A Study (Phase 1b/2) of GenSci134 in Children With Idiopathic Short Stature (ISS)

NCT07450053 | Not Yet Recruiting Phase 1

• 1 other identifier: GenSci134-203
• Study Type: interventional
• Target: 128
• Locations: 1 country
• Sites: 1

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.

Conditions

Idiopathic Short Stature

Keywords

ISS; GenSci134

Outcome Measures

Primary Outcomes (2)

• Phase Ib: Incidence of Treatment Emergent Adverse Events （TEAEs）

  From Day 1 to Day 35

• Phase II: Annualized height velocity (AHV) at Week 24 of treatment

  24 weeks

Secondary Outcomes (13)

• 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

Study Arms (2)

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

Drug: GenSci134 Injection

Recombinant Human Growth Hormone Injection (Norditropin®)

ACTIVE COMPARATOR

Active control group

Drug: Recombinant Human Growth Hormone Injection(Norditropin® FlexPro®)

Interventions

GenSci134 Injection DRUG

only one dose of GenSci134 to be given, subcutaneous , 6 dose levels will be assigned.

Also known as: GenSci134

Dose Level 1~ Dose Level 6

Recombinant Human Growth Hormone Injection(Norditropin® FlexPro®) DRUG

multiple doses of Norditropin® FlexPro® quaque die (QD) for 28 consecutive days by subcutaneous injections.

Also known as: Norditropin®

Recombinant Human Growth Hormone Injection (Norditropin®)

Eligibility Criteria

• Age: 3 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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).

You may not qualify if:

• Presence of any suspected or confirmed condition known to affect growth, including but not limited to:
• GHD.
• 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).
• 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.

Sponsors & Collaborators

• Changchun GeneScience Pharmaceutical Co., Ltd.: lead

Study Sites (1)

Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology

Wuhan, Hubei, 430000, China

Location

MeSH Terms

Interventions

Human Growth Hormone

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Central Study Contacts

Jinbo Li

CONTACT

+86 15001322766; lijinbo@genscigroup.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 12, 2026
• First Posted: March 4, 2026
• Study Start: March 6, 2026
• Primary Completion (Estimated): July 18, 2028
• Study Completion (Estimated): December 31, 2028
• Last Updated: March 4, 2026

Record last verified: 2026-02

Locations

CN (1)