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

Dwarfism Clinical Trial

Official 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.

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03255694
• Other study ID #: GenSci 033 CT-Extension Period
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: May 12, 2017
• Est. completion date: June 2030

Study information

• Verified date: February 2023
• Source: GeneScience Pharmaceuticals Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 360
• Est. completion date: June 2030
• Est. primary completion date: June 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 9 Years

Eligibility

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..

Related Conditions & MeSH terms

• Dwarfism

Intervention

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.

Locations

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

Sponsors (8)

Lead Sponsor	Collaborator
GeneScience Pharmaceuticals Co., Ltd.	Affiliated Hospital of Jiangnan University, Children's Hospital of Fudan University, 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

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change of yearly height velocity (?HV)	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	Standard deviation score of height at the actual age (?HT SDS)	Standard deviation score of height at the actual age.	Baseline,every 3 months,the end of 3-year addendum
Secondary	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
Secondary	Change of IGF-1 SDS (?IGF-1 SDS)	Change of IGF-1 SDS before and after treatement	Baseline,every 3 months,the end of 3-year addendum
Secondary	Changes of standard deviation scores of body mass index (?BMI SDS)	Changes of standard deviation scores of body mass index	Baseline,every 3 months,the end of 3-year addendum
Secondary	The yearly average dose of PEG-rhGH injection		Baseline,every 3 months,the end of 3-year addendum
Secondary	Final height (FH)	Final height	Baseline,every 3 months,the end of 3-year addendum
Secondary	The improvement of FH compared with the baseline predicted adult height (PAH)		Baseline,every 3 months,the end of 3-year addendum
Secondary	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
Secondary	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
Secondary	The changes of the scores evaluated by the Quality of Life Scale		Baseline,every 3 months,the end of 3-year addendum
Secondary	The changes of lean body mass (LBM) (optional)		Baseline,every 3 months,the end of 3-year addendum
Secondary	The changes of fat mass (torso) (FM) (optional)		Baseline,every 3 months,the end of 3-year addendum
Secondary	The changes of the percentage of body fat (optional)		Baseline,every 3 months,the end of 3-year addendum
Secondary	The changes of bone mineral density (BMD) (optional)		Baseline,every 3 months,the end of 3-year addendum