Pediatric Growth Hormone DeficiencyPhase 3NCT04633057
A Study to Evaluating the Efficacy and Safety of Recombinant Long-acting Human Growth Hormone (TJ101) in Children With Growth Hormone Deficiency
A Phase 3 study testing TJ101 and NordiFlex in people with Pediatric Growth Hormone Deficiency, run by TJ Biopharma Co., Ltd. at 30 sites in China.
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. Read more about this study
- Who is it for?
- Ages 3 to 12 years
- Conditions the study is for
- Pediatric Growth Hormone Deficiency
- What is being tested?
- TJ101 and NordiFlex
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 168 participants
- Where
- 30 sitesChina
- Study dates
- 25 Jan 2021to 31 Jul 2023
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
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- 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)
Who cannot join
- 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)]
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- 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
- Subject who has received an investigational product or has participated in a clinical study within 30 days before screening or during the clinical trials.
- Known or suspected to be HIV positive, serologically positive for syphilis, or other chronic infectious diseases, such as AIDS, tuberculosis, hepatitis, etc
- The history of drug, drug or alcohol abuse
- Other conditions not considered suitable for inclusion by the researchers
Trial locations and contacts
30 sites in 1 country.
30 sites in 1 country
China30 sites
Beijing Children's Hospital,Capital Medical University
Beijing, Beijing Municipality
Children's Hospital, Capital Institute of Paediatrics
Beijing, Beijing Municipality
The First Bethune Hospital of Jilin University
Changchun, Jilin
Hunan Children's Hospital
Changsha, Hunan
Chengdu Women's and Children's Central Hospital
Chengdu, Sichuan
West China Second Hospital of Sichuan University
Chengdu, Sichuan
Sun Yat-sen Memorial Hospital, Sun Yat-sen University
Guangzhou, Guangdong
The Third Affiliated Hospital of Sun Yet-sen University
Guangzhou, Guangdong
Second Affiliated Hospital of Anhui Medical University
Hefei, Anhui
The First Affiliated Hospital of Nanhua University
Hengyang, Hunan
Affiliated Hospital of Jining Medical College
Jining, Shandong
Linyi Women and Children's Hospital
Linyi, Shandong
Liuzhou Maternity and Children Healthcare Hospital
Liuzhou, Guangxi
Taizhou First People's Hospital
Liuzhou, Zhejiang
Jiangxi Provincial Children's Hospital
Nanchang, Jiangxi
Children's Hospital of Nanjing Medical University
Nanjing, Jiangsu
Jiangsu Provincial Maternal and Child Health Hospital
Nanjing, Jiangsu
Ningbo Women & Children's Hospital
Ningbo, Zhejiang
Pingxiang Maternity and Child Care Hospital
Pingxiang, Jiangxi
Hainan Third People's Hospital
Sanya, Hainan
Shanghai Children's Medical Center
Shanghai, Shanghai Municipality
Shanghai Jiaotong University School of Medicine Ruijin Hospital
Shanghai, Shanghai Municipality
Shengjing Hospital of China Medical University
Shenyang, Liaoning
Children's Hospital affiliated to Soochow University
Suzhou, Jiangsu
Tangshan Women and Children's Hospital
Tangshan, Hebei
Chongqing Three Gorges Central Hospital
Wanzhou, Chongqing Municipality
Tongji Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology
Wuhan, Hubei, 430030
Wuhan Children's Hospital
Wuhan, Hubei
General Hospital of Ningxia Medical University
Yinchuan, Ningxia
Henan Children's Hospital
Zhengzhou, Henan
About this study
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
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
- Being tested
- TJ101, NordiFlex
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TJ101Drug
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.
NordiFlexDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| TJ101 | Experimental | TJ101 1.2 mg/kg once a week for 52weeks Receives: Drug: TJ101 |
| NordiFlex | Active comparator | NordiFlex Injection 0.034 mg/kg once a day for 52 weeks Receives: Drug: NordiFlex |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Annualized Height velocity at 52 week
The Annualized Height velocity at 52 week
Measured: 52weeks after first dose
Secondary outcomes (2)
change in Annualized Height velocity at 26 week (compared to Baseline value)
Measured: 26weeks after first dose
change in Annualized Height velocity at 52 week (compared to Baseline value)
Measured: 52weeks after first dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2020
- Study start25 Jan 2021
- Primary completion31 Jul 2023
- Study completion31 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric Growth Hormone DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 168 participants (actual)
Sponsor and investigators
- Lead sponsor
- TJ Biopharma Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04633057
- Sponsor's protocol ID
- CTJ101PGHD301
- Status last verified
- Apr 2024
- First submitted
- 12 Nov 2020
- Last updated
- 15 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04633057
- Acronym / sponsor ID
- CTJ101PGHD301
- Conditions
- Pediatric Growth Hormone Deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 168 participants (actual)
- Lead sponsor
- TJ Biopharma Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 25 Jan 2021 (actual)
- Primary completion
- 31 Jul 2023 (actual)
- Study completion
- 31 Jul 2023 (actual)
- First posted
- 18 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| TJ101 | Experimental | Drug: TJ101TJ101 1.2 mg/kg once a week for 52weeks |
| NordiFlex | Active comparator | Drug: NordiFlexNordiFlex Injection 0.034 mg/kg once a day for 52 weeks |
Who may qualify
- Ages
- 3 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- 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);
Key exclusion criteria (19 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TJ101TJ101/ 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. | Drug | TJ101 |
| NordiFlexTJ101/ 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. | Drug | NordiFlex |
Primary outcome
The Annualized Height velocity at 52 week
Time frame: 52weeks after first dose
The Annualized Height velocity at 52 week
2 secondary outcomes
change in Annualized Height velocity at 26 week (compared to Baseline value)
Time frame: 26weeks after first dose
change in Annualized Height velocity at 52 week (compared to Baseline value)
Time frame: 52weeks after first dose
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
30 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing TJ101 and NordiFlex in people with Pediatric Growth Hormone Deficiency, run by TJ Biopharma Co., Ltd. at 30 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Annualized Height velocity at 52 week (time frame: 52weeks after first dose). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Apr 2024.