A Study of PEG-somatropin Injection to Treat Children of Turner Syndrome

A Phase 2 Study of Pegylated Recombinant Human Growth Hormone Injection to Treat Children of Turner Syndrome

This study aims to explore the optimal dose of pegylated recombinant human growth hormone (PEG-rhGH) injection to treat children of Turner syndrome (TS), preliminarily evaluate its safety and efficacy and provide scientific and reliable evidence for the medication dosage in Phase 3 clinical trial.

Study Overview

• Status: Unknown
• Conditions: Turner Syndrome
• Intervention / Treatment: Biological: PEG-rhGH low dose; Biological: PEG-rhGH high dose; Other: Non-treatment control group

• Study Type: Interventional
• Enrollment (Anticipated): 180
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Beijing, China
    • Recruiting
    • Beijing Children's Hospital,Capital Medical University
    • Contact: Chunxiu Gong, PhD
  • Shanghai, China
    • Recruiting
    • Children's Hospital of Fudan University
  • Shanghai, China
    • Recruiting
    • Shanghai Children's Hospital
    • Contact: Pin Li
  • Shanghai, China
    • Recruiting
    • Xinhua Hospital of Shanghai Jiao Tong University School of Medicine
    • Contact: Jun Ye
  • Jiangsu
    • Nanjing, Jiangsu, China
      • Recruiting
      • The First Affiliated Hospital with Nanjing Medical University
      • Contact: Yuhua Hu
    • Wuxi, Jiangsu, China
      • Recruiting
      • Affiliated Hospital of Jiangnan University
  • Jiangxi
    • Nanchang, Jiangxi, China
      • Recruiting
      • Jiangxi Provincial Children's Hospital
  • Jilin
    • Changchun, Jilin, China
      • Recruiting
      • First Hospital of Jilin University
  • Zhejiang
    • Hangzhou, Zhejiang, China
      • Recruiting
      • The Children's Hospital of Zhejiang University School of Medicine
      • Contact: Junfeng Fu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 18 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Female

Description

Inclusion Criteria:

• Female
• Bone age <12 years
• Karyotype: 45, X; 45, X / 46, XXqi; 45, X / 46, XXr; 45, X / 46, XX; 46, XXqi; 46, XXpi; 45, X / 47, XXX; 46, XXp-; 45, X / 46, XXp-; 46, XXq-; 45X / 46, XXq-; 45, X / 46, XX / 47, XXX, etc. (count 50 or more cells);
• Facial appearance and abnormalities: Patients with at least one of the following signs, which include but are not limited to facial pigmented nevus, short neck, webbed neck, low posterior hairline, low-set ears, micrognathia, high-voulted arch, shield-like chest, cubitus valgus, genu valgum, short 4th and 5th metacarpals, nail dysplasia, scoliosis, ptosis and strabismus, cardiovascular abnormalities (such as aortic stenosis, bicuspid aortic valve and hypertension), reproductive abnormalities (such as primary gonadal dysfunction), renal abnormalities, thyroid hypofunction, middle ear lesion, etc.
• Short stature: height below -2.5SD of the mean height of the same age and gender.
• Pre-pubertal (Tanner Stage I ) patients
• No history of growth hormone treatment
• The subject and his/her guardian sign the informed consent (if the subject is incapable to sign the informed consent, his/her legal guardian shall sign the name of the subject instead)

Exclusion Criteria:

• Subjects with abnormal liver and kidney functions (ALT > upper limit of normal value; Cr > upper limit of normal value)
• Subjects positive for anti-HBc, HbsAg or HbeAg in Hepatitis B virus tests;
• Subjects with highly allergic constitution or allergy to proteins or investigational product or its excipient
• Subjects with systemic chronic disease and immune deficiency
• Patients diagnosed with tumor
• For patients whose tumor markers exceeding normal range in combination with other information, considering as potential high risks of tumor, they may be excluded from the treatment.
• Patients with mental disease
• Subjects with impaired glucose regulation (IGR) (including impaired fasting glucose (IFG) and/or impaired glucose tolerance (IGT) ) or diabetes
• Subjects who took part in other clinical trials within 3 months
• Subjects who received medicines which may interfere GH secretion or GH function, or other hormones within 3 months (such as sex steroids, glucocorticoids, etc.)
• Other conditions which are unsuitable for this study in the opinion of the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: PEG-rhGH low dose; PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.1 mg/kg/w by subcutaneous injection for 52 weeks.	Biological: PEG-rhGH low dose; PEG-rhGH Injection 0.1 mg/kg/w by subcutaneous injection for 52 weeks.; Other Names: Polyethylene Glycol Recombinant Human Somatropin Injection; Biological: PEG-rhGH high dose; PEG-rhGH Injection 0.2 mg/kg/w by subcutaneous injection for 52 weeks.; Other Names: Polyethylene Glycol Recombinant Human Somatropin Injection; Other: Non-treatment control group
EXPERIMENTAL: PEG-rhGH high dose; PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.2 mg/kg/w by subcutaneous injection for 52 weeks.	Biological: PEG-rhGH low dose; PEG-rhGH Injection 0.1 mg/kg/w by subcutaneous injection for 52 weeks.; Other Names: Polyethylene Glycol Recombinant Human Somatropin Injection; Biological: PEG-rhGH high dose; PEG-rhGH Injection 0.2 mg/kg/w by subcutaneous injection for 52 weeks.; Other Names: Polyethylene Glycol Recombinant Human Somatropin Injection; Other: Non-treatment control group
NO_INTERVENTION: Non-treatment control group

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change of height standard deviation score before and after treatment (ΔHT SDS)	The change of height standard deviation score of chronological age before and after treatment	52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Height Velocity	Annual growth rate at the end of treatment	52 weeks
ΔBA/ΔCA	Bone maturation (changes of bone age/ chang of chronological age)	52 weeks
IGF-1(Insulin-like growth factor 1) SDS		52 weeks

Collaborators and Investigators

• Sponsor: GeneScience Pharmaceuticals Co., Ltd.
• Collaborators: The First Affiliated Hospital with Nanjing Medical University; Affiliated Hospital of Jiangnan University; Children's Hospital of Fudan University; Shanghai Children's Hospital; The First Hospital of Jilin University; Xinhua Hospital, Shanghai Jiao Tong University School of Medicine; Beijing Children's Hospital; The Children's Hospital of Zhejiang University School of Medicine; Jiangxi Province Children's Hospital
• Investigators: Principal Investigator: Chunxiu Gong, PhD, Beijing Children's Hospital

Study record dates

Study Major Dates

• Study Start: March 1, 2016
• Primary Completion (ANTICIPATED): March 1, 2018

Study Registration Dates

• First Submitted: June 13, 2017
• First Submitted That Met QC Criteria: June 14, 2017
• First Posted (ACTUAL): June 16, 2017

Study Record Updates

• Last Update Posted (ACTUAL): December 12, 2017
• Last Update Submitted That Met QC Criteria: December 10, 2017
• Last Verified: June 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Heart Diseases; Cardiovascular Diseases; Endocrine System Diseases; Disease; Gonadal Disorders; Disorders of Sex Development; Urogenital Abnormalities; Congenital Abnormalities; Genetic Diseases, Inborn; Heart Defects, Congenital; Cardiovascular Abnormalities; Chromosome Disorders; Sex Chromosome Disorders; Sex Chromosome Disorders of Sex Development; Syndrome; Turner Syndrome; Gonadal Dysgenesis
• Other Study ID Numbers: GenSci 032 CT

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No