A Study to Evaluating the Efficacy and Safety of Recombinant Long-acting Human Growth Hormone (TJ101) in Children With Growth Hormone Deficiency

Pediatric Growth Hormone Deficiency Clinical Trial

Official title:

A Phase III, Randomized, Open-label, Positive-controlled, Multi-center Study to Evaluating the Efficacy and Safety of Recombinant Long-acting Human Growth Hormone (TJ101) in Children With Growth Hormone Deficiency

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04633057
• Other study ID #: CTJ101PGHD301
• Status: Completed
• Phase: Phase 3
• Start date: January 25, 2021
• Est. completion date: July 31, 2023

Study information

• Verified date: April 2024
• Source: TJ Biopharma Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A Phase III, Randomized, open-label, positive-drug parallel control, Study to Evaluate the Efficacy and Safety of TJ101 in Child subject with growth hormone deficieney.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 168
• Est. completion date: July 31, 2023
• Est. primary completion date: July 31, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 12 Years

Eligibility

Inclusion Criteria:

1. Boys: 3 years = boy's age = 10 years;Girls: 3 years = girl's age = 9 years

2. Pre-pubertal children(Tanner stage I)

3. GHD confirmed by 2 different GH provocation tests with peak GH concentration below 10 ng/mL in screening or one month

4. Height (HT) of at least 2.0 standard deviations (SD) below the mean height for chronological age (CA) and gender according to Table of standard deviation unit values of age and height of Chinese children aged 3-12 years , (HT SDS = -2.0)

5. Height velocity=5.0cm/years (Including height records before at least three months);

6. IGF-1 SDS=-1.0

7. Bone age (BA) is no more than two chronological age

8. Body mass Index (BMI) must be within ±2 SD of mean BMI for the chronological age and sex according to Table of standard deviation unit values of Age and BMI of Chinese children aged 3-12 years

9. Without prior exposure to any rhGH therapy

10. For children with growth hormone deficiency that is one of its multiple pituitary hormone deficiencies, alternative therapies targeting the hypothalamus-pituitary-target gland axis must be used for at least 3 months before screening

11. Written informed consent of the parent or legal guardian of the subject and assent of the subject (if the subject can read)

Exclusion Criteria:

1. beyond physiological dosage of glucocorticoid therapy

2. Evidence of closed epiphyses

3. Any other chronic condition that can cause short stature and cannot be treated with hormone replacement therapy(Including but not limited Chronic kidney disease, malnutrition, absorption disorders, uncontrolled hypothyroidism, celiac disease, rickets and social-psychological dwarfism)

4. Abnormal liver and renal function (ALT>1.5 times the upper limit of normal range and Cr exceeding the upper limit of normal range)

5. Presence of anti-hGH antibodies at screening

6. Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids, with the exception of ADHD(attention deficit hyperactivity disorder) drug hormone replacement therapies [thyroxine, hydrocortisone, desmopressin (DDAVP)]

7. Mutations in growth hormone receptors are suspected, or any syndrome that causes insensitivity to growth hormone

8. At screening, ophthalmologic examination (including fundus microscopy) indicated increased intracranial pressure and/or retinopathy.

9. At screening, previous or existing intracranial tumor growth was confirmed by cranial magnetic resonance imaging (MRI) scan (using contrast agent) (MRI results up to 1 year prior to screening were acceptable)

10. Diseases such as severe cardiopulmonary, blood system, malignant tumor or potential tumor (family history), or systemic infection, low immune function and mental diseases

11. Significant spinal abnormalities, including scoliosis (Cobb Angle & GT;60 °), kyphosis and spina bifida.

12. Subjects diagnosed with type 2 or type 1 diabetes who were considered to have received no standard treatment, did not follow their prescribed treatment, or exhibited poor metabolic control, or had fasting glucose > 5.6 mmol/L twice in a row

13. Chromosomal abnormalities and medical syndromes (Turner's syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, SHOX mutations/deletions and skeletal dysplasias), with the exception of septo-optic dysplasia

14. Children with low birth weight (birth weight and/or body length are 2 SD below average according to the standard of the general Chinese population of the same gestational age and sex)

15. The subject and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct

16. Subject who has received an investigational product or has participated in a clinical study within 30 days before screening or during the clinical trials.

