The Dosage Exploration Study of PEG-rhGH for Treating Short Stature in Prepubertal and Pubertal Children

This study is dedicated to addressing the lack of research on the most effective dosage of long-acting growth hormone for children with short stature. By employing clinical trial design, we are committed to investigating the therapeutic benefits and safety profiles associated with varying doses of long-acting growth hormone. Our ultimate goal is to offer clinicians more precise treatment guidance and assist patients in attaining optimal growth and developmental outcomes.

Study Overview

• Status: Not yet recruiting
• Conditions: Short Stature
• Intervention / Treatment: Drug: PEG-rhGH; Drug: GnRHa

• Study Type: Interventional
• Enrollment (Estimated): 240
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Xiaoou Shan; Phone Number: +86 1377779922; Email: 2296037178@qq.com
• Study Contact Backup: Name: Mengjie Yang; Phone Number: +86 159 5871 0186; Email: 540718644@qq.com

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

1. Patients with short stature or a genetic target height below 2 standard deviations;
2. Individuals who are pre-pubertal (Tanner stage I) and pubertal (Tanner stages II-IV).
3. Legal guardians consenting to participate in the study and signing informed consent forms.

Exclusion Criteria:

1. Known or suspected hypersensitivity reactions to the investigational product or related products;
2. Presence of severe systemic diseases;
3. Patients with malignant tumors;
4. Participation in any other clinical trial and receipt of drug or non-drug interventions within the 3 months prior to screening;
5. Patients unable to adhere to follow-up or receive treatment as scheduled;
6. Other circumstances in which the investigator deems the patient unsuitable for inclusion in this clinical trial.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: prepubertal children of short stature; prepubertal children of short stature with different Initial doses of PEG-rhGH	Drug: PEG-rhGH; 1= 0.2mg/kg/week Initial doses of PEG-rhGH 2=0.22mg/kg/week Initial doses of PEG-rhGH; Other Names: 1; 2
Other: pubertal children of short stature; pubertal children of short stature with different Initial doses of PEG-rhGH with or without GnRHa treatment	Drug: PEG-rhGH; 1= 0.2mg/kg/week Initial doses of PEG-rhGH 2=0.22mg/kg/week Initial doses of PEG-rhGH; Other Names: 1; 2; Drug: GnRHa; pubertal children of short stature without GnRHa treatment; pubertal children of short stature with GnRHa treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
height growth rate	Measure the height and observe how many centimeters it grows each month	2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Sexual development status	Evaluate Tanner staging, The Tanner Staging, also known as Sexual Maturity Rating (SMR), is a classification system that healthcare providers use to document and track the development of secondary sex characteristics of children during puberty. It's a tool that outlines the stages of puberty for children and when they're likely to occur.	two years
IGF-1	IGF-1, known as insulin-like growth factor, is utilized to evaluate the efficacy of growth hormone therapy. By measuring IGF-1 levels, doctors can determine the effectiveness of the treatment and whether the patient's growth falls within the normal range	two years

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
bone age	Bone age assessment	2 years

Collaborators and Investigators

• Sponsor: Second Affiliated Hospital of Wenzhou Medical University

Study record dates

Study Major Dates

• Study Start (Estimated): January 15, 2025
• Primary Completion (Estimated): December 30, 2026
• Study Completion (Estimated): June 1, 2027

Study Registration Dates

• First Submitted: December 24, 2024
• First Submitted That Met QC Criteria: January 6, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 6, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Endocrine System Diseases; Bone Diseases; Musculoskeletal Diseases; Genetic Diseases, Inborn; Bone Diseases, Developmental; Dwarfism
• Other Study ID Numbers: SAHoWMU-CR2024-06-223

Drug and device information, study documents

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