NCT07126288

Evaluating the Efficacy and Safety of GB08 Injection in Pediatric Patients With Growth Hormone Deficiency

A Phase II/III, Seamless, Multicenter, Randomized, Open-Label, Positive-Comparator Controlled Clinical Trial to Evaluate the Efficacy and Safety of GB08 Injection in Pediatric Patients With Growth Hormone Deficiency

• Status: Not Yet Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 268 (estimated)
• Sponsor: Shenzhen Kexing Pharmaceutical Co., Ltd. · Network
• Sex: All
• Age: 3 Years – 11 Years
• Healthy volunteers: Not accepted

Summary

This study aims to evaluate the efficacy and safety of GB08 injection compared to Norditropin NordiFlex in pediatric patients with growth hormone deficiency (PGHD). It seeks to resolve the following questions: * 1: Does GB08 injection demonstrate comparable efficacy in treating PGHD at 24 weeks compared to Norditropin NordiFlex? * 2: Which dose (0.4 mg/kg, 0.8 mg/kg, and 1.2 mg/kg) of GB08 injection best balances efficacy and safety in treating PGHD at 24 weeks? * 3: Does GB08 injection maintain its efficacy in treating PGHD at 52 weeks compared to Norditropin NordiFlex? To achieve these, GB08 injection will be compared to Norditropin NordiFlex to see if it provides a more effective or safer treatment option for PGHD. This is a Phase II/III, Seamless, Multicenter, Randomized, Open-Label, Positive-Comparator Controlled Clinical Trial with two stages. Stage 1 answers questions #1 and #2 by comparing the efficacy and safety of GB08 injection and Norditropin NordiFlex intervention among PGHD at 24 weeks. It involves four groups (n=16 each): GB08 0.4 mg/kg, GB08 0.8 mg/kg, GB08 1.2 mg/kg, and Norditropin NordiFlex 0.035 mg/kg. GB08 and Norditropin NordiFlex will be administered once weekly and once daily, respectively. The primary outcome measurement is annualized height velocity (AHV) at 24 weeks. Other measurements include growth hormone levels, safety parameters, immunogenicity markers, and pharmacokinetic/pharmacodynamic profiles. The optimal GB08 dose will be further investigated in Stage 2, which answers question #3. At this stage, PGHD patients will randomly receive either GB08 injection or Norditropin NordiFlex intervention for 52 weeks (n=102 for each). After that, the efficacy and safety of GB08 will also be detected.

Detailed description

This is a multicenter, randomized, open-label, positive-controlled Phase II/III seamless design clinical study evaluating the efficacy and safety of GB08 injection in pediatric patients with growth hormone deficiency (PGHD). It aims to compare the efficacy and safety of GB08 injection with Norditropin NordiFlex in PGHD patients, thereby benefiting this population. This study involves two parts: Phase II and Phase III. Phase II: Identifying the Optimal Dose This part assesses the efficacy and safety of three doses of GB08 in treating PGHD to recommend the optimal dose for the phase III. Eligible patients with PGHD will be randomly divided into four groups (n=16 per group) and stratified by GH peak value (≤5 μg/L and \>5 μg/L). They will receive GB08 0.4 mg/kg, GB08 0.8 mg/kg, GB08 1.2 mg/kg, or Norditropin NordiFlex 0.035 mg/kg (positive control) treatment for 24 weeks with another 28 weeks of follow-up. GB08 and Norditropin NordiFlex therapy will be provided once weekly and once daily, respectively. All participants will have blood samples collected for pharmacokinetics (GB08 groups only), pharmacodynamics, immunogenicity, and safety and efficacy assessments, as well as records of concomitant medications/treatments and adverse events. The primary outcome measurement is the annualized height velocity (AHV) at 24 weeks among the four groups. The optimal GB08 dose will be identified based on the 24-week data. Phase III: The 52-week efficacy and safety of GB08 injection compared to Norditropin NordiFlex This part aims to evaluate the efficacy of GB08 injection compared to Norditropin NordiFlex in PGHD patients at 52 weeks. It contains a treatment group (GB08 injection) and a control group (Norditropin NordiFlex), each with 102 participants. Accordingly, they will receive GB08 or Norditropin NordiFlex intervention for 52 weeks. The dose of GB08 will be determined by Phase II. Stratified randomization will be based on age (\<6 years vs. ≥6 years), gender (male vs. female), and GH peak value (≤ 5 ug/L, 5-7 ug/L, \>7 ug/L). The treatment period will last for 52 weeks with a 2-week follow-up. The primary endpoint measurement is the efficacy of GB08 injection at the 52nd week compared to Norditropin NordiFlex.

Conditions

• Growth Hormone Deficiency in Children
• Growth Hormone Deficiency (GHD)
• Growth Hormone

Interventions

Type	Name	Description
DRUG	GB08	In Phase II trial, eligible PGHD patients will receive GB08 0.4mg/kg, 0.8mg/kg, or 1.2mg/kg subcutaneous injection, once a week for 24 weeks. In Phase III trial, the individuals will receive GB08 subcutaneous injection, once a week for 52 weeks. The dose of GB08 at this stage will be determined by the outcomes of Phase II.
DRUG	Norditropin NordiFlex	Norditropin NordiFlex 0.035 mg/kg subcutaneous injection, daily, for 24 weeks (Phase II) or 52 weeks (Phase III)

Timeline

• Start date: 2025-08-31
• Primary completion: 2028-09-15
• Completion: 2028-12-23
• First posted: 2025-08-17
• Last updated: 2025-08-22

Locations

16 sites across 1 country: China