NCT07450001
Not yet recruiting
Phase 1
A Phase Ib/II Multicenter, Randomized, Open-label, Active-controlled, Single/Multiple-dose, Dose-finding, Clinical Study of GenSci134 in Children With Growth Hormone Deficiency
Changchun GeneScience Pharmaceutical Co., Ltd.1 site in 1 country128 target enrollmentStarted: March 6, 2026Last updated:
Overview
- Phase
- Phase 1
- Status
- Not yet recruiting
- Enrollment
- 128
- Locations
- 1
- Primary Endpoint
- Phase Ib: Treatment Emergent Adverse Events （TEAEs）
Overview
Brief 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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 3 Years to 12 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
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:
- •Girls: age ≥3and ≤11 years, breast development at Tanner stage 1, body weight ≥16 kg; Boys: age ≥3 and ≤12 years, testis volume \<4 mL, body weight ≥16 kg.
- •Girls: age ≥3 and ≤9 years, breast development at Tanner stage 1; Boys: age ≥3 and ≤10 years, testis volume \<4 mL.
- •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);
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:
- •Turner syndrome
- •Noonan syndrome
- •Laron Syndrome
- •Other genetic syndromes with short stature that are caused by chromosomal abnormalities or gene mutations, including but not limited to Prader-Willi syndrome, abnormal SHOX-1 gene analysis, or GH receptor deficiency.
- •Born small for gestational age
- •Growth retardation due to malnutrition
- •Growth retardation due to hypothyroidism.
- •Short stature with any other clearly identified etiology.
Arms & Interventions
Dose Level 1~ Dose Level 6
Experimental
dose level 1、dose level 2、dose level 3、dose level 4、 dose level 5、dose level 6
Intervention: GenSci134 Injection (Drug)
Recombinant Human Growth Hormone Injection (Norditropin®)
Active Comparator
Active control group
Intervention: Recombinant Human Growth Hormone Injection(Norditropin® FlexPro®) (Drug)
Outcomes
Primary Outcomes
Phase Ib: Treatment Emergent Adverse Events （TEAEs）
Time Frame: From the first dose to Day 35
Phase II: Annualized height velocity (AHV) at Week 24 of treatment
Time Frame: 24 weeks
Secondary Outcomes
- Phase II: Incidence and timing of positive ADA and/or NAb (if applicable).(From baseline to Week 28 of the extension period)
- Phase II: Serum level of IGF-1and IGFBP-3 and their changes from baseline.(From baseline to Week 28 of the extension period)
- Phase II: Change from baseline in HT SDS at each visit(From baseline to Week 28 of the extension period)
- Phase II: Change from baseline in AHV at each visit(From baseline to Week 28 of the extension period)
- Phase II: Change from baseline in BA/CA at each visit.(From baseline to Week 28 of the extension period)
- Phase II: Treatment Emergent Adverse Events（TEAEs）(From the first dose to Week 29 of the extension period)
- Phase Ib: Half-life (t1/2) of GenSci134(From Day 1 to Day 29)
- Phase Ib: Serum level of IGF-1 and IGFBP-3 and their changes from baseline.(From Day 1 to Day 29)
- Phase Ib: Incidence and timing of positive anti-drug antibody (ADA) and/or neutralizing antibody (NAb) (if applicable)(From Day 1 to Day 29)
- Phase Ib: Areas under the drug concentration-time curve (AUC0-t, AUC0-∞) of GenSci134(From Day 1 to Day 29)
- Phase Ib: Time to maximum concentration (Tmax) of GenSci134(From Day 1 to Day 29)
- Phase Ib: Maximum concentration (Cmax) of GenSci134(From Day 1 to Day 29)
- Phase II: Serum concentration of GenSci134.(From baseline to Week 28 of the extension period)
Investigators
Study Sites (1)
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