A Study of GenSci134 in Children With Growth Hormone Deficiency (PGHD) (NCT07450001) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
A Study of GenSci134 in Children With Growth Hormone Deficiency (PGHD)
China128 participantsStarted 2026-03-06
Plain-language summary
This study comprises two phases: Phase Ib and Phase II. Phase Ib is a multicenter, randomized, open-label, active-controlled, single-dose, dose-escalation to assess safety, tolerability, PK/PD profile, and immunogenicity of GenSci134 in children with GHD.
Phase II is a multicenter, randomized, open-label, active-controlled, multiple-dose, parallel-group study to assess the efficacy and safety of multiple subcutaneous doses of GenSci134 at different levels versus Norditropin® in children with GHD. It will also evaluate PK/PD profile and immunogenicity to support dose selection for Phase III.
Who can participate
Age range3 Years – 12 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Informed consent of parent or legal representative of participant and child assent, as age appropriate must be obtained before any study-related activities.
✓. At the time of signing the Informed consent form (ICF), the following conditions must be met:
✓. BMI within the range of ±2 SD of the mean BMI for age and sex at screening
✓. Height is at least 2 SD below the mean HT for age and sex (HT SDS ≤ -2.0) at screening according to the normal pediatric population standards
✓. Diagnosis of GHD confirmed by two different GH stimulation tests performed at screening or within 12 months prior to screening.
✓. No prior exposure to GH or IGF-1therapy.
✓. Absence of intracranial tumor, as confirmed by MRI or CT. Images or scans obtained within 1 year prior to screening can be used as screening data if accompanied by a medical evaluation and conclusion.
✓. AHV \<5 cm/year at screening (Phase II only);
. Presence of one or more pituitary hormone deficiencies in addition to growth hormone deficiency.
✕. Any suspected or known disease likely to affect growth, or any clinically significant abnormality that would preclude the accurate assessment of standing height (Phase II only), including but not limited to:
✕. Epiphyseal closure (Phase II only).
✕. Abnormal liver function, renal function, or coagulation profile
✕. Current or prior history of any malignant disease; or a family history of malignancy.
✕. Presence of impaired glucose metabolism, or HbA1c ≥ 5.7%, or a confirmed diagnosis of diabetes mellitus.
✕. Clear medical history of cardiovascular, hepatic, renal, gastrointestinal, respiratory, hematological, neurological, or metabolic disorders, or any other condition that, in the opinion of the investigator, makes the participant unsuitable for participation in the study.
✕. Any clinically significant abnormality in vital signs, physical examinations, laboratory tests, 12-lead ECG, full spine anteroposterior and lateral X-ray, or B-mode ultrasound, other than those associated with the study disease, as judged by the investigator and will make the participant unsuitable for the study.