A Study to Investigate the Safety and Efficacy of Once-weekly PEG-somatropin (GenSci004) in Children With Growth Hormone Deficiency (ESCALATE)

The Safety and Efficacy of Once-weekly PEG-somatropin (GenSci004) in Children With Growth Hormone Deficiency: An Open-label, Single-arm, Multicenter Phase 3 Study

The purpose of this Phase 3 study is to evaluate the safety and efficacy of once-weekly GenSci004 over 108 weeks in children with GHD aged from 6 months to 17 years old, inclusive.

Study Overview

• Status: Not yet recruiting
• Conditions: GHD
• Intervention / Treatment: Drug: PEG-somatropin

Detailed Description

The purpose of this Phase 3 study is to evaluate the safety and efficacy of once-weekly GenSci004 over 108 weeks in children with GHD aged from 6 months to 17 years old, inclusive.

• Study Type: Interventional
• Enrollment (Estimated): 180
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Duan Peng; Phone Number: +86-431-85195060; Email: info@gensci-china.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Investigator-determined diagnosis of GHD prior to the historical initiation of daily rhGH trerapy
2. Participant must be 6 months to 17 years old
3. Tanner stage <5 at Visit 1
4. Open epiphyses
5. Normal fundoscopy at Screening
6. Written, signed, informed consent of the participant's parent(s)/LAR(s) and written assent of the participant

Exclusion Criteria:

1. History or presence of malignant disease
2. Children with diabetes mellitus
3. Major medical conditions and/or presence of contraindications to GH treatment
4. Pregnancy
5. Participation in any other study of an investigational agent within three months prior to Visit 1
6. Prior exposure to investigational drug or any other long-acting growth hormone
7. Any other reason per investigator's discretion

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: GenSci004	Drug: PEG-somatropin; PEG-somatropin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Events of once-weekly GenSci004 in children with GHD	Incidents of adverse events	108 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized Height Velocity (AHV)	Measured in centimeter per year (cm/year)	104 weeks

Collaborators and Investigators

• Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.
• Investigators: Study Chair: Lawrence Silverman, MD, Morristown Medical Center

Study record dates

Study Major Dates

• Study Start (Estimated): December 1, 2023
• Primary Completion (Estimated): May 1, 2025
• Study Completion (Estimated): September 1, 2025

Study Registration Dates

• First Submitted: August 9, 2023
• First Submitted That Met QC Criteria: August 29, 2023
• First Posted (Actual): September 6, 2023

Study Record Updates

• Last Update Posted (Actual): September 11, 2023
• Last Update Submitted That Met QC Criteria: September 7, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: growth hormone deficiency; GHD
• Other Study ID Numbers: GenSci004-302

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes