Clinical trial NCT03255694: 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Dwarfism
Lead sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Tongji Hospital
name: The First Hospital of Jilin University
name: Affiliated Hospital of Jiangnan University
name: The First Affiliated Hospital with Nanjing Medical University
name: Shanghai Children's Hospital
name: The Children's Hospital of Zhejiang University School of Medicine
name: Children's Hospital of Fudan University
First posted: 2017-08-21
Enrollment: 360
Sex: ALL
Minimum age (years): 4
Maximum age (years): 9
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.
* 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..

Primary outcomes:
measure: Change of yearly height velocity (ΔHV)
timeFrame: Baseline，the end of 3-year addendum
Secondary outcomes:
measure: Standard deviation score of height at the actual age (ΔHT SDS)
timeFrame: Baseline，every 3 months，the end of 3-year addendum
measure: Change fo Bone maturation
timeFrame: Baseline，every 3 months，the end of 3-year addendum
measure: Change of IGF-1 SDS (ΔIGF-1 SDS)
timeFrame: Baseline，every 3 months，the end of 3-year addendum
measure: Changes of standard deviation scores of body mass index (ΔBMI SDS)
timeFrame: Baseline，every 3 months，the end of 3-year addendum
measure: The yearly average dose of PEG-rhGH injection
timeFrame: Baseline，every 3 months，the end of 3-year addendum
measure: Final height (FH)
timeFrame: Baseline，every 3 months，the end of 3-year addendum
measure: The improvement of FH compared with the baseline predicted adult height (PAH)
timeFrame: Baseline，every 3 months，the end of 3-year addendum
measure: Improvement of NAH (near adult height)
timeFrame: Baseline，every 3 months，the end of 3-year addendum
measure: the improvement of PAH
timeFrame: Baseline，every 3 months，the end of 3-year addendum
measure: The changes of the scores evaluated by the Quality of Life Scale
timeFrame: Baseline，every 3 months，the end of 3-year addendum
measure: The changes of lean body mass (LBM) (optional)
timeFrame: Baseline，every 3 months，the end of 3-year addendum
measure: The changes of fat mass (torso) (FM) (optional)
timeFrame: Baseline，every 3 months，the end of 3-year addendum
measure: The changes of the percentage of body fat (optional)
timeFrame: Baseline，every 3 months，the end of 3-year addendum
measure: The changes of bone mineral density (BMD) (optional)
timeFrame: Baseline，every 3 months，the end of 3-year addendum
Other outcomes:
Locations:
city: Wuhan
country: China
facility: Department of Pediatrics of Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology, Wuhan
state: Hubei
city: Nanjing
country: China
facility: The First Affiated Hospital of Nanjing Medical Universit
state: Jiangsu
city: Wuxi
country: China
facility: Affiliated Hospital of Jiangnan University
state: Jiangsu
city: Changchun
country: China
facility: The First Hospital of Jilin University
state: Jilin
city: Hangzhou
country: China
facility: The Children's Hospital of Zhejiang University School of Medicine
state: Zhejiang
city: Shanghai
country: China
facility: Shanghai Children's Hospital of Fudan University
city: Shanghai
country: China
facility: Shanghai Children's Hospital
Study officials:
affiliation: Department of Pediatrics of Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology
name: Xiaoping Luo
role: PRINCIPAL_INVESTIGATOR
References: