verified: 2024-12

Locations

CN (1)