Clinical trial NCT06722079: A Real-world Study of Long-acting Growth Hormone Injection for Turner Syndrome
Official title: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Turner Syndrome
Lead sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Tongji Hospital
First posted: 2024-12-09
Enrollment: 743
Sex: FEMALE
Minimum age (years): 0
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The indication in this study is Turner syndrome, which only occurred in females.

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Change in height standard deviation scores (HT SDS) after 1 year (52 weeks) of treatment compared to before (i.e., baseline) (ΔHT SDS).
timeFrame: Week 52
Secondary outcomes:
measure: The annualized height velocity (AHV, cm/ year) after 1 year (52 weeks) of treatment.
timeFrame: Week 52
Other outcomes:
Locations:
city: Wuhan
country: China
facility: Tongji hospital affiliated to tongji medicalcollege of huazhong university of science＆technology
state: Hubei
Study officials:
affiliation: Tongji hospital affiliated to tongji medicalcollege of huazhong university of science＆technology
name: xiaoping Luo, Doctor
role: STUDY_CHAIR
References: