A Study to Investigate Efficacy and Safety of Weekly PEG-somatropin (GenSci004) in Treatment Naive Children With Growth Hormone Deficiency

Phase 3

Not yet recruiting

• Conditions: GHD
• Interventions: Drug: GenSci004; Drug: Genotropin

• Registration Number: NCT06007417
• Lead Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of weekly GenSci004 compared with daily Genotropin in treatment-naive children with growth failure due to GHD.

Detailed Description

The purpose of this Phase 3 study is to evaluate the efficacy, safety, and tolerability of weekly GenSci004 compared to daily Genotropin over 52 weeks in prepubertal treatment-naïve children with growth failure due to GHD.

Study Timeline

• Study First Submitted: 2023-08-09
• Study First Submitted QC: 2023-08-22
• Study First Posted: 2023-08-23
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-07
• Last Update Posted: 2023-09-11
• Study Start: 2023-12-01
• Primary Completion: 2025-05-01
• Study Completion: 2025-08-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 162

Inclusion Criteria

1. Prepubertal children with GHD in Tanner Stage 1
2. Baseline HT at least -2.0 SD below the mean HT for CA and sex (HT SDS ≤ 2.0).
3. Body mass index (BMI) within ±2.0 SD of the mean BMI for BA and sex.
4. Growth hormone stimulation tests: ≤10 ng/mL
5. Baseline IGF 1 level of at least 1.0 SD below the mean IGF 1 level standardized for age and sex (IGF 1 SDS ≤-1.0)
6. Normal 46 XX karyotype for girls.
7. Children with multiple hormonal deficiencies must be on stable replacement therapy for other hypothalamo-pituitary axes for at least 3 months
8. Written, signed informed consent of the parent(s) or legal guardian(s) of the participant and written assent of the participant

Exclusion Criteria

1. BA≥CA
2. Prior exposure to rhGH, long-acting growth hormones, or IGF 1 therapy.
3. Major medical conditions or presence of contraindication to human growth hormone (hGH) treatment
4. Participation in any other trial of an investigational agent within 3 months prior to Screening.
5. Any reason per investigator's discretion

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
GenSci004	GenSci004	-
Genotropin	Genotropin	-

Primary Outcome Measures

Name	Time	Method
Annualized Height Velocity (AHV) for GenSci004 and Genotropin groups	52 weeks	Measured in centimeter per year (cm/year)

Secondary Outcome Measures

Name	Time	Method
Annualized Height Velocity (AHV) for GenSci004 and Genotropin groups	104 weeks	Measured in centimeter per year (cm/year)

Trial Locations

Locations (1)

Cook Childrens; 🇺🇸 Fort Worth, Texas, United States