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

NCT ID: NCT07126288

Last Updated: 2025-08-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

268 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-08-31

Study Completion Date

2028-12-23

Brief Summary

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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

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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

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Phase II/III seamless design clinical study
Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors

Study Groups

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PhaseII GB08 0.4 mg/kg

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

Group Type EXPERIMENTAL

GB08

Intervention Type 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 0.8 mg/kg

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

Group Type EXPERIMENTAL

GB08

Intervention Type 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 1.2 mg/kg

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

Group Type EXPERIMENTAL

GB08

Intervention Type 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 Norditropin NordiFlex

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

Group Type ACTIVE_COMPARATOR

Norditropin NordiFlex

Intervention Type DRUG

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

PhaseIII GB08

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

Group Type EXPERIMENTAL

GB08

Intervention Type 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.

PhaseIII Norditropin NordiFlex

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

Group Type ACTIVE_COMPARATOR

Norditropin NordiFlex

Intervention Type DRUG

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

Interventions

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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.

Intervention Type DRUG

Norditropin NordiFlex

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

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1\. 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:

1. 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.
2. Annualized height velocity (AHV) ≤ 5.0 cm/year, calculated from measurement of 6 to 18 months before screening.
3. GH peak ≤ 10 ng/ml proved by two different GH stimulation tests within a year before screening.
4. 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.

2\. 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).

5\. Body mass index (BMI) within ±2 SD of the mean BMI for children and adolescents of the same age and sex.

6\. For subjects with GHD as part of multiple pituitary hormone deficiencies, must be stable for ≥1 month, as determined by the investigator.

7\. 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.

Exclusion Criteria

1. History of systematic growth-promoting therapy, including growth hormone and sex hormones.
2. Severe allergic constitution or known allergy to growth hormone or its excipients, such as mannitol, lysine, or sodium chloride.
3. Closed epiphyses.
4. Other types of growth disorders such as idiopathic short stature, Turner syndrome, Noonan syndrome, Prader-Willi syndrome, and Russell-Silver syndrome.
5. 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.
6. 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.
7. 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).
8. Use of corticosteroids or other steroids within the past 12 months, such as long-term steroid use for asthma.
9. History of pituitary or hypothalamic tumors, or other intracranial tumors on MRI; history of leukemia, lymphoma, or other malignancies.
10. History of radiation therapy or chemotherapy.
11. Congenital intracranial hypertension.
12. Femoral head slipped epiphysis (SCFE).
13. Spinal scoliosis \> 15°.
14. Participation in any other drug clinical trial within the past 3 months (as a subject).
15. Other factors deemed unsuitable for participation in the study by the investigator.
Minimum Eligible Age

3 Years

Maximum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shenzhen Kexing Pharmaceutical Co., Ltd.

NETWORK

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Junfen Fu

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Zhejiang University School of Medicine

Yijia Zhang

Role: STUDY_DIRECTOR

Shenzhen Kexing Pharmaceutical Co., Ltd.

Locations

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Xiamen Maternal and Child Health Hospital

Xiamen, Fujian, China

Site Status

Nanyang Central Hospital

Nanyang, Henan, China

Site Status

The First Affiliated Hospital of Nanyang Medical College

Nanyang, Henan, China

Site Status

The Third Affiliated Hospital of Xinxiang Medical University

Xinxiang, Henan, China

Site Status

Wuhan Children's Hospital

Wuhan, Hubei, China

Site Status

Suzhou University Children's Hospital

Suzhou, Jiangsu, China

Site Status

Jiangxi Children's Hospital

Nanchang, Jiangxi, China

Site Status

Pingxiang Maternal and Child Health Hospital

Pingxiang, Jiangxi, China

Site Status

The First Hospital of Jilin University

Changchun, Jilin, China

Site Status

Linyi Maternal and Child Health Hospital

Linyi, Shandong, China

Site Status

Chengdu Women's and Children's Central Hospital

Chengdu, Sichuan, China

Site Status

Meishan People's Hospital

Meishan, Sichuan, China

Site Status

The Second People's Hospital of Yibin

Yibin, Sichuan, China

Site Status

Zhejiang Provincial People's Hospital

Hangzhou, Zhejiang, China

Site Status

Children's Hospital of Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Site Status

Ningbo Women's and Children's Hospital

Ningbo, Zhejiang, China

Site Status

Countries

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China

Central Contacts

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Yanqing Lin

Role: CONTACT

86-0755-23018589

Jinhai Lin

Role: CONTACT

86-13760806104

References

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Christiansen JS, Backeljauw PF, Bidlingmaier M, Biller BM, Boguszewski MC, Casanueva FF, Chanson P, Chatelain P, Choong CS, Clemmons DR, Cohen LE, Cohen P, Frystyk J, Grimberg A, Hasegawa Y, Haymond MW, Ho K, Hoffman AR, Holly JM, Horikawa R, Hoybye C, Jorgensen JO, Johannsson G, Juul A, Katznelson L, Kopchick JJ, Lee KO, Lee KW, Luo X, Melmed S, Miller BS, Misra M, Popovic V, Rosenfeld RG, Ross J, Ross RJ, Saenger P, Strasburger CJ, Thorner MO, Werner H, Yuen K. Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations. Eur J Endocrinol. 2016 Jun;174(6):C1-8. doi: 10.1530/EJE-16-0111. Epub 2016 Mar 23.

Reference Type BACKGROUND
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Rosenfeld RG, Bakker B. Compliance and persistence in pediatric and adult patients receiving growth hormone therapy. Endocr Pract. 2008 Mar;14(2):143-54. doi: 10.4158/EP.14.2.143.

Reference Type BACKGROUND
PMID: 18308651 (View on PubMed)

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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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Cunningham BC, Ultsch M, De Vos AM, Mulkerrin MG, Clauser KR, Wells JA. Dimerization of the extracellular domain of the human growth hormone receptor by a single hormone molecule. Science. 1991 Nov 8;254(5033):821-5. doi: 10.1126/science.1948064.

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PMID: 1948064 (View on PubMed)

Kopchick JJ, Parkinson C, Stevens EC, Trainer PJ. Growth hormone receptor antagonists: discovery, development, and use in patients with acromegaly. Endocr Rev. 2002 Oct;23(5):623-46. doi: 10.1210/er.2001-0022.

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DOI:10.3969/j.issn.2095-1736.2020.05.103.

Reference Type BACKGROUND

de Fries Jensen L, Antavalis V, Odgaard-Jensen J, Rossi A, Pietropoli A, Hojby M. Efficacy and Safety of Somapacitan Relative to Somatrogon and Lonapegsomatropin in Pediatric Growth Hormone Deficiency: Systematic Literature Review and Network Meta-analysis. Adv Ther. 2024 Nov;41(11):4098-4124. doi: 10.1007/s12325-024-02966-y. Epub 2024 Sep 11.

Reference Type BACKGROUND
PMID: 39261416 (View on PubMed)

Other Identifiers

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KXZY-GB08-201

Identifier Type: -

Identifier Source: org_study_id

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