Clinical trial: A Study to Evaluating the Efficacy and Safety of Recombinant Long-acting Human Growth Hormone (TJ101) in Children With Growth Hormone Deficiency (NCT04633057)

Eligibility criteria:
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
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 3 Years old.
Subject must be at most 10 Years

Patient description (medical language): A 9-year-old girl is brought to the office for evaluation of short stature and overweight body habitus. The patient's mother and father are 170 cm and 181 cm tall, respectively.  On physical examination, the patient's height is in the 5th percentile of her age.  Other findings include low-set ears, a high arched palate, a webbed neck, and cubitus valgus.  Chromosomal analysis reveals a 45, XO karyotype.

Patient description (patient's own words): I took my 9-year-old daughter to the doctor because my mother-in-law kept saying she seemed small and overweight. My husband and I are 170 cm and 181 cm tall, respectively. The physical health highlighted that she is in the 5th percentile of her age. The doctor said that she has low-set ears, a high-arched palate, a webbed neck, and cubitus valgus. She also did a chromosomal analysis, which revealed a 45, XO karyotype.

Does the patient match the trial criteria? 0