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

NCT07126288 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: KXZY-GB08-201
• Study Type: interventional
• Target: 268
• Locations: 1 country
• Sites: 16

Brief 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.

Conditions

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

Keywords

growth hormone deficiency; annualized height velocity; growth hormone; clinical trial; GB08 injection

Outcome Measures

Primary Outcomes (2)

• PhaseII-Comparison of annualized height velocity at 24 weeks

  Comparison of annualized height velocity of PGHD patients receiving 24-week GB08 and Norditropin NordiFlex treatment

  From enrollment to end of treatment at 24 weeks

• PhaseIII-Comparison of annualized height velocity at week 52

  Comparison of annualized height velocity between PGHD patients receiving GB08 and Norditropin NordiFlex at 52 weeks

  From enrollment to end of treatment at week 52

Secondary Outcomes (24)

• PhaseII-annualized height velocity at weeks 4, 12, 36, 48, 52

  From enrollment to weeks 4, 12, 36, 48, 52

• PhaseII-Standard deviation (SD) scores of AHV of GB08 injection and Norditropin NordiFlex at baseline, and at weeks 4, 12, 36, 48, 52

  From enrollment to weeks 4, 12, 36, 48, 52

• PhaseII-Standard deviation of actual height SDS (Ht SDS CA) of GB08 injection and Norditropin NordiFlex at weeks 4, 12, 24, 36, 48, 52

  From enrollment to weeks 4, 12, 24, 36, 48, 52

• PhaseII-Standard deviation of bone age (BA) SDS (Ht SDS BA) of GB08 injection and Norditropin NordiFlex at week 24 and 52

  From enrollment to weeks 24 and 52

• PhaseII-Tanner stage changes at baseline and at weeks 12, 24, 52

  From enrollment to weeks 12, 24, and 52

Study Arms (6)

PhaseII GB08 0.4 mg/kg

EXPERIMENTAL

GB08 0.4mg/kg subcutaneous injection, once a week for 24 weeks

Drug: GB08

PhaseII GB08 0.8 mg/kg

EXPERIMENTAL

GB08 0.8mg/kg subcutaneous injection, once a week for 24 weeks

Drug: GB08

PhaseII GB08 1.2 mg/kg

EXPERIMENTAL

GB08 1.2mg/kg subcutaneous injection, once a week for 24 weeks

Drug: GB08

PhaseII Norditropin NordiFlex

ACTIVE COMPARATOR

Norditropin NordiFlex 0.035 mg/kg subcutaneous injection, daily, for 24 weeks

Drug: Norditropin NordiFlex

PhaseIII GB08

EXPERIMENTAL

GB08 subcutaneous injection (The dose will be determined by data from PhaseII), once a week for 52 weeks

Drug: GB08

PhaseIII Norditropin NordiFlex

ACTIVE COMPARATOR

Norditropin NordiFlex 0.035 mg/kg subcutaneous injection, daily, for 52 weeks

Drug: Norditropin NordiFlex

Interventions

GB08 DRUG

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.

PhaseII GB08 0.4 mg/kg; PhaseII GB08 0.8 mg/kg; PhaseII GB08 1.2 mg/kg; PhaseIII GB08

Norditropin NordiFlex DRUG

Norditropin NordiFlex 0.035 mg/kg subcutaneous injection, daily, for 24 weeks (Phase II) or 52 weeks (Phase III)

PhaseII Norditropin NordiFlex; PhaseIII Norditropin NordiFlex

Eligibility Criteria

• Age: 3 Years - 11 Years
• Gender Eligibility Details: males: 3-11 years old; females: 3-10 years old
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• \. Diagnosed with Growth Hormone Deficiency (GHD) based on medical history, clinical symptoms and signs, GH stimulation tests, and imaging studies. The participant must meet the following:
• Absolute height 2 standard deviations (SD) below the mean height of children of the same age and sex, according to the standardized growth curves for children and adolescents in China (0-18 years old) published in 2009.
• Annualized height velocity (AHV) ≤ 5.0 cm/year, calculated from measurement of 6 to 18 months before screening.
• GH peak ≤ 10 ng/ml proved by two different GH stimulation tests within a year before screening.
• Bone age lagging at least 1 year behind actual age (bone age assessment within the past 6 months before screening), with girls \< 10 years old and boys \< 11 years old.
• \. Aging \> 3 years and ≤ 10 years (girls) or ≤ 11 years (boys) based on birth date; Tanner stage I (testicular volume \< 4 ml for boys and no palpable breast tissue for girls) 3. Uniform short stature with normal intellectual development. 4. IGF-1 levels below the mean for children and adolescents of the same age and sex, at least 1 SD below (IGF-1 SDS ≤ -1.0).
• \. Body mass index (BMI) within ±2 SD of the mean BMI for children and adolescents of the same age and sex.
• \. For subjects with GHD as part of multiple pituitary hormone deficiencies, must be stable for ≥1 month, as determined by the investigator.
• \. Guardians understand and sign the Informed Consent Form (ICF). If the participant is ≥8 years old, they must also sign the ICF. For participants \<8 years old who can express consent, their consent must be recorded.

