Safety and Efficacy of Y-shape Pegylated Somatropin in Growth Hormone Deficiency Children

Growth Hormone Deficiency Clinical Trial

Official title:

A Multi-center, Randomized, Positive-control, Phase 2&3 Combined Study of Y-shape Pegylated Somatropin in Prepubertal Children With Growth Hormone Deficiency.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04513171
• Other study ID #: TB1805GH
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: December 26, 2018
• Est. completion date: July 10, 2023

Study information

• Verified date: January 2024
• Source: Xiamen Amoytop Biotech Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a multicenter, randomized, open-labeled, positive controlled phase 2&3 combined study to evaluate the safety and efficacy of weekly Y-shape pegylated somatropin, compared to daily somatropin (Norditropin®), in prepubertal, treatment-naive children with growth hormone deficiency.

Description:

This multicenter, randomized, open-labeled, positive controlled study is divided into two stages. The first one is aimed to exploit the optimal dose of Y-shape pegylated somatropin, while the second one is aimed to confirm the efficacy and safety of the study drug. A total of 400 prepubertal children with growth hormone deficiency were expected to enrolled. Subjects will firstly undergo a 52 weeks treatment, and then followed for 5 weeks.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 434
• Est. completion date: July 10, 2023
• Est. primary completion date: June 7, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 11 Years

Eligibility

Inclusion Criteria:

• Diagnosis of GHD confirmed by two different GH stimulation tests, defined as a peak of GH level of <10.0 ng/ml, determined with a validated assay. Bone age (BA) at least 2 years less than the chronological age. Growth velocity less than 5.0 cm/year. Impaired HT defined as at least 2.0 standard deviations (SD) below of the mean height for chronological age and sex (HT SDS<-2.0).

• Prepubertal (Tanner ?) males and females by physical examination, aged older than 3 years and younger than10 years for girls and 11 years for boys.

• Short stature with normal intelligence.

• Baseline IGF-1 level below the median IGF-1 level standardized for age and sex.

• Written, signed informed consent of the parent(s) or legal guardian(s) of the subject and written assent of the subject (if the subject is 8 years old or above).

Exclusion Criteria:

• Prior exposure to growth promotion treatment, such as recombinant human growth hormone or gonadal hormones, for more than 1 month.

• Known hypersensitivity to somatropin or excipients, such as mannitol, lysine, sodium chloride.

• Children with closed epiphyses.

• Short stature etiologies other than GHD, such as idiopathic short stature, Turner syndrome, Prader-Willi syndrome, Russell-Silver syndrome, born small for gestational age regardless of GH status.

• Other causes of short stature such as hypothyroidism, adrenocortical hormone deficiency, antidiuretic hormone deficiency.

• Any medical conditions and/or presence that may affect growth velocity such as liver dysfunction, kidney dysfunction, malnutrition, diabetes mellitus, severe dysfunction in major organ such as heart, sever systemic infections, severe immune dysfunction, mental disorders, and other congenital malformations.

• Suffering from chronic infectious diseases such as chronic hepatitis B, AIDS or tuberculosis.

• Receiving non-physiological adrenal corticosteroids.

• Confirmed pituitary and/or hypothalamic malignance by MRI within one year prior to screening. History or presence of any other malignance disease, any evidence of present tumor growth.

• Evidence of congenital intracranial hypertension.

• Evidence of slipped capital femoral epiphysis.

• Evidence of scoliosis over 15°.

• Participation in any other trial of an investigational agent within 3 months prior to screening.

• Any other conditions which in the opinion of the investigator precluded enrollment into the study.

Related Conditions & MeSH terms

• Dwarfism, Pituitary
• Endocrine System Diseases
• Growth Hormone Deficiency

Intervention

Drug:
• Y-shape pegylated somatropin
Y-shape pegylated somatropin 100µg/kg, subcutaneous injection, Once weekly.
• Y-shape pegylated somatropin
Y-shape pegylated somatropin 120µg/kg, subcutaneous injection, Once weekly.
• Y-shape pegylated somatropin
Y-shape pegylated somatropin 140µg/kg, subcutaneous injection, Once weekly.
• Norditropin®
Norditropin 245µg/kg/week, subcutaneous injection, Once daily.
• Y-shape pegylated somatropin
Y-shape pegylated somatropin 140µg/kg, subcutaneous injection, Once weekly.
• Norditropin
Norditropin 245µg/kg/week, subcutaneous injection, Once daily.

Locations

Country	Name	City	State
China	Tongji Hospital, Tongji Medical College of HUST	Wuhan	Hubei

Sponsors (2)

Lead Sponsor	Collaborator
Xiamen Amoytop Biotech Co., Ltd.	Tongji Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Phase 2: Change of areas under the curve of IGF-1 concentration from baseline (?IGF-1 AUC).		12 weeks
Primary	Phase 3: Height velocity.		52 weeks
Secondary	Change of height standard deviation score according to chronological age.		52 weeks
Secondary	Change of height standard deviation score according to bone age.		52 weeks
Secondary	Serum IGF-l level		change from baseline to 52 weeks
Secondary	Serum IGFBP-3 level		change from baseline to 52 weeks