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 range
3 Years – 12 Years
Sex
ALL
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. 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);
Exclusion criteria
. 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.