The Efficacy and Safety of Inpegsomatropin Injection in Children With Idiopathic Short Stature

Официальное название

A Multicenter, Randomized, Open-Label, Active-Controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of Inpegsomatropin Injection, s.c Once a Week, Compared With Recombinant Human Growth Hormone (rhGH) Injection in Children With Idiopathic Short Stature (ISS).

Обзор

This is a multicenter, randomized, open-label, active-controlled Phase III clinical trial to evaluate the efficacy and safety of Inpegsomatropin injection，once a week，compared with recombinant human growth hormone (rhGH) in children with idiopathic short stature (ISS). It plans to enroll 300 children with ISS, who will be randomized , stratified by gender and age, and assigned to either the experimental group or the positive controlled group. Each participant will undergo a screening period (up to 12 weeks), a treatment period (52 weeks), and a post-treatment follow-up period (5 weeks). And the safety and efficacy will be evaluated.

Вмешательства

• Препарат Inpegsomatropin Injection
  Inpegsomatropin injection, 280 μg/kg/week, s.c., once weekly, for 52 consecutive weeks.
• Препарат Recombinant Human Growth Hormone Injection
  Recombinant Human Growth Hormone Injection, 350 μg/kg/week, s.c., divided into 7 doses, i.e., 50 μg/kg/day (0.15 IU/kg/day), once daily for 52 consecutive weeks.

Первичные конечные точки

• Growth velocity (HV, cm/year). [Срок оценки: Week 52]

Вторичные конечные точки (4)

• Change in height standard deviation score from baseline (△HT SDS) [Срок оценки: From baseline to all follow-up time points at Week 52]
• Change in growth velocity from baseline (△HV) [Срок оценки: From baseline to all follow-up time points at Week 52]
• Change in insulin-like growth factor 1 standard deviation score from baseline (△IGF-1 SDS) [Срок оценки: From baseline to all follow-up time points at Week 52]
• The ratio of the change in bone age to the change in chronological age (△BA/△CA) [Срок оценки: From baseline to all follow-up time points at Week 52]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 38 центров

• Anhui Provincial Children's Hospital — Хэфэй
• The Second Affiliated Hospital of Anhui Medical University — Хэфэй
• Capital Institute of Pediatrics, Beijing (SHOUER) — Пекин
• Children's Hospital of Chongqing Medical University — Чунцин
• The First Affiliated Hospital of Xiamen University — Xiamen
• Gansu Provincial Maternity and Child Care Hospital — Lanzhou
• Guangzhou Women and Children's Medical Center — Гуанчжоу
• Sun Yat-sen Memorial Hospital, Sun Yat-sen University — Гуанчжоу
• … и ещё 30 центров

Идентификаторы

NCT: NCT06927310 · PEGPESEN-4-2-001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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