Once-weekly inpegsomatropin versus daily recombinant growth hormone for children with idiopathic short stature

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a multicenter, randomized, open-label, active-controlled Phase III trial enrolling about 300 prepubertal children with idiopathic short stature. Participants are stratified by sex and age and randomized to receive either once-weekly inpegsomatropin or standard daily recombinant human growth hormone. After a screening period (up to 12 weeks), participants receive 52 weeks of treatment followed by a 5-week safety follow-up. The trial compares growth and safety outcomes between the two treatments to determine if the weekly regimen provides comparable efficacy and tolerability to daily injections.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Prepubertal children: boys aged ≥3 years and \<11 years at screening with testicular volume \<4 mL; girls aged ≥3 years and \<10 years at screening with breast development at Tanner Stage I, i.e., no palpable breast glandular tissue.
* Compared to chronological age, bone age is advanced by no more than 1 year or delayed by no more than 2 years (i.e., -2 years ≤ bone age - chronological age ≤ 1 year).
* Height at screening is below -2 standard deviations (SD) from the mean for age and sex, with height reference to Appendix 1.
* Body mass index (BMI) is within the 5th to 95th percentile for age and sex of healthy children, with reference to Appendix 2.
* Peak GH level ≥10.0 ng/ml in at least one GH stimulation test.
* No prior systemic pharmacological treatment for growth promotion (continuous use ≥1 month), including but not limited to growth hormone, insulin-like growth factor 1 (IGF-1), etc.
* The legal guardian understands and signs the informed consent form. If the participants is ≥8 years old, they must also sign the informed consent form. For participants under 8 years old who are able to express their consent, their assent should be clearly documented.

Exclusion Criteria:

* Individuals with closed epiphyses;
* Other types of growth and developmental abnormalities, including confirmed or highly suspected growth hormone deficiency (GHD), Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, Turner syndrome, small for gestational age, short stature due to SHOX gene abnormalities, growth delay due to malnutrition, growth delay due to hypothyroidism, and other short statures with a clear etiology; genetic testing results must be obtained before randomization to exclude short stature caused by definite genetic abnormalities;
* Individuals who have participated in any other clinical trial within 3 months prior to screening and have received pharmacological or non-pharmacological interventions;
* Individuals who received inhaled corticosteroids for more than 2 consecutive weeks, or oral or intravenous corticosteroids for more than 1 consecutive week, within the 3 months prior to screening;
* Individuals who are currently receiving or require long-term treatment with other therapies that may affect growth, including but not limited to methylphenidate, sex hormones, gonadotropin-releasing hormone analogs, aromatase inhibitors, anabolic agents, insulin, etc.;
* Individuals with abnormal liver or kidney function at screening (ALT \> 1.5 times the upper limit of normal, Cr \> upper limit of normal);
* Individuals diagnosed with diabetes mellitus, or with fasting blood glucose ≥6.1 mmol/L on two consecutive occasions;
* Individuals with chronic infectious diseases, such as chronic hepatitis B;
* Individuals with systemic chronic diseases, such as moderate to severe anemia, malnutrition, hypothyroidism, chronic kidney disease, cardiovascular diseases (e.g., dilated cardiomyopathy, etc.), psychiatric and psychological disorders, or those with congenital anomalies requiring clinical intervention as determined by the investigator;
* Individuals with congenital skeletal abnormalities, or those with scoliosis exceeding 15°, limping, or a history of slipped capital femoral epiphysis;
* Individuals with a history of increased intracranial pressure;
* Individuals with a history of malignancy or currently having active malignancy, including intracranial tumors;
* Individuals with severe allergic constitutions, or known allergies to growth hormone or its excipients;
* Individuals with any other disease that the investigator deems may endanger the subject's safety or compromise compliance with the study protocol.

Where it's running

• Anhui Provincial Children's Hospital
  Hefei, Anhui, China Recruiting
• The Second Affiliated Hospital of Anhui Medical University
  Hefei, Anhui, China Recruiting
• Capital Institute of Pediatrics, Beijing (SHOUER)
  Beijing, Beijing Municipality, China Recruiting
• Children's Hospital of Chongqing Medical University
  Chongqing, Chongqing Municipality, China Recruiting
• The First Affiliated Hospital of Xiamen University
  Xiamen, Fujian, China Recruiting
• Gansu Provincial Maternity and Child Care Hospital
  Lanzhou, Gansu, China Recruiting

Study contacts

• Principal investigator: Xiaoping Luo, Ph.D — Tongji Hospital
• Study coordinator: Xiaoping Luo, Ph.D
• Email: xpluo@tjh.tjmu.edu.cn
• Phone: 15671671188

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Long-term Inpegsomatropin Treatment for Idiopathic Short Stature: Extension of Phase III Study

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• Evaluating Vosoritide for Children with Short Stature

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• Assessing Growth in Children with Short Stature

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• Multidisciplinary evaluation and whole-genome testing for children with unexplained short stature

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• Evaluating the long-term safety and effectiveness of Growtropin®-II in children with short stature

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.