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

NCT04513171 | Completed Phase 2

• 1 other identifier: TB1805GH
• Study Type: interventional
• Target: 434
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Growth Hormone Deficiency

Keywords

Growth Hormone Deficiency; somatropin; recombinated human growth hormone; Growth Hormone

Outcome Measures

Primary Outcomes (2)

• Phase 2: Change of areas under the curve of IGF-1 concentration from baseline (ΔIGF-1 AUC).

  12 weeks

• Phase 3: Height velocity.

  52 weeks

Secondary Outcomes (4)

• Change of height standard deviation score according to chronological age.

  52 weeks

• Change of height standard deviation score according to bone age.

  52 weeks

• Serum IGF-l level

  change from baseline to 52 weeks

• Serum IGFBP-3 level

  change from baseline to 52 weeks

Study Arms (6)

Y-shape pegylated somatropin low dose

EXPERIMENTAL

Drug: Y-shape pegylated somatropin

Y-shape pegylated somatropin middle dose

EXPERIMENTAL

Drug: Y-shape pegylated somatropin

Y-shape pegylated somatropin high dose

EXPERIMENTAL

Drug: Y-shape pegylated somatropin

Norditropin-1

ACTIVE COMPARATOR

Drug: Norditropin®

Y-shape pegylated somatropin optimal dose

EXPERIMENTAL

Drug: Y-shape pegylated somatropin

Norditropin-2

ACTIVE COMPARATOR

Drug: Norditropin

Interventions

Y-shape pegylated somatropin DRUG

Y-shape pegylated somatropin 100μg/kg, subcutaneous injection, Once weekly.

Y-shape pegylated somatropin low dose

Norditropin DRUG

Norditropin 245μg/kg/week, subcutaneous injection, Once daily.

Norditropin-2

Eligibility Criteria

• Age: 3 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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 Ⅰ) 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Xiamen Amoytop Biotech Co., Ltd.: lead
• Tongji Hospital: collaborator

Study Sites (1)

Tongji Hospital, Tongji Medical College of HUST

Wuhan, Hubei, China

Location

Related Publications (1)

• Liang Y, Zhang C, Wei H, Du H, Zhang G, Yang Y, Zhang H, Gong H, Li P, Song F, Xu Z, He R, Zhou W, Zheng H, Sun L, Luo X. The pharmacokinetic and pharmacodynamic properties and short-term outcome of a novel once-weekly PEGylated recombinant human growth hormone for children with growth hormone deficiency. Front Endocrinol (Lausanne). 2022 Aug 11;13:922304. doi: 10.3389/fendo.2022.922304. eCollection 2022.

  PMID: 36034448 DERIVED

MeSH Terms

Conditions

Dwarfism, Pituitary

Interventions

Human 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)

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

Study Officials

• Xiaoping Luo, MD, Ph.D

  Tongji Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 7, 2020
• First Posted: August 14, 2020
• Study Start: December 26, 2018
• Primary Completion: June 7, 2023
• Study Completion: July 10, 2023
• Last Updated: January 24, 2024

Record last verified: 2024-01

Locations

CN (1)