Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT03255694

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Dwarfism Bone Diseases Bone Diseases, Developmental Congenital, Hereditary, and Neonatal Diseases and Abnormalities Endocrine System Diseases Genetic Diseases, Inborn Musculoskeletal Diseases

Age range

4 year–9 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, Beijing Municipality, China

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

PEG-somatropin

Drug

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

Primary outcomes

  1. Change of yearly height velocity (ΔHV)

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

    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)

Secondary outcomes

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

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

    Standard deviation score of height at the actual age.

  2. Change fo Bone maturation

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

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

  3. Change of IGF-1 SDS (ΔIGF-1 SDS)

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

    Change of IGF-1 SDS before and after treatement

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

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

    Changes of standard deviation scores of body mass index

  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)

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

    Final height

  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)

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

    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

  9. the improvement of PAH

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

    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

  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

Sponsors and collaborators

Lead sponsor

Changchun GeneScience Pharmaceutical Co., Ltd.

Industry

Collaborators

  • Affiliated Hospital of Jiangnan University
  • Beijing Children's Hospital
  • Children's Hospital of Fudan University
  • Children's Hospital of Soochow University
  • Hunan Children's Hospital
  • Jiangxi Province Children's Hospital
  • Shanghai Children's Hospital
  • The Children's Hospital of Zhejiang University School of Medicine
  • The First Affiliated Hospital with Nanjing Medical University
  • The First Hospital of Jilin University
  • Tongji Hospital
  • Wuxi Women's & Children's Hospital

Registry information

Official study title

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.

Important dates

Study start
2017
Primary completion
2030
Study completion
2030
First posted
Aug 21, 2017
Registry last updated
Aug 15, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.