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

Primary Purpose

Dwarfism

Status

Active

Phase

Phase 2

Locations

China

Study Type

Interventional

Intervention

PEG-somatropin

About this trial

This is an interventional treatment trial for Dwarfism

Eligibility Criteria

4 Years - 9 Years (Child)All SexesDoes not accept healthy volunteers

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..

Sites / Locations

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

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PEG-somatropin

Arm Description

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.

Outcomes

Primary Outcome Measures

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)

Secondary Outcome Measures

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

Standard deviation score of height at the actual age.

Change fo Bone maturation

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

Change of IGF-1 SDS (ΔIGF-1 SDS)

Change of IGF-1 SDS before and after treatement

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

Changes of standard deviation scores of body mass index

The yearly average dose of PEG-rhGH injection

Final height (FH)

Final height

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

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

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

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

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

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

The changes of the percentage of body fat (optional)

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

Full Information

NCT ID

NCT03255694

First Posted

July 14, 2017

Last Updated

June 21, 2023

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

1. Study Identification

Unique Protocol Identification Number

NCT03255694

Brief Title

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

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.

Study Type

Interventional

2. Study Status

Record Verification Date

February 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

May 12, 2017 (Actual)

Primary Completion Date

June 2030 (Anticipated)

Study Completion Date

June 2030 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Changchun GeneScience Pharmaceutical Co., Ltd.

Collaborators

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Dwarfism

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

360 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

PEG-somatropin

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

PEG-somatropin

Other Intervention Name(s)

Polyethylene Glycol Recombinant Human Somatropin Injection

Intervention Description

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.

Primary Outcome Measure Information:

Title

Change of yearly height velocity (ΔHV)

Description

Time Frame

Baseline，the end of 3-year addendum

Secondary Outcome Measure Information:

Title

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

Description

Standard deviation score of height at the actual age.

Time Frame