17. Known or suspected to be HIV positive, serologically positive for syphilis, or other chronic infectious diseases, such as AIDS, tuberculosis, hepatitis, etc

18. The history of drug, drug or alcohol abuse

19. Other conditions not considered suitable for inclusion by the researchers

Related Conditions & MeSH terms

• Dwarfism, Pituitary
• Endocrine System Diseases
• Pediatric Growth Hormone Deficiency

Intervention

Drug:
• TJ101
TJ101/ Recombinant Human Somatropin Injection treatment: after screening, subjects will be randomized to receive TJ101 1.2 mg/kg once a week until 52 weeks back site, investigator evaluate effectiveness and safety.
• NordiFlex
TJ101/ Recombinant Human Somatropin Injection treatment: after screening, subjects will be randomized to receive Recombinant Human Somatropin Injection 0.034 mg/kg once a day, until 52 weeks back site, investigator evaluate effectiveness and safety.

Locations

Country	Name	City	State
China	Beijing Children's Hospital,Capital Medical University	Beijing	Beijing
China	Children's Hospital, Capital Institute of Paediatrics	Beijing	Beijing
China	The First Bethune Hospital of Jilin University	Changchun	Jilin
China	Hunan Children's Hospital	Changsha	Hunan
China	Chengdu Women's and Children's Central Hospital	Chengdu	Sichuan
China	West China Second Hospital of Sichuan University	Chengdu	Sichuan
China	Sun Yat-sen Memorial Hospital, Sun Yat-sen University	Guangzhou	Guangdong
China	The Third Affiliated Hospital of Sun Yet-sen University	Guangzhou	Guangdong
China	Second Affiliated Hospital of Anhui Medical University	Hefei	Anhui
China	The First Affiliated Hospital of Nanhua University	Hengyang	Hunan
China	Affiliated Hospital of Jining Medical College	Jining	Shandong
China	Linyi Women and Children's Hospital	Linyi	Shandong
China	Liuzhou Maternity and Children Healthcare Hospital	Liuzhou	Guangxi
China	Taizhou First People's Hospital	Liuzhou	Zhejiang
China	Jiangxi Provincial Children's Hospital	Nanchang	Jiangxi
China	Children's Hospital of Nanjing Medical University	Nanjing	Jiangsu
China	Jiangsu Provincial Maternal and Child Health Hospital	Nanjing	Jiangsu
China	Ningbo Women & Children's Hospital	Ningbo	Zhejiang
China	Pingxiang Maternity and Child Care Hospital	Pingxiang	Jiangxi
China	Hainan Third People's Hospital	Sanya	Hainan
China	Shanghai Children's Medical Center	Shanghai	Shanghai
China	Shanghai Jiaotong University School of Medicine Ruijin Hospital	Shanghai	Shanghai
China	Shengjing Hospital of China Medical University	Shenyang	Liaoning
China	Children's Hospital affiliated to Soochow University	Suzhou	Jiangsu
China	Tangshan Women and Children's Hospital	Tangshan	Hebei
China	Chongqing Three Gorges Central Hospital	Wanzhou	Chongqing
China	Tongji Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology	Wuhan	Hubei
China	Wuhan Children's Hospital	Wuhan	Hubei
China	General Hospital of Ningxia Medical University	Yinchuan	Ningxia
China	Henan Children's Hospital	Zhengzhou	Henan

Sponsors (1)

Lead Sponsor	Collaborator
TJ Biopharma Co., Ltd.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The Annualized Height velocity at 52 week	The Annualized Height velocity at 52 week	52weeks after first dose
Secondary	change in Annualized Height velocity at 26 week (compared to Baseline value)		26weeks after first dose
Secondary	change in Annualized Height velocity at 52 week (compared to Baseline value)		52weeks after first dose