You may not qualify if:

• History of systematic growth-promoting therapy, including growth hormone and sex hormones.
• Severe allergic constitution or known allergy to growth hormone or its excipients, such as mannitol, lysine, or sodium chloride.
• Closed epiphyses.
• Other types of growth disorders such as idiopathic short stature, Turner syndrome, Noonan syndrome, Prader-Willi syndrome, and Russell-Silver syndrome.
• Short stature due to other causes, such as intrauterine growth restriction, familial short stature, thyroid hormone deficiency, adrenal insufficiency, antidiuretic hormone deficiency, celiac disease, rickets, psychological factors, chronic kidney disease, infections, or trauma.
• Any clinically significant abnormalities that may affect growth or growth assessment; subjects with liver or kidney dysfunction (ALT \> 1.5 times upper limit of normal, creatinine \> upper limit of normal), chronic diseases (malnutrition, fasting blood glucose ≥ 126 mg/dL or HbA1c ≥ 6.5%), diabetes, severe cardiac, pulmonary, hematological, or systemic infections, immunodeficiency, psychiatric disorders, or congenital malformations.
• Infectious diseases, such as hepatitis B, hepatitis C, AIDS, syphilis, or tuberculosis (HBV surface antigen-positive subjects must undergo HBV DNA testing; HCV antibody-positive subjects must undergo HCV RNA testing; if HBV DNA or HCV RNA \> detection limit, they are excluded).
• Use of corticosteroids or other steroids within the past 12 months, such as long-term steroid use for asthma.
• History of pituitary or hypothalamic tumors, or other intracranial tumors on MRI; history of leukemia, lymphoma, or other malignancies.
• History of radiation therapy or chemotherapy.
• Congenital intracranial hypertension.
• Femoral head slipped epiphysis (SCFE).
• Spinal scoliosis \> 15°.
• Participation in any other drug clinical trial within the past 3 months (as a subject).
• Other factors deemed unsuitable for participation in the study by the investigator.

Sponsors & Collaborators

• Shenzhen Kexing Pharmaceutical Co., Ltd.: lead

Study Sites (16)

Xiamen Maternal and Child Health Hospital

Xiamen, Fujian, 361000, China

Location

Nanyang Central Hospital

Nanyang, Henan, 473000, China

Location

The First Affiliated Hospital of Nanyang Medical College

Nanyang, Henan, 473000, China

Location

The Third Affiliated Hospital of Xinxiang Medical University

Xinxiang, Henan, 453003, China

Location

Wuhan Children's Hospital

Wuhan, Hubei, 430016, China

Location

Suzhou University Children's Hospital

Suzhou, Jiangsu, 215000, China

Location

Jiangxi Children's Hospital

Nanchang, Jiangxi, 330006, China

Location

Pingxiang Maternal and Child Health Hospital

Pingxiang, Jiangxi, 337000, China

Location

The First Hospital of Jilin University

Changchun, Jilin, 130021, China

Location

Linyi Maternal and Child Health Hospital

Linyi, Shandong, 276000, China

Location

Chengdu Women's and Children's Central Hospital

Chengdu, Sichuan, 610017, China

Location

Meishan People's Hospital

Meishan, Sichuan, 620010, China

Location

The Second People's Hospital of Yibin

Yibin, Sichuan, 644000, China

Location

Zhejiang Provincial People's Hospital

Hangzhou, Zhejiang, 310014, China

Location

Children's Hospital of Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310051, China

Location

Ningbo Women's and Children's Hospital

Ningbo, Zhejiang, 315010, China

Location

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• Yuen KCJ, Biller BMK, Radovick S, Carmichael JD, Jasim S, Pantalone KM, Hoffman AR. AMERICAN ASSOCIATION OF CLINICAL ENDOCRINOLOGISTS AND AMERICAN COLLEGE OF ENDOCRINOLOGY GUIDELINES FOR MANAGEMENT OF GROWTH HORMONE DEFICIENCY IN ADULTS AND PATIENTS TRANSITIONING FROM PEDIATRIC TO ADULT CARE. Endocr Pract. 2019 Nov;25(11):1191-1232. doi: 10.4158/GL-2019-0405.

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• Kim JH, Chae HW, Chin SO, Ku CR, Park KH, Lim DJ, Kim KJ, Lim JS, Kim G, Choi YM, Ahn SH, Jeon MJ, Hwangbo Y, Lee JH, Kim BK, Choi YJ, Lee KA, Moon SS, Ahn HY, Choi HS, Hong SM, Shin DY, Seo JA, Kim SH, Oh S, Yu SH, Kim BJ, Shin CH, Kim SW, Kim CH, Lee EJ. Diagnosis and Treatment of Growth Hormone Deficiency: A Position Statement from Korean Endocrine Society and Korean Society of Pediatric Endocrinology. Endocrinol Metab (Seoul). 2020 Jun;35(2):272-287. doi: 10.3803/EnM.2020.35.2.272. Epub 2020 Jun 24.

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MeSH Terms

Conditions

Dwarfism, Pituitary

Interventions

Growth Hormone

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

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

Study Officials

• Junfen Fu

  Children's Hospital of Zhejiang University School of Medicine

  PRINCIPAL INVESTIGATOR

• Yijia Zhang

  Shenzhen Kexing Pharmaceutical Co., Ltd.

  STUDY DIRECTOR

Central Study Contacts

Yanqing Lin

CONTACT

86-0755-23018589; linyanqing@kexing.com

Jinhai Lin

CONTACT

86-13760806104; linjinhai@kexing.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Model Details: Phase II/III seamless design clinical study

Study Record Dates

• First Submitted: August 10, 2025
• First Posted: August 17, 2025
• Study Start: August 31, 2025
• Primary Completion (Estimated): September 15, 2028
• Study Completion (Estimated): December 23, 2028
• Last Updated: August 22, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (16)