The Efficacy and Safety of Inpegsomatropin Injection in Children With Short Stature Born Small for Gestational Age

Official title

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 Short Stature Born Small for Gestational Age（SGA）

Overview

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 short stature born small for gestational age (SGA).It plans to enroll 141 children with short stature born small for gestational age (SGA), who will be stratified by gender and age and randomized in a 1:1:1 ratio to either Experimental Group 1, Experimental Group 2, or the Active Control Group. All participants will undergo a screening period (up to 12 weeks), a treatment period (52 weeks), and a post-treatment follow-up period (5 weeks). Safety and efficacy will be comprehensively evaluated.

Interventions

• Drug Inpegsomatropin Injection
  Inpegsomatropin injection, 140 μg/kg/week, s.c., once weekly, for 52 weeks.
• Drug Inpegsomatropin Injection
  Inpegsomatropin injection, 280 μg/kg/week, s.c., once weekly, for 52 weeks.
• Drug Recombinant Human Growth Hormone Injection
  Recombinant human growth hormone injection, 0.033mg/kg/day, s.c.,once daily, for 52 weeks.

Primary outcome measures

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

Secondary outcome measures (4)

• Change in height standard deviation score from baseline (△HT SDS) [Time frame: From baseline to all follow-up time points at Week 52]
• Change in growth velocity from baseline (△HV) [Time frame: 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) [Time frame: From baseline to all follow-up time points at Week 52]
• Ratio of the change in bone age to the change in chronological age [Time frame: Week 52]

Eligibility criteria

Inclusion criteria

• Prepubertal (Tanner stage I) children: boys aged ≥2 and <11 years, and girls aged ≥2 and <10 years at screening.
• Bone age is not more than 1 year advanced or more than 2 years delayed compared to chronological age (i.e., -2 years ≤ bone age - chronological age ≤ 1 year).
• Birth weight and/or length below the 10th percentile for gestational age and sex, according to the reference values in Appendix 1.
• Gestational age at birth ≥28 weeks.
• Height at screening below -2 SD for age and sex, according to the reference values in Appendix 2.
• Body mass index (BMI) between the 5th and 95th percentiles for age and sex, according to the reference values in Appendix 3.
• Peak GH level ≥10.0 ng/mL in at least one prior GH stimulation test.
• No prior systemic growth-promoting therapy (used continuously for ≥1 month), including but not limited to growth hormone, insulin-like growth factor-1 (IGF-1), etc.
• Legal guardian has provided written informed consent. If the participant is ≥8 years old, they must also provide written assent. For participants under 8 years old who are capable of expressing agreement, their assent should be formally documented.

Exclusion criteria

• Subjects with closed epiphyses.
• Subjects with other types of growth abnormalities, including confirmed or highly suspected growth hormone deficiency (GHD), Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, Turner syndrome, short stature due to GH receptor deficiency, short stature due to growth plate-related gene abnormalities (e.g., SHOX gene anomalies), growth retardation due to malnutrition, or growth retardation due to hypothyroidism.
• Participation in any other clinical trial with drug or non-drug interventions within 3 months prior to screening.
• Use of inhaled corticosteroids for more than 2 consecutive weeks, or oral/intravenous corticosteroids for more than 1 consecutive week, within 3 months prior to screening.
• Current or long-term requirement for therapies that may affect growth, including but not limited to methylphenidate, sex hormones, gonadotropin-releasing hormone analogs, aromatase inhibitors, anabolic agents, or insulin.
• Abnormal liver or kidney function at screening (ALT > 1.5 times the upper limit of normal \[ULN\], Cr > ULN).
• Diagnosis of diabetes mellitus, or two consecutive fasting blood glucose measurements ≥ 6.1 mmol/L prior to randomization.
• Chronic infectious diseases which, in the investigator's judgment, may affect study participation (e.g., chronic hepatitis B).
• Systemic chronic diseases, such as chronic kidney disease, severe cardiovascular diseases, or psychiatric/psychological disorders.
• Congenital skeletal dysplasia, scoliosis exceeding 15°, limping gait, or a prior diagnosis of slipped capital femoral epiphysis.
• History of intracranial hypertension.
• Past or current history of malignant tumors, including intracranial tumors.
• Known allergy to growth hormone or any of its excipients.
• Any other condition deemed by the investigator as inappropriate for participation in this clinical trial.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Open label

Primary purpose

Treatment

Study locations

China · 49 centers

• Beijing Children's Hospital, Capital Medical University — Beijing
• Beijing Children's Hospital, Capital Medical University — Beijing
• Peking Union Medical College Hospital, Chinese Academy of Medical Sciences — Beijing
• The First Hospital of Jilin University — Changchun
• Hunan Children's Hospital — Changsha
• Chengdu Women's and Children's Central Hospital — Chengdu
• West China Second University Hospital, Sichuan University — Chengdu
• Children's Hospital of Chongqing Medical University — Chongqing
• … and 41 more centers

Identifiers

NCT: NCT07309562 · PEGPESEN-4-2-002

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