Clinical trial NCT03189160: A Study of PEG-somatropin Injection to Treat Children of Turner Syndrome
Official title: A Phase 2 Study of Pegylated Recombinant Human Growth Hormone Injection to Treat Children of Turner Syndrome
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Turner Syndrome
Lead sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Beijing Children's Hospital
name: The First Affiliated Hospital with Nanjing Medical University
name: Shanghai Children's Hospital
name: Children's Hospital of Fudan University
name: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
name: The First Hospital of Jilin University
name: Jiangxi Province Children's Hospital
name: Affiliated Hospital of Jiangnan University
name: The Children's Hospital of Zhejiang University School of Medicine
First posted: 2017-06-16
Enrollment: 180
Sex: FEMALE
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Change of height standard deviation score before and after treatment (ΔHT SDS)
timeFrame: 52 weeks
Secondary outcomes:
measure: Height Velocity
timeFrame: 52 weeks
measure: ΔBA/ΔCA
timeFrame: 52 weeks
measure: IGF-1(Insulin-like growth factor 1) SDS
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Nanjing
country: China
facility: The First Affiliated Hospital with Nanjing Medical University
state: Jiangsu
city: Wuxi
country: China
facility: Affiliated Hospital of Jiangnan University
state: Jiangsu
city: Nanchang
country: China
facility: Jiangxi Provincial Children's Hospital
state: Jiangxi
city: Changchun
country: China
facility: 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: Beijing
country: China
facility: Beijing Children's Hospital,Capital Medical University
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
city: Shanghai
country: China
facility: Shanghai Children's Hospital
city: Shanghai
country: China
facility: Xinhua Hospital of Shanghai Jiao Tong University School of Medicine
Study officials:
affiliation: Beijing Children's Hospital
name: Chunxiu Gong, PhD
role: PRINCIPAL_INVESTIGATOR
References: