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Active, not recruitingNCT03255694Updated Aug 15, 2025

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

A Phase 2 interventional study of PEG-somatropin in Dwarfism, sponsored by Changchun GeneScience Pharmaceutical Co., Ltd.. Active, not recruiting at 12 sites in China. Open to participants aged 4 Years to 9 Years. Per ClinicalTrials.gov, last updated 2025-08-15.

Sponsored by Changchun GeneScience Pharmaceutical Co., Ltd. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
360
Allocation
Not applicable
Ages
4 Years to 9 Years
Sex
All
01

Study summary

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.

02

Conditions studied

  • Dwarfism

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03

Who can participate

Ages eligible
4 Years to 9 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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..
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
360 participants (estimated)

Study arms

  • 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

Interventions

  • DrugPEG-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.

    Also known as: Polyethylene Glycol Recombinant Human Somatropin Injection

05

What researchers measure

Primary outcomes

  1. 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)

    Time frame: Baseline，the end of 3-year addendum

Secondary outcomes

  1. Standard deviation score of height at the actual age (ΔHT SDS)

    Standard deviation score of height at the actual age.

    Time frame: Baseline，every 3 months，the end of 3-year addendum

  2. Change fo Bone maturation

    Change fo Bone maturation before and after treatement (bone age/chronological age)

    Time frame: Baseline，every 3 months，the end of 3-year addendum

  3. Change of IGF-1 SDS (ΔIGF-1 SDS)

    Change of IGF-1 SDS before and after treatement

    Time frame: Baseline，every 3 months，the end of 3-year addendum

  4. Changes of standard deviation scores of body mass index (ΔBMI SDS)

    Changes of standard deviation scores of body mass index

    Time frame: Baseline，every 3 months，the end of 3-year addendum

  5. The yearly average dose of PEG-rhGH injection

    Time frame: Baseline，every 3 months，the end of 3-year addendum

  6. Final height (FH)

    Final height

    Time frame: Baseline，every 3 months，the end of 3-year addendum

  7. The improvement of FH compared with the baseline predicted adult height (PAH)

    Time frame: Baseline，every 3 months，the end of 3-year addendum

  8. 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

    Time frame: Baseline，every 3 months，the end of 3-year addendum

  9. 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

    Time frame: Baseline，every 3 months，the end of 3-year addendum

  10. The changes of the scores evaluated by the Quality of Life Scale

    Time frame: Baseline，every 3 months，the end of 3-year addendum

  11. The changes of lean body mass (LBM) (optional)

    Time frame: Baseline，every 3 months，the end of 3-year addendum

  12. The changes of fat mass (torso) (FM) (optional)

    Time frame: Baseline，every 3 months，the end of 3-year addendum

  13. The changes of the percentage of body fat (optional)

    Time frame: Baseline，every 3 months，the end of 3-year addendum

  14. The changes of bone mineral density (BMD) (optional)

    Time frame: Baseline，every 3 months，the end of 3-year addendum

06

Study locations

12 sites
  • Beijing Children's Hospital, Capital Medical University, National Center for Children's Health
    Beijing, Beijing Municipality, China
  • Department of Pediatrics of Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology, Wuhan
    Wuhan, Hubei, China
  • Hunan Children's Hospital
    Changsha, Hunan, China
  • Children's Hospital of Soochow University
    Naning, Jiangsu, China
  • The First Affiated Hospital of Nanjing Medical Universit
    Nanjing, Jiangsu, China
  • Affiliated Hospital of Jiangnan University
    Wuxi, Jiangsu, China
  • Wuxi Children's Hospital
    Wuxi, Jiangsu, China
  • Jiangxi Provincial Children's Hospital
    Nanchang, Jiangxi, China
  • The First Hospital of Jilin University
    Changchun, Jilin, China
  • The Children's Hospital of Zhejiang University School of Medicine
    Hangzhou, Zhejiang, China
  • Shanghai Children's Hospital of Fudan University
    Shanghai, China
  • Shanghai Children's Hospital
    Shanghai, China
07

Registry details

Key details

Study ID
NCT03255694
Lead sponsor
Changchun GeneScience Pharmaceutical Co., Ltd.
Collaborators
Tongji Hospital, The First Hospital of Jilin University, Affiliated Hospital of Jiangnan University, The First Affiliated Hospital with Nanjing Medical University, Shanghai Children's Hospital, The Children's Hospital of Zhejiang University School of Medicine, Children's Hospital of Fudan University, Jiangxi Province Children's Hospital, Hunan Children's Hospital, Beijing Children's Hospital, Children's Hospital of Soochow University, Wuxi Women's & Children's Hospital
Responsible party
Sponsor
First posted
Aug 21, 2017
Start date
May 12, 2017
Primary completion
Jun 2030 (estimated)
Completion
Jun 2030 (estimated)
Last update
Aug 15, 2025

Study contacts

Xiaoping Luo
principal investigator · Department of Pediatrics of Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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