A Study Extension Period of PEG-somatropin (Pegylated-somatropin) in the Treatment of Children With Idiopathic Short Stature

A Phase 2 Study Extension Period of Pegylated Somatropin (PEG-somatropin) in the Treatment of Children With Idiopathic Short Stature: An Open, Non-controlled Observational Study.

After the first stage (52 weeks) of Phase II clinical trial, Pegylated recombinant human growth hormone (PEG-rhGH) injection of appropriate dose in compliance with ISS clinical treatment strategy is used to treat children with ISS (Idiopathic Short Stature). The long-term efficacy and safety of the investigational product are evaluated, which can provide more scientific and reliable medication guidance information for clinical diagnosis and treatment.

Study Overview

• Status: Active, not recruiting
• Conditions: Dwarfism
• Intervention / Treatment: Drug: PEG-somatropin

• Study Type: Interventional
• Enrollment (Estimated): 360
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Shanghai, China
    • Shanghai Children's Hospital
  • Shanghai, China
    • Shanghai Children's Hospital of Fudan University
  • Hubei
    • Wuhan, Hubei, China
      • Department of Pediatrics of Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology, Wuhan
  • Jiangsu
    • Nanjing, Jiangsu, China
      • The First Affiated Hospital of Nanjing Medical Universit
    • Wuxi, Jiangsu, China
      • Affiliated Hospital of Jiangnan University
  • Jilin
    • Changchun, Jilin, China
      • The First Hospital of Jilin University
  • Zhejiang
    • Hangzhou, Zhejiang, China
      • The Children's Hospital of Zhejiang University School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 9 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• All subjects who have completed the first stage (52 weeks) of Phase II clinical trial (including negative controls) with completed follow-up records may be enrolled in the extension period study.
• Before the extension period study, the investigator shall fully inform the subjects and their guardians of all the information about the extension period study, including detailed follow-up procedure, treatment plan, laboratory examination items during follow-ups and possible benefits and risks. The extension period study shall only be initiated after the subjects and their guardians are well informed, and agree to cooperate and complete the treatment, follow-ups and examinations during the study, and sign the written informed consent.

Exclusion Criteria:

• Subjects who have taken the following medications within 2 months before entering the extension period study:

  1. Aromatase inhibitors (which include but are not limited to Lelrozol and Anastrozole), with continuous medication ≥1 month;
  2. Gonadotropin releasing hormone analogues (which include but are not limited to Triptorelin, Leuprorelin and Goserelin),, with continuous medication ≥1 month;
  3. Sex steroids (which include but are not limited to any type of estrogen, progestin and androgen) , with continuous medication ≥1 month;
  4. Protein anabolic drugs (which include but are not limited to Oxandrolone, Danazol and Strombafort), with continuous medication ≥1 month;
  5. Glucocorticoids via oral/intravenous administration for more than 1 month..

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: PEG-somatropin; After the first stage (52 weeks) of Phase II clinical trial, the initial medication dose of this extension period is 0.2 mg/kg weight/week of PEG-rhGH for the high dose group, low dose group and negative control group, and it is adjusted in accordance with yearly height velocity (HV) and IGF-1 SDS of each visit. The maximum dose shall not exceed 0.4 mg/kg weight/week.

Drug: PEG-somatropin; After the first stage (52 weeks) of Phase II clinical trial, the initial medication dose of this extension period is 0.2 mg/kg weight/week of PEG-rhGH for high dose group, low dose group and negative control group, and it is adjusted in accordance with yearly height velocity (HV) and IGF-1 SDS of each visit. The maximum dose shall not exceed 0.4 mg/kg weight/week.; Other Names: Polyethylene Glycol Recombinant Human Somatropin Injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change of yearly height velocity (ΔHV)	Change of yearly height velocity before and after treatment. Yearly Height Velocity=12×(Height Yx - Height at Baseline)/(Date of Yx - Date of Baseline)(Yx refers to the height value at particular timepoint x)	Baseline，the end of 3-year addendum

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Standard deviation score of height at the actual age (ΔHT SDS)	Standard deviation score of height at the actual age.	Baseline，every 3 months，the end of 3-year addendum
Change fo Bone maturation	Change fo Bone maturation before and after treatement (bone age/chronological age)	Baseline，every 3 months，the end of 3-year addendum
Change of IGF-1 SDS (ΔIGF-1 SDS)	Change of IGF-1 SDS before and after treatement	Baseline，every 3 months，the end of 3-year addendum
Changes of standard deviation scores of body mass index (ΔBMI SDS)	Changes of standard deviation scores of body mass index	Baseline，every 3 months，the end of 3-year addendum
The yearly average dose of PEG-rhGH injection		Baseline，every 3 months，the end of 3-year addendum
Final height (FH)	Final height	Baseline，every 3 months，the end of 3-year addendum
The improvement of FH compared with the baseline predicted adult height (PAH)		Baseline，every 3 months，the end of 3-year addendum
Improvement of NAH (near adult height)	For subjects who reach NAH with treatment but fail to follow-ups before reaching FH, the improvement of NAH in comparison with the baseline PAH (predicted adult height) shall be evaluated	Baseline，every 3 months，the end of 3-year addendum
the improvement of PAH	For subjects who fail to reach NAH with treatment and fail to follow-ups before reaching FH, the improvement of PAH in comparison with the baseline PAH shall be evaluated	Baseline，every 3 months，the end of 3-year addendum
The changes of the scores evaluated by the Quality of Life Scale		Baseline，every 3 months，the end of 3-year addendum
The changes of lean body mass (LBM) (optional)		Baseline，every 3 months，the end of 3-year addendum
The changes of fat mass (torso) (FM) (optional)		Baseline，every 3 months，the end of 3-year addendum
The changes of the percentage of body fat (optional)		Baseline，every 3 months，the end of 3-year addendum
The changes of bone mineral density (BMD) (optional)		Baseline，every 3 months，the end of 3-year addendum

Collaborators and Investigators

• Sponsor: GeneScience Pharmaceuticals Co., Ltd.
• Collaborators: The First Affiliated Hospital with Nanjing Medical University; Tongji Hospital; Affiliated Hospital of Jiangnan University; Children's Hospital of Fudan University; Shanghai Children's Hospital; The First Hospital of Jilin University; The Children's Hospital of Zhejiang University School of Medicine
• Investigators: Principal Investigator: Xiaoping Luo, Department of Pediatrics of Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology

Study record dates

Study Major Dates

• Study Start (Actual): May 12, 2017
• Primary Completion (Estimated): June 1, 2030
• Study Completion (Estimated): June 1, 2030

Study Registration Dates

• First Submitted: July 14, 2017
• First Submitted That Met QC Criteria: August 16, 2017
• First Posted (Actual): August 21, 2017

Study Record Updates

• Last Update Posted (Actual): June 22, 2023
• Last Update Submitted That Met QC Criteria: June 21, 2023
• Last Verified: February 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Endocrine System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Bone Diseases; Bone Diseases, Developmental; Dwarfism
• Other Study ID Numbers: GenSci 033 CT-Extension Period

Drug and device information, study documents

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