NCT07450053
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 Idiopathic Short Stature
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: Incidence of 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 study to evaluate the safety, tolerability, PK/PD profile, and immunogenicity of a single subcutaneous dose of GenSci134 in children with idiopathic short stature (ISS).
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 ISS. It will also evaluate PK/PD profile, immunogenicity, and biomarkers 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
- •sInformed 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:
- •Phase Ib:
- •Girls: age ≥3 and ≤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.
- •● Phase II: Girls: age ≥3 and ≤9 years, breast development at Tanner stage 1; Boys: age ≥3 and ≤10 years, testis volume \<4 mL.
- •Diagnosis of ISS at the time of ICF signing .
- •BMI within the range of ±2 SD of the mean BMI for age and sex at screening（Phase II only）.
- •No prior exposure to GH or IGF-1 therapy.
- •Historical measurements of body height within 6-18 months prior to screening are available(Phase II only).
- •BA-CA ≤ 1 year at screening(Phase II only).
Exclusion Criteria
- •Presence of any suspected or confirmed condition known to affect growth, 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.
- •Epiphyseal closure (Phase II only).
Arms & Interventions
Recombinant Human Growth Hormone Injection (Norditropin®)
Active Comparator
Active control group
Intervention: Recombinant Human Growth Hormone Injection(Norditropin® FlexPro®) (Drug)
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)
Outcomes
Primary Outcomes
Phase Ib: Incidence of Treatment Emergent Adverse Events （TEAEs）
Time Frame: From Day 1 to Day 35
Phase II: Annualized height velocity (AHV) at Week 24 of treatment
Time Frame: 24 weeks
Secondary Outcomes
- Phase II: Change from baseline in BA/CA at each visit(From baseline to Week 24 of the treatment period)
- Phase II: Incidence of TEAEs(From the first dose to the end of the trial)
- Phase II: Serum concentration of GenSci134.(From baseline to Week 24 of the treatment period)
- Phase II: Serum level of IGF-1and IGFBP-3 and their changes from baseline.(From baseline to Week 24 of the treatment period)
- Phase II: Incidence and timing of positive ADA and/or NAb (if applicable).(From baseline to Week 24 of the extension period)
- 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 II: Change from baseline in HT SDS at each visit(From baseline to Week 24 of the treatment period)
- 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 Ib: Half-life (t1/2) of GenSci134(From Day 1 to Day 29)
- Phase Ib: Serum level of IGF-1and IGFBP-3 and their changes from baseline.(rom Day 1 to Day 29)
- Phase II: Change from baseline in AHV at each visit(From baseline to Week 24 of the treatment period)
Investigators
Study Sites (1)
Loading locations...
Similar Trials
Not yet recruiting
Phase 1
JSKN016 in Combination With D-0502 for Locally Advanced or Metastatic HR-Positive, HER2-Negative Breast CancerNCT07336771Jiangsu Alphamab Biopharmaceuticals Co., Ltd60
Recruiting
Not Applicable
A Study Comparing the Efficacy and Safety of HRS9531 Injection With Semaglutide Injection in Subjects With ObesityNCT07285902Fujian Shengdi Pharmaceutical Co., Ltd.750
Recruiting
Phase 3
A Phase III Study Comparing HRS-4357 With Novel Androgen Receptor Pathway Inhibitors in Patients With Progressive, PSMA-Positive Metastatic Castration-Resistant Prostate CancerNCT07311694Jiangsu HengRui Medicine Co., Ltd.370
Not yet recruiting
Phase 3
A Phase III Study of KN026 in Combination With HB1801 as Adjuvant Therapy for Resectable HER2-Positive Breast CancerNCT07441460Shanghai JMT-Bio Inc.1,800
Not yet recruiting
Phase 2
Glofitamab as a Bridge to and/or Consolidation Post Autologous Stem Cell Transplant in Patients With Relapsed B Cell LymphomasNCT07387848American University of Beirut Medical Center40