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

NCT04633057 | Completed Phase 3

• 1 other identifier: CTJ101PGHD301
• Study Type: interventional
• Target: 168
• Locations: 1 country
• Sites: 30

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.

Conditions

Pediatric Growth Hormone Deficiency

Outcome Measures

Primary Outcomes (1)

• The Annualized Height velocity at 52 week

  The Annualized Height velocity at 52 week

  52weeks after first dose

Secondary Outcomes (2)

• 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

Study Arms (2)

TJ101

EXPERIMENTAL

TJ101 1.2 mg/kg once a week for 52weeks

Drug: TJ101

NordiFlex

ACTIVE COMPARATOR

NordiFlex Injection 0.034 mg/kg once a day for 52 weeks

Drug: NordiFlex

Interventions

TJ101 DRUG

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.

TJ101

NordiFlex DRUG

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.

NordiFlex

Eligibility Criteria

• Age: 3 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Boys: 3 years ≤ boy's age ≤ 10 years；Girls: 3 years ≤ girl's age ≤ 9 years
• Pre-pubertal children(Tanner stage I)
• GHD confirmed by 2 different GH provocation tests with peak GH concentration below 10 ng/mL in screening or one month
• 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)
• Height velocity≤5.0cm/years (Including height records before at least three months)；
• IGF-1 SDS≤-1.0
• Bone age (BA) is no more than two chronological age
• 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
• Without prior exposure to any rhGH therapy
• 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
• Written informed consent of the parent or legal guardian of the subject and assent of the subject (if the subject can read)

You may not qualify if:

• beyond physiological dosage of glucocorticoid therapy
• Evidence of closed epiphyses
• 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)
• Abnormal liver and renal function (ALT\>1.5 times the upper limit of normal range and Cr exceeding the upper limit of normal range)
• Presence of anti-hGH antibodies at screening
• 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 \[thyroxine, hydrocortisone, desmopressin (DDAVP)\]
• Mutations in growth hormone receptors are suspected, or any syndrome that causes insensitivity to growth hormone
• At screening, ophthalmologic examination (including fundus microscopy) indicated increased intracranial pressure and/or retinopathy.
• 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)
• Diseases such as severe cardiopulmonary, blood system, malignant tumor or potential tumor (family history), or systemic infection, low immune function and mental diseases
• Significant spinal abnormalities, including scoliosis (Cobb Angle \& GT;60 ˚), kyphosis and spina bifida.
• 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
• 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
• 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)
• The subject and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct

Sponsors & Collaborators

• TJ Biopharma Co., Ltd.: lead

Study Sites (30)

Second Affiliated Hospital of Anhui Medical University

Hefei, Anhui, China

Location

Beijing Children's Hospital,Capital Medical University

Beijing, Beijing Municipality, China

Location

Children's Hospital, Capital Institute of Paediatrics

Beijing, Beijing Municipality, China

Location

Chongqing Three Gorges Central Hospital

Wanzhou, Chongqing Municipality, China

Location

Sun Yat-sen Memorial Hospital, Sun Yat-sen University

Guangzhou, Guangdong, China

Location

The Third Affiliated Hospital of Sun Yet-sen University

Guangzhou, Guangdong, China

Location

Liuzhou Maternity and Children Healthcare Hospital

Liuchow, Guangxi, China

Location

Hainan Third People's Hospital

Sanya, Hainan, China

Location

Tangshan Women and Children's Hospital

Tangshan, Hebei, China

Location

Henan Children's Hospital

Zhengzhou, Henan, China

Location

Tongji Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology

Wuhan, Hubei, 430030, China

Location

Wuhan Children's Hospital

Wuhan, Hubei, China

Location

Hunan Children's Hospital

Changsha, Hunan, China

Location

The First Affiliated Hospital of Nanhua University

Hengyang, Hunan, China

Location

Children's Hospital of Nanjing Medical University

Nanjing, Jiangsu, China

Location

Jiangsu Provincial Maternal and Child Health Hospital

Nanjing, Jiangsu, China

Location

Children's Hospital affiliated to Soochow University

Suzhou, Jiangsu, China

Location

Jiangxi Provincial Children's Hospital

Nanchang, Jiangxi, China

Location

Pingxiang Maternity and Child Care Hospital

Pingxiang, Jiangxi, China

Location

The First Bethune Hospital of Jilin University

Changchun, Jilin, China

Location

Shengjing Hospital of China Medical University

Shenyang, Liaoning, China

Location

General Hospital of Ningxia Medical University

Yinchuan, Ningxia, China

Location

Affiliated Hospital of Jining Medical College

Jining, Shandong, China

Location

Linyi Women and Children's Hospital

Linyi, Shandong, China

Location

Shanghai Children's Medical Center

Shanghai, Shanghai Municipality, China

Location

Shanghai Jiaotong University School of Medicine Ruijin Hospital

Shanghai, Shanghai Municipality, China

Location

Chengdu Women's and Children's Central Hospital

Chengdu, Sichuan, China

Location

West China Second Hospital of Sichuan University

Chengdu, Sichuan, China

Location

Taizhou First People's Hospital

Liuzhou, Zhejiang, China

Location

Ningbo Women & Children's Hospital

Ningbo, Zhejiang, China

Location

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 12, 2020
• First Posted: November 18, 2020
• Study Start: January 25, 2021
• Primary Completion: July 31, 2023
• Study Completion: July 31, 2023
• Last Updated: April 15, 2024

Record last verified: 2024-04

Locations

CN (30)