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

Phase 3

Completed

• Conditions: Pediatric Growth Hormone Deficiency
• Interventions: Drug: NordiFlex; Drug: TJ101

• Registration Number: NCT04633057
• Lead Sponsor: TJ Biopharma Co., Ltd.

Brief 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.

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations
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Related News

Study Timeline

• Study First Submitted: 2020-11-12
• Study First Submitted QC: 2020-11-16
• Study First Posted: 2020-11-18
• Study Start: 2021-01-25
• Primary Completion: 2023-07-31
• Study Completion: 2023-07-31
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-11
• Last Update Posted: 2024-04-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 168

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)

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
NordiFlex	NordiFlex	NordiFlex Injection 0.034 mg/kg once a day for 52 weeks
TJ101	TJ101	TJ101 1.2 mg/kg once a week for 52weeks

Primary Outcome Measures

Name	Time	Method
The Annualized Height velocity at 52 week	52weeks after first dose	The Annualized Height velocity at 52 week

Secondary Outcome Measures

Name	Time	Method
change in Annualized Height velocity at 26 week (compared to Baseline value)	26weeks after first dose
change in Annualized Height velocity at 52 week (compared to Baseline value)	52weeks after first dose

Trial Locations

Locations (30)

Beijing Children's Hospital,Capital Medical University; 🇨🇳 Beijing, Beijing, China

Henan Children's Hospital; 🇨🇳 Zhengzhou, Henan, China

Pingxiang Maternity and Child Care Hospital; 🇨🇳 Pingxiang, Jiangxi, China

Tongji Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology; 🇨🇳 Wuhan, Hubei, China

Children's Hospital affiliated to Soochow University; 🇨🇳 Suzhou, Jiangsu, China

Jiangsu Provincial Maternal and Child Health Hospital; 🇨🇳 Nanjing, Jiangsu, China

Chongqing Three Gorges Central Hospital; 🇨🇳 Wanzhou, Chongqing, China

Liuzhou Maternity and Children Healthcare Hospital; 🇨🇳 Liuzhou, Guangxi, China

Second Affiliated Hospital of Anhui Medical University; 🇨🇳 Hefei, Anhui, China

Children's Hospital, Capital Institute of Paediatrics; 🇨🇳 Beijing, Beijing, 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

Hainan Third People's Hospital; 🇨🇳 Sanya, Hainan, China

Tangshan Women and Children's Hospital; 🇨🇳 Tangshan, Hebei, China

Hunan Children's Hospital; 🇨🇳 Changsha, Hunan, China

Wuhan Children's Hospital; 🇨🇳 Wuhan, Hubei, China

The First Affiliated Hospital of Nanhua University; 🇨🇳 Hengyang, Hunan, China

Jiangxi Provincial Children's Hospital; 🇨🇳 Nanchang, Jiangxi, China

The First Bethune Hospital of Jilin University; 🇨🇳 Changchun, Jilin, China

Children's Hospital of Nanjing Medical University; 🇨🇳 Nanjing, Jiangsu, China

Affiliated Hospital of Jining Medical College; 🇨🇳 Jining, Shandong, China

Linyi Women and Children's Hospital; 🇨🇳 Linyi, Shandong, China

Shengjing Hospital of China Medical University; 🇨🇳 Shenyang, Liaoning, China

General Hospital of Ningxia Medical University; 🇨🇳 Yinchuan, Ningxia, China

Chengdu Women's and Children's Central Hospital; 🇨🇳 Chengdu, Sichuan, China

West China Second Hospital of Sichuan University; 🇨🇳 Chengdu, Sichuan, China

Taizhou First People's Hospital; 🇨🇳 Liuzhou, Zhejiang, China

Ningbo Women & Children's Hospital; 🇨🇳 Ningbo, Zhejiang, China

Shanghai Jiaotong University School of Medicine Ruijin Hospital; 🇨🇳 Shanghai, Shanghai, China

Shanghai Children's Medical Center; 🇨🇳 Shanghai, Shanghai, China