A Study Extension Period of PEG-somatropin (Pegylated-somatropin) in the Treatment of Children With Idiopathic Short Stature
Study Details
Study Description
Brief 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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2 |
Study Design
Arms and Interventions
| 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:
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Outcome Measures
Primary Outcome Measures
- Change of yearly height velocity (ΔHV) [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 Outcome Measures
- Standard deviation score of height at the actual age (ΔHT SDS) [Baseline，every 3 months，the end of 3-year addendum]
Standard deviation score of height at the actual age.
- Change fo Bone maturation [Baseline，every 3 months，the end of 3-year addendum]
Change fo Bone maturation before and after treatement (bone age/chronological age)
- Change of IGF-1 SDS (ΔIGF-1 SDS) [Baseline，every 3 months，the end of 3-year addendum]
Change of IGF-1 SDS before and after treatement
- Changes of standard deviation scores of body mass index (ΔBMI SDS) [Baseline，every 3 months，the end of 3-year addendum]
Changes of standard deviation scores of body mass index
- The yearly average dose of PEG-rhGH injection [Baseline，every 3 months，the end of 3-year addendum]
- Final height (FH) [Baseline，every 3 months，the end of 3-year addendum]
Final height
- 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) [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
- the improvement of PAH [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
- 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]
Eligibility Criteria
Criteria
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.
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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;
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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;
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Protein anabolic drugs (which include but are not limited to Oxandrolone, Danazol and Strombafort), with continuous medication ≥1 month;
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Glucocorticoids via oral/intravenous administration for more than 1 month..
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Department of Pediatrics of Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology, Wuhan | Wuhan | Hubei | China | |
| 2 | The First Affiated Hospital of Nanjing Medical Universit | Nanjing | Jiangsu | China | |
| 3 | Affiliated Hospital of Jiangnan University | Wuxi | Jiangsu | China | |
| 4 | The First Hospital of Jilin University | Changchun | Jilin | China | |
| 5 | The Children's Hospital of Zhejiang University School of Medicine | Hangzhou | Zhejiang | China | |
| 6 | Shanghai Children's Hospital of Fudan University | Shanghai | China | ||
| 7 | Shanghai Children's Hospital | Shanghai | China |
Sponsors and Collaborators
- GeneScience Pharmaceuticals Co., Ltd.
- 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
Investigators
- Principal Investigator: Xiaoping Luo, Department of Pediatrics of Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- GenSci 033 CT-Extension Period