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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT06722079

Enrollment

743

Registered

2024-12-09

Start date

2022-11-08

Completion date

2023-06-05

Last updated

2024-12-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Turner Syndrome

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.

Interventions

None listed

Study design

Observational model

OTHER

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

FEMALE

Age

0 Years to 18 Years

Healthy volunteers

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.

Design outcomes

Primary

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

Secondary

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

Countries

China

Outcome